Rare Disease Trial Updates: September 2026

In September 2026, Trial Friend tracked 895 rare disease clinical trial updates across 115 conditions, including 10 FDA-related actions and 162 trial status changes, alongside enrollment updates and new trial site openings sourced from ClinicalTrials.gov and FDA announcements.

This is a permanent monthly archive. For the latest updates, see the live current events feed.

Major Updates: FDA Actions, Status Changes & Results

Status ChangeChronic Graft-versus-Host DiseaseSep 30University of GiessenNCT06409624
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseSep 30University of Texas at AustinNCT05970341
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 30St. Olavs HospitalNCT06554886
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 30Virginia Commonwealth UniversityNCT05580419
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophySep 30Satellos Bioscience, Inc.NCT07287189
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDilated CardiomyopathySep 30Joshua M HareNCT04476901
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDiffuse Intrinsic Pontine GliomaSep 30University of FloridaNCT04837547
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 30Nanordica Medical OUNCT06667752
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 30Boston Children's HospitalNCT06611501
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 30The Danish Center for Expertise in RheumatologyNCT06299917
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 30Virginia Commonwealth UniversityNCT05548452
No longer enrolling new participants

No longer enrolling new participants

FDA NewsSep 29
FDA Investigation Leads to Charges Against Two Indian Nationals Involved in Transnational Counterfeit Drug Distribution Scheme

The U.S. Attorney’s Office for the Middle District of Florida and the Criminal Division of the U.S. Department of Justice today announced federal charges against Swapnadip Roy, 33, and Vicky Ramancha, 37, both Indian nationals.

Status ChangeBecker Muscular DystrophySep 29Satellos Bioscience, Inc.NCT07287189
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeSep 29Ionis Pharmaceuticals, Inc.NCT06914609
No longer enrolling new participants

No longer enrolling new participants

company_8kSep 29
Lexeo Therapeutics disclosed a material event (acquisition or disposition completed)

Lexeo Therapeutics (LXEO) filed an 8-K with the SEC on 2026-09-29 covering: acquisition or disposition completed. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 29
Cadrenal Therapeutics: CURRENT REPORT

Cadrenal Therapeutics (CVKD) filed an 8-K with the SEC on 2026-09-29 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kMyotonic DystrophySep 29
Dyne Therapeutics disclosed a material event involving Myotonic Dystrophy (company event)

Dyne Therapeutics (DYN) filed an 8-K with the SEC on 2026-09-29 covering: company event. The filing mentions Myotonic Dystrophy. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeAmyotrophic Lateral SclerosisSep 29Corcept TherapeuticsNCT05407324
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalMCT8 DeficiencySep 28
FDA Approves First Treatment for MCT8 Deficiency

The U.S. Food and Drug Administration today approved Emcitate (tiratricol) tablets for oral suspension to treat peripheral thyrotoxicosis (excess thyroid hormone levels in the blood that causes symptoms, such as rapid heart rate, increased blood pressure and adverse effects on metabolism) in patient

company_8kFibrodysplasia Ossificans ProgressivaSep 28
Mirum Pharmaceuticals disclosed a material event involving Fibrodysplasia Ossificans Progressiva (company event)

Mirum Pharmaceuticals (MIRM) filed an 8-K with the SEC on 2026-09-28 covering: company event. The filing mentions Fibrodysplasia Ossificans Progressiva. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeThyroid Eye DiseaseSep 27Minghui Pharmaceutical (Hangzhou) LtdNCT07257185
No longer enrolling new participants

No longer enrolling new participants

Status ChangeStargardt DiseaseSep 27Alkeus Pharmaceuticals, Inc.NCT02402660
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNoonan SyndromeSep 26BioMarin PharmaceuticalNCT06668805
No longer enrolling new participants

No longer enrolling new participants

company_8kSep 25
Stoke Therapeutics disclosed a material event (leadership change)

Stoke Therapeutics (STOK) filed an 8-K with the SEC on 2026-09-25 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeMyotonic DystrophySep 25Dyne TherapeuticsNCT05481879
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophySep 25Dyne TherapeuticsNCT05481879
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 25Aristotle University Of ThessalonikiNCT04806568
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 25TG Therapeutics, Inc.NCT06143514
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathySep 24Calliditas Therapeutics ABNCT06712407
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 24Sun Yat-sen UniversityNCT05949944
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgG4-Related DiseaseSep 24Tongji HospitalNCT07339540
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisSep 24Boehringer IngelheimNCT06238622
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHunter SyndromeSep 24AO GENERIUMNCT05208281
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 24Memorial Sloan Kettering Cancer CenterNCT06756308
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary AngioedemaSep 24Pharvaris Netherlands B.V.NCT06679881
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

company_8kSep 23
Corcept Therapeutics disclosed a material event (leadership change)

Corcept Therapeutics (CORT) filed an 8-K with the SEC on 2026-09-23 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kIgA NephropathySep 23
Ionis Pharmaceuticals: CURRENT REPORT FILING

Ionis Pharmaceuticals (IONS) filed an 8-K with the SEC on 2026-09-23 covering: company event. The filing mentions IgA Nephropathy. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 23
Jazz Pharmaceuticals disclosed a material event (material agreement)

Jazz Pharmaceuticals (JAZZ) filed an 8-K with the SEC on 2026-09-23 covering: material agreement. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 23
Sarepta Therapeutics disclosed a material event (material agreement)

Sarepta Therapeutics (SRPT) filed an 8-K with the SEC on 2026-09-23 covering: material agreement. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 23
Regenxbio disclosed a material event (material agreement)

Regenxbio (RGNX) filed an 8-K with the SEC on 2026-09-23 covering: material agreement. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeGeographic AtrophySep 23Astellas Pharma Global Development, Inc.NCT06779773
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFabry DiseaseSep 23General University Hospital, PragueNCT07495410
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeCystic FibrosisSep 22Children's Hospital Medical Center, CincinnatiNCT05517655
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22University of Texas at AustinNCT04277117
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22SanofiNCT06290128
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22AstraZenecaNCT06942910
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22University of ZurichNCT07524023
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Guangdong Provincial People's HospitalNCT04381143
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Children's Hospital of PhiladelphiaNCT07425691
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Children's Hospital Medical Center, CincinnatiNCT04715685
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22University of Illinois at ChicagoNCT07006818
No longer enrolling new participants

No longer enrolling new participants

company_8kSep 22
Lexeo Therapeutics disclosed a material event (material agreement, company event)

Lexeo Therapeutics (LXEO) filed an 8-K with the SEC on 2026-09-22 covering: material agreement, company event. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeDuchenne Muscular DystrophySep 22NS Pharma, Inc.NCT05996003
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophySep 22Insmed Gene Therapy LLCNCT06817382
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDravet SyndromeSep 22Longboard PharmaceuticalsNCT06660394
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisSep 22Jaeb Center for Health ResearchNCT06449677
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisSep 22Heidelberg UniversityNCT03925194
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Inflammatory Demyelinating PolyneuropathySep 22Rigshospitalet, DenmarkNCT06040567
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Tampere UniversityNCT06286917
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22David RuttensNCT06890767
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Northwestern UniversityNCT05719714
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Rigshospitalet, DenmarkNCT06040567
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Philip Morris Products S.A.NCT07108166
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22University of Illinois at ChicagoNCT06754280
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 22Jaseng Medical FoundationNCT06591221
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCharcot-Marie-Tooth DiseaseSep 22Rigshospitalet, DenmarkNCT06040567
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisSep 21Haukeland University HospitalNCT05095571
No longer enrolling new participants

No longer enrolling new participants

FDA SafetySep 21
FDA Updates Regulations to Advance Innovative Alternatives to Animal Testing

The FDA today issued a direct final rule that updates its regulations to clarify that non-animal methods can be used where appropriate for testing the safety of drugs and biological products intended for human use before they’re tried in humans.

company_8kFabry DiseaseSep 21
PTC Therapeutics disclosed a material event involving Fabry Disease (company event)

PTC Therapeutics (PTCT) filed an 8-K with the SEC on 2026-09-21 covering: company event. The filing mentions Fabry Disease. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 21
Retrophin (now Travere Therapeutics) disclosed a material event (leadership change)

Retrophin (now Travere Therapeutics) (TVTX) filed an 8-K with the SEC on 2026-09-21 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeBecker Muscular DystrophySep 21Insmed Gene Therapy LLCNCT06817382
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBecker Muscular DystrophySep 21NS Pharma, Inc.NCT05996003
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisSep 21Campus Bio-Medico UniversityNCT06834269
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlport SyndromeSep 21BayerNCT07211685
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaSep 19Bristol-Myers SquibbNCT05664737
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophySep 19University of OxfordNCT07578454
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionSep 18Istanbul University - CerrahpasaNCT07667673
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaSep 18Octant, Inc.NCT07408232
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

FDA ApprovalSanfilippo SyndromeSep 17
FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.

Status ChangeMyelodysplastic SyndromesSep 17National Cancer Institute (NCI)NCT03816319
No longer enrolling new participants

No longer enrolling new participants

company_8kSanfilippo SyndromeSep 17
Ultragenyx Pharmaceutical disclosed a material event involving Mucopolysaccharidosis Type III (company event)

Ultragenyx Pharmaceutical (RARE) filed an 8-K with the SEC on 2026-09-17 covering: company event. The filing mentions Mucopolysaccharidosis Type III. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeMyelodysplastic SyndromesSep 17Institute of Hematology and Blood Transfusion, Czech RepublicNCT06765876
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 17Hoffmann-La RocheNCT05704361
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 17Texas Woman's UniversityNCT07555015
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMedullary Thyroid CancerSep 17National Cancer Institute (NCI)NCT01660984
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16CelgeneNCT04884035
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16Jacobs University Bremen gGmbHNCT06899308
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16Fondazione Italiana Linfomi - ETSNCT05300282
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16M.D. Anderson Cancer CenterNCT06108232
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHodgkin LymphomaSep 16Wake Forest University Health SciencesNCT05595577
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16McMaster UniversityNCT06225232
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16Treeline Biosciences, Inc.NCT06733441
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16Fred Hutchinson Cancer CenterNCT03779854
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 16Fondazione Italiana Linfomi - ETSNCT06492837
No longer enrolling new participants

No longer enrolling new participants

FDA NewsSep 15
FDA Launches Expedited IND Pilot, Begins Accepting Applications

The U.S. Food and Drug Administration today announced the final design of the Expedited Investigational New Drug (IND) Pilot.

company_8kNoonan SyndromeSep 15
BioMarin Pharmaceutical disclosed a material event involving Noonan Syndrome (company event)

BioMarin Pharmaceutical (BMRN) filed an 8-K with the SEC on 2026-09-15 covering: company event. The filing mentions Noonan Syndrome. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangePheochromocytomaSep 14National Cancer Institute (NCI)NCT01660984
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParagangliomaSep 14National Cancer Institute (NCI)NCT01660984
No longer enrolling new participants

No longer enrolling new participants

company_8kSep 14
Cabaletta Bio disclosed a material event (company event)

Cabaletta Bio (CABA) filed an 8-K with the SEC on 2026-09-14 covering: company event. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 14
BioMarin Pharmaceutical disclosed a material event (leadership change)

BioMarin Pharmaceutical (BMRN) filed an 8-K with the SEC on 2026-09-14 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeShort Bowel SyndromeSep 14TakedaNCT06973304
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionSep 14University of GiessenNCT06409624
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePolycythemia VeraSep 14Novartis PharmaceuticalsNCT07340138
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisSep 13Novartis PharmaceuticalsNCT07340138
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesSep 13Seagen, a wholly owned subsidiary of PfizerNCT04227847
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 13Novartis PharmaceuticalsNCT06655896
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 13Novartis PharmaceuticalsNCT06675864
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 13University of CalgaryNCT06261489
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMultiple SclerosisSep 13Fondazione Policlinico Universitario Agostino Gemelli IRCCSNCT06551649
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple Endocrine Neoplasia Type 1Sep 13J.M. de LaatNCT05037461
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSanfilippo SyndromeSep 13GC Biopharma CorpNCT06567769
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHunter SyndromeSep 12Denali Therapeutics Inc.NCT05371613
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 12Guangzhou Lupeng Pharmaceutical Company LTD.NCT06251180
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 12Ohio State University Comprehensive Cancer CenterNCT07166419
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 12GenmabNCT06191744
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia ASep 11Hangzhou Gensciences Biopharmaceutical Co., Ltd.NCT07663903
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia ASep 11Novo Nordisk A/SNCT05685238
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGraves’ DiseaseSep 11Zai Lab (Shanghai) Co., Ltd.NCT07211776
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaSep 11Novartis PharmaceuticalsNCT07340138
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Wilderman Medical ClinicNCT06916403
Status changed from Active Not Recruiting to Enrolling By Invitation

Status changed from Active Not Recruiting to Enrolling By Invitation

Status ChangeChronic Graft-versus-Host DiseaseSep 10Gruppo Italiano Malattie EMatologiche dell'AdultoNCT04282811
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Medical University of South CarolinaNCT05095532
No longer enrolling new participants

No longer enrolling new participants

company_8kFocal EpilepsySep 10
Biohaven disclosed a material event involving Focal Epilepsy (company event)

Biohaven (BHVN) filed an 8-K with the SEC on 2026-09-10 covering: company event. The filing mentions Focal Epilepsy. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeCystic FibrosisSep 10AstraZenecaNCT07245407
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Vejle HospitalNCT06473701
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10University of PittsburghNCT06605807
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Father Flanagan's Boys' HomeNCT06852664
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10EnsomaNCT06876363
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Cardiovascular and Interventional Radiological Society of EuropeNCT06149650
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Boston UniversityNCT05503173
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10SanofiNCT06087627
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Washington University School of MedicineNCT07020273
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10Chang Gung University of Science and TechnologyNCT07310849
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeChronic Graft-versus-Host DiseaseSep 10Soren Schou OlesenNCT06426160
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10University of South CarolinaNCT06096701
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 10BiocadNCT07119372
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaSep 10Washington University School of MedicineNCT06910670
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeSep 9Ultragenyx Pharmaceutical IncNCT07157254
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeSep 9Ultragenyx Pharmaceutical IncNCT06415344
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisSep 9Fondazione Policlinico Universitario Agostino Gemelli IRCCSNCT06551649
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophySep 8CelgeneNCT06764485
No longer enrolling new participants

No longer enrolling new participants

company_8kSep 8
Mirum Pharmaceuticals disclosed a material event (leadership change)

Mirum Pharmaceuticals (MIRM) filed an 8-K with the SEC on 2026-09-08 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 8
Keros Therapeutics disclosed a material event (company event)

Keros Therapeutics (KROS) filed an 8-K with the SEC on 2026-09-08 covering: company event. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kHereditary AngioedemaSep 8
Intellia Therapeutics disclosed a material event involving Hereditary Angioedema (company event)

Intellia Therapeutics (NTLA) filed an 8-K with the SEC on 2026-09-08 covering: company event. The filing mentions Hereditary Angioedema. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeSystemic MastocytosisSep 8Fred Hutchinson Cancer CenterNCT03779854
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinocerebellar AtaxiaSep 8Kissei Pharmaceutical Co., Ltd.NCT07040137
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParoxysmal Nocturnal HemoglobinuriaSep 7Swedish Orphan BiovitrumNCT05776472
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeurofibromatosis Type 1Sep 6David MillerNCT06515860
Status changed from Active Not Recruiting to Enrolling By Invitation

Status changed from Active Not Recruiting to Enrolling By Invitation

Status ChangeNarcolepsy Type 1Sep 6TakedaNCT07363720
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNarcolepsy Type 1Sep 6TakedaNCT07299097
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesSep 6Auron Therapeutics, Inc.NCT06846606
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyelodysplastic SyndromesSep 6Swedish Orphan BiovitrumNCT05776472
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesSep 6Fred Hutchinson Cancer CenterNCT03779854
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisSep 6Novartis PharmaceuticalsNCT06704269
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisSep 6Cartesian TherapeuticsNCT06799247
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 6Fondazione I.R.C.C.S. Istituto Neurologico Carlo BestaNCT06699095
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 6Accelerated Cure Project for Multiple SclerosisNCT06441617
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMultiple SclerosisSep 6Novartis PharmaceuticalsNCT06617793
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 6Fondation Ophtalmologique Adolphe de RothschildNCT05999604
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisSep 6National Institute of Allergy and Infectious Diseases (NIAID)NCT05285891
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMixed Connective Tissue DiseaseSep 6Fred Hutchinson Cancer CenterNCT03779854
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMastocytosisSep 6Fred Hutchinson Cancer CenterNCT03779854
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseSep 5Hoffmann-La RocheNCT07246941
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 5Shanghai Junshi Bioscience Co., Ltd.NCT07081022
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 5University of WashingtonNCT06817122
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 5National Cancer Institute (NCI)NCT04739813
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaSep 5Baylor College of MedicineNCT04288726
No longer enrolling new participants

No longer enrolling new participants

FDA NewsSep 4
FDA Takes Steps to Maintain Newborn Access to Life-Saving Starter Nutrition Products

The U.S. Food and Drug Administration today announced it is taking action, consistent with the Trump administration’s commitment to protecting vulnerable patients and maintaining access to critical medical products, to help address a potential supply gap of neonatal starter parenteral nutrition (PN)

FDA ApprovalSep 4
FDA Grants Accelerated Approval to a New Breast Cancer Treatment

The U.S. Food and Drug Administration today expanded treatment options for adult patients with advanced breast cancer, reflecting the FDA’s commitment to advancing medical innovation and getting new treatments to patients who need them.

Status ChangeHemophilia ASep 4Hangzhou Gensciences Biopharmaceutical Co., Ltd.NCT07684898
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGranulomatosis with PolyangiitisSep 4Novartis PharmaceuticalsNCT06868290
No longer enrolling new participants

No longer enrolling new participants

company_8kHereditary AngioedemaSep 4
Intellia Therapeutics disclosed a material event involving Hereditary Angioedema (material agreement)

Intellia Therapeutics (NTLA) filed an 8-K with the SEC on 2026-09-04 covering: material agreement. The filing mentions Hereditary Angioedema. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kAlexander DiseaseSep 4
Ionis Pharmaceuticals disclosed a material event involving Alexander Disease (company event)

Ionis Pharmaceuticals (IONS) filed an 8-K with the SEC on 2026-09-04 covering: company event. The filing mentions Alexander Disease. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeFibrodysplasia Ossificans ProgressivaSep 4Amsterdam UMC, location VUmcNCT04307953
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalAlexander DiseaseSep 3
FDA Approves First Drug to Treat Alexander Disease

The U.S. Food and Drug Administration has approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in pediatric and adult patients.

Status ChangeChronic Graft-versus-Host DiseaseSep 3Rush University Medical CenterNCT06044649
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3European Society of CardiologyNCT06495177
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Pardis Specialized Wellness InstituteNCT07591714
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeChronic Graft-versus-Host DiseaseSep 3University of Illinois at ChicagoNCT05275192
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Il-Yang Pharm. Co., Ltd.NCT06665412
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3HealthPartners InstituteNCT07491861
No longer enrolling new participants

No longer enrolling new participants

FDA NewsSep 3
FDA Issues Emergency Use Authorization for Drugs to Prevent and Treat New World Screwworm in Dogs and Cats

The U.S. Food and Drug Administration today issued an Emergency Use Authorization (EUA) for Capstar (nitenpyram) tablets and revised the EUA for Nitenpyram Tablets, the generic version of Capstar

FDA NewsSep 3
FDA Seeks Public Input to Advance Development of Botanical Drug Products

The U.S. Food and Drug Administration today announced a request for information (RFI) to gather public input on opportunities to advance the development of botanical drug products.

Status ChangeEosinophilic EsophagitisSep 3Dr. Falk Pharma GmbHNCT06596252
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDilated CardiomyopathySep 3Tenaya TherapeuticsNCT05112237
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3University of MichiganNCT06771713
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Janssen Research & Development, LLCNCT05963074
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Stanford UniversityNCT06867757
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Connect Biopharm LLCNCT06940154
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3University Hospital, BrestNCT05299294
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Arizona State UniversityNCT06720909
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3AstraZenecaNCT06712563
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseSep 3Mt. Sinai Medical Center, MiamiNCT03982693
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisSep 2Dallas VA Medical CenterNCT04244630
No longer enrolling new participants

No longer enrolling new participants

company_8kAngelman SyndromeSep 2
Ultragenyx Pharmaceutical disclosed a material event involving Angelman Syndrome (company event)

Ultragenyx Pharmaceutical (RARE) filed an 8-K with the SEC on 2026-09-02 covering: company event. The filing mentions Angelman Syndrome. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 2
Cogent Biosciences disclosed a material event (material agreement)

Cogent Biosciences (COGT) filed an 8-K with the SEC on 2026-09-02 covering: material agreement. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Status ChangeAmyotrophic Lateral SclerosisSep 2Fondazione I.R.C.C.S. Istituto Neurologico Carlo BestaNCT06856850
No longer enrolling new participants

No longer enrolling new participants

company_8kSep 1
BioMarin Pharmaceutical disclosed a material event (company event)

BioMarin Pharmaceutical (BMRN) filed an 8-K with the SEC on 2026-09-01 covering: company event. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 1
Vertex Pharmaceuticals disclosed a material event (leadership change)

Vertex Pharmaceuticals (VRTX) filed an 8-K with the SEC on 2026-09-01 covering: leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

company_8kSep 1
Crinetics Pharmaceuticals disclosed a material event (agreement terminated, acquisition or disposition completed, stock listing notice, leadership change)

Crinetics Pharmaceuticals (CRNX) filed an 8-K with the SEC on 2026-09-01 covering: agreement terminated, acquisition or disposition completed, stock listing notice, leadership change. Companies must file 8-Ks for material events within 4 business days, and clinical, regulatory, and business news sometimes appears here before any press release.

Trial Site & Enrollment Activity

sites_addedCongenital Adrenal HyperplasiaSep 30Crinetics Pharmaceuticals Inc.NCT07159841
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedCongenital Adrenal HyperplasiaSep 30Crinetics Pharmaceuticals Inc.NCT07144163
Added 3 new sites: Campinas, Brazil, Halifax, Canada, Setagaya-ku, Japan

Added 3 new sites: Campinas, Brazil, Halifax, Canada, Setagaya-ku, Japan

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 30SanofiNCT06859099
Added 3 new sites: Montpellier, France, Nice, France, Kodaira, Japan

Added 3 new sites: Montpellier, France, Nice, France, Kodaira, Japan

eligibility_changeChronic Inflammatory Demyelinating PolyneuropathySep 30Immunovant Sciences GmbHNCT07032662
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30Novartis PharmaceuticalsNCT07354074
Added 26 new sites: New Brunswick, New Jersey, New York, New York, Cincinnati, Ohio and 23 more

Added 26 new sites: New Brunswick, New Jersey, New York, New York, Cincinnati, Ohio and 23 more

sites_removedChronic Graft-versus-Host DiseaseSep 30Marche Region Regional Health AgencyNCT07149610
Reduced from 13 to 11 sites

Reduced from 13 to 11 sites

sites_addedChronic Graft-versus-Host DiseaseSep 30SanofiNCT06082037
Added 9 new sites: Salt Lake City, Utah, Strasbourg, France, Milan, Italy and 6 more

Added 9 new sites: Salt Lake City, Utah, Strasbourg, France, Milan, Italy and 6 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 30National University of Natural MedicineNCT07125027
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30University of GiessenNCT06409624
Added 1 new site: Bad Nauheim, Germany

Added 1 new site: Bad Nauheim, Germany

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 30University of GiessenNCT06409624
Target enrollment increased from 350 to 400

Target enrollment increased from 350 to 400

eligibility_changeChronic Graft-versus-Host DiseaseSep 30University of GiessenNCT06409624
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30argenxNCT07091630
Added 66 new sites: Birmingham, Alabama, Rancho Mirage, California, Naples, Florida and 63 more

Added 66 new sites: Birmingham, Alabama, Rancho Mirage, California, Naples, Florida and 63 more

sites_removedChronic Graft-versus-Host DiseaseSep 30Imugene LimitedNCT03666000
Reduced from 23 to 15 sites

Reduced from 23 to 15 sites

eligibility_changeChronic Graft-versus-Host DiseaseSep 30Imugene LimitedNCT03666000
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30Vector Vitale LLCNCT06506643
Added 1 new site: Kropyvnytskyi, Ukraine

Added 1 new site: Kropyvnytskyi, Ukraine

sites_addedChronic Graft-versus-Host DiseaseSep 30TakedaNCT06948318
Added 5 new sites: San Diego, California, New York, New York, The Bronx, New York and 2 more

Added 5 new sites: San Diego, California, New York, New York, The Bronx, New York and 2 more

sites_addedChronic Graft-versus-Host DiseaseSep 30UCB Biopharma SRLNCT07725939
Added 2 new sites: Hangzhou, China, Shijiazhuang, China

Added 2 new sites: Hangzhou, China, Shijiazhuang, China

sites_addedChronic Graft-versus-Host DiseaseSep 30Kedrion S.p.A.NCT06752356
Added 2 new sites: Stamford, Connecticut, Miami, Florida

Added 2 new sites: Stamford, Connecticut, Miami, Florida

sites_addedChronic Graft-versus-Host DiseaseSep 30Ausper Biopharma Co., Ltd.NCT07069569
Added 13 new sites: Chongqing, China, Fuzhou, China, Guangzhou, China and 10 more

Added 13 new sites: Chongqing, China, Fuzhou, China, Guangzhou, China and 10 more

sites_addedChronic Graft-versus-Host DiseaseSep 30Boston Scientific CorporationNCT06827262
Added 3 new sites: Stanford, California, Saint Cloud, Minnesota, The Bronx, New York

Added 3 new sites: Stanford, California, Saint Cloud, Minnesota, The Bronx, New York

sites_addedDuchenne Muscular DystrophySep 30Avidity Biosciences, Inc.NCT07587242
Added 2 new sites: Milan, Italy, Leiden, Netherlands

Added 2 new sites: Milan, Italy, Leiden, Netherlands

Enrollment UpdateDilated CardiomyopathySep 30Joshua M HareNCT04476901
Target enrollment decreased from 136 to 107

Target enrollment decreased from 136 to 107

sites_addedDiffuse Intrinsic Pontine GliomaSep 30National Cancer Institute (NCI)NCT05099003
Added 1 new site: Temple, Texas

Added 1 new site: Temple, Texas

sites_addedDesmoplastic Small Round Cell TumorSep 30National Cancer Institute (NCI)NCT04616560
Added 2 new sites: Valhalla, New York, Portland, Oregon

Added 2 new sites: Valhalla, New York, Portland, Oregon

sites_addedDermatomyositisSep 30AstraZenecaNCT06455449
Added 1 new site: Bangalore, India

Added 1 new site: Bangalore, India

Enrollment UpdateCystic FibrosisSep 30Vertex Pharmaceuticals IncorporatedNCT07437105
Target enrollment increased from 128 to 160

Target enrollment increased from 128 to 160

Enrollment UpdateCystic FibrosisSep 30Assiut UniversityNCT07147803
Target enrollment increased from 60 to 200

Target enrollment increased from 60 to 200

eligibility_changeCystic FibrosisSep 30Assiut UniversityNCT07147803
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 30argenxNCT07091630
Added 3 new sites: Rio de Janeiro, Brazil, Pôrto Alegre, Brazil, Ribeirão Preto, Brazil

Added 3 new sites: Rio de Janeiro, Brazil, Pôrto Alegre, Brazil, Ribeirão Preto, Brazil

eligibility_changeChronic Graft-versus-Host DiseaseSep 30VA Office of Research and DevelopmentNCT07149870
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30Merck Sharp & Dohme LLCNCT05947851
Added 27 new sites: Clermont, Florida, Fort Wayne, Indiana, Bethesda, Maryland and 24 more

Added 27 new sites: Clermont, Florida, Fort Wayne, Indiana, Bethesda, Maryland and 24 more

sites_addedChronic Graft-versus-Host DiseaseSep 30BeOne MedicinesNCT06846671
Added 14 new sites: San Diego, California, Golden, Colorado, Grand Rapids, Michigan and 11 more

Added 14 new sites: San Diego, California, Golden, Colorado, Grand Rapids, Michigan and 11 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 30National Institute of Allergy and Infectious Diseases (NIAID)NCT07113743
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 30Nanordica Medical OUNCT06667752
Target enrollment decreased from 170 to 136

Target enrollment decreased from 170 to 136

sites_addedChronic Graft-versus-Host DiseaseSep 30IntegerBioNCT07389044
Added 4 new sites: Hong Kong, Hong Kong, Hong Kong, Hong Kong, Auckland, New Zealand and 1 more

Added 4 new sites: Hong Kong, Hong Kong, Hong Kong, Hong Kong, Auckland, New Zealand and 1 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 30IntegerBioNCT07389044
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 30Magnetic TidesNCT07631078
Target enrollment decreased from 12 to 6

Target enrollment decreased from 12 to 6

eligibility_changeChronic Graft-versus-Host DiseaseSep 30Magnetic TidesNCT07631078
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30TakedaNCT06722235
Added 12 new sites: Hot Springs, Arkansas, San Diego, California, Indianapolis, Indiana and 9 more

Added 12 new sites: Hot Springs, Arkansas, San Diego, California, Indianapolis, Indiana and 9 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 30TakedaNCT06722235
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30Zenas BioPharma (USA), LLCNCT07067463
Added 56 new sites: Pheonix, Arizona, West Hollywood, California, Tampa, Florida and 53 more

Added 56 new sites: Pheonix, Arizona, West Hollywood, California, Tampa, Florida and 53 more

sites_addedChronic Graft-versus-Host DiseaseSep 30XyloCor Therapeutics, Inc.NCT07048808
Added 13 new sites: Birmingham, Alabama, Phoenix, Arizona, Atlanta, Georgia and 10 more

Added 13 new sites: Birmingham, Alabama, Phoenix, Arizona, Atlanta, Georgia and 10 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 30Johns Hopkins UniversityNCT07059091
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30Altesa Biosciences, Inc.NCT07610395
Added 31 new sites: Mobile, Alabama, Inglewood, California, Bradenton, Florida and 28 more

Added 31 new sites: Mobile, Alabama, Inglewood, California, Bradenton, Florida and 28 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 30Altesa Biosciences, Inc.NCT07610395
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 30AstraZenecaNCT07520162
Added 4 new sites: Guangzhou, China, Guangzhou, China, Hangzhou, China and 1 more

Added 4 new sites: Guangzhou, China, Guangzhou, China, Hangzhou, China and 1 more

sites_addedChronic Graft-versus-Host DiseaseSep 30Janssen Research & Development, LLCNCT06788509
Added 10 new sites: Macquarie University, Australia, Melbourne, Australia, Randwick, Australia and 7 more

Added 10 new sites: Macquarie University, Australia, Melbourne, Australia, Randwick, Australia and 7 more

sites_addedChronic Graft-versus-Host DiseaseSep 30AstraZenecaNCT06749457
Added 1 new site: Las Vegas, Nevada

Added 1 new site: Las Vegas, Nevada

eligibility_changeChronic Graft-versus-Host DiseaseSep 30AstraZenecaNCT06749457
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseSep 30National Institute of Allergy and Infectious Diseases (NIAID)NCT06253507
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 30University of UtahNCT05607381
Target enrollment increased from 150 to 188

Target enrollment increased from 150 to 188

sites_addedChronic Graft-versus-Host DiseaseSep 30Ottawa Hospital Research InstituteNCT06392776
Added 3 new sites: Kingston, Canada, Toronto, Canada, Québec, Canada

Added 3 new sites: Kingston, Canada, Toronto, Canada, Québec, Canada

sites_addedChronic Graft-versus-Host DiseaseSep 30Windward BioNCT07646587
Added 17 new sites: Los Angeles, California, Medford, Oregon, DuBois, Pennsylvania and 14 more

Added 17 new sites: Los Angeles, California, Medford, Oregon, DuBois, Pennsylvania and 14 more

sites_addedCholangiocarcinomaSep 30AstraZenecaNCT07221253
Added 6 new sites: Miami, Florida, Mineola, New York, Oklahoma City, Oklahoma and 3 more

Added 6 new sites: Miami, Florida, Mineola, New York, Oklahoma City, Oklahoma and 3 more

Enrollment UpdateCholangiocarcinomaSep 30Massachusetts General HospitalNCT06302621
Target enrollment decreased from 70 to 32

Target enrollment decreased from 70 to 32

sites_addedBecker Muscular DystrophySep 29Avidity Biosciences, Inc.NCT07587242
Added 2 new sites: Milan, Italy, Leiden, Netherlands

Added 2 new sites: Milan, Italy, Leiden, Netherlands

sites_addedBecker Muscular DystrophySep 29Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Nantes, France

Added 1 new site: Nantes, France

sites_addedBecker Muscular DystrophySep 29Satellos Bioscience, Inc.NCT06867107
Added 3 new sites: Los Angeles, California, Columbus, Ohio, Memphis, Tennessee

Added 3 new sites: Los Angeles, California, Columbus, Ohio, Memphis, Tennessee

Enrollment UpdateBecker Muscular DystrophySep 29Satellos Bioscience, Inc.NCT06867107
Target enrollment increased from 10 to 30

Target enrollment increased from 10 to 30

eligibility_changeBecker Muscular DystrophySep 29Satellos Bioscience, Inc.NCT06867107
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Sep 29Novo Nordisk A/SNCT07207811
Added 2 new sites: Allentown, Pennsylvania, Buenos Aires, Argentina

Added 2 new sites: Allentown, Pennsylvania, Buenos Aires, Argentina

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Sep 29BayerNCT07298044
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeAplastic AnemiaSep 29National Heart, Lung, and Blood Institute (NHLBI)NCT05012111
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisSep 29Eli Lilly and CompanyNCT07571200
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

sites_addedAmyotrophic Lateral SclerosisSep 29Eli Lilly and CompanyNCT07571174
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

sites_addedAlpha-1 Antitrypsin DeficiencySep 29Tessera Therapeutics, Inc.NCT07227207
Added 1 new site: Jacksonville, Florida

Added 1 new site: Jacksonville, Florida

eligibility_changeAlpha-1 Antitrypsin DeficiencySep 29Weill Medical College of Cornell UniversityNCT06996756
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAchondroplasiaSep 29BioMarin PharmaceuticalNCT07441876
Added 4 new sites: Dallas, Texas, Québec, Canada, Milan, Italy and 1 more

Added 4 new sites: Dallas, Texas, Québec, Canada, Milan, Italy and 1 more

sites_addedAchondroplasiaSep 29SYSNAVNCT07388966
Added 3 new sites: Liège, Belgium, Liège, Belgium, London, United Kingdom

Added 3 new sites: Liège, Belgium, Liège, Belgium, London, United Kingdom

Enrollment UpdateAchondroplasiaSep 29SYSNAVNCT07388966
Target enrollment increased from 40 to 140

Target enrollment increased from 40 to 140

eligibility_changeAchondroplasiaSep 29SYSNAVNCT07388966
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedX-Linked HypophosphatemiaSep 28Kyowa Kirin Co., Ltd.NCT06525636
Added 1 new site: Oakville, Canada

Added 1 new site: Oakville, Canada

Enrollment UpdateX-Linked HypophosphatemiaSep 28Kyowa Kirin Co., Ltd.NCT06525636
Target enrollment increased from 24 to 39

Target enrollment increased from 24 to 39

eligibility_changeX-Linked HypophosphatemiaSep 28Kyowa Kirin Co., Ltd.NCT06525636
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeWild-Type ATTR Cardiac AmyloidosisSep 28BayerNCT07298044
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedThyroid Eye DiseaseSep 27argenxNCT07596849
Added 12 new sites: Pazardzhik, Bulgaria, Varna, Bulgaria, Varna, Bulgaria and 9 more

Added 12 new sites: Pazardzhik, Bulgaria, Varna, Bulgaria, Varna, Bulgaria and 9 more

sites_addedThyroid Eye DiseaseSep 27argenxNCT07570316
Added 3 new sites: Jacksonville, Florida, Miami, Florida, Albany, New York

Added 3 new sites: Jacksonville, Florida, Miami, Florida, Albany, New York

sites_addedTay-Sachs DiseaseSep 27Azafaros B.V.NCT07082543
Added 1 new site: Paris, France

Added 1 new site: Paris, France

sites_addedTay-Sachs DiseaseSep 27Azafaros B.V.NCT07054515
Added 1 new site: Paris, France

Added 1 new site: Paris, France

sites_addedSystemic SclerosisSep 27argenxNCT06655155
Added 1 new site: Lille, France

Added 1 new site: Lille, France

sites_addedSystemic MastocytosisSep 27Blueprint Medicines CorporationNCT04910685
Added 1 new site: Santiago, Chile

Added 1 new site: Santiago, Chile

sites_addedSystemic Juvenile Idiopathic ArthritisSep 27SanofiNCT02991469
Added 7 new sites: Athens, Greece, Budapest, Hungary, Mérida, Mexico and 4 more

Added 7 new sites: Athens, Greece, Budapest, Hungary, Mérida, Mexico and 4 more

eligibility_changeSystemic Juvenile Idiopathic ArthritisSep 27National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NCT03510442
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseSep 27Disc Medicine, IncNCT07187973
Added 1 new site: Pittsburgh, Pennsylvania

Added 1 new site: Pittsburgh, Pennsylvania

sites_addedRetinitis PigmentosaSep 27Octant, Inc.NCT07408232
Added 1 new site: Portland, Oregon

Added 1 new site: Portland, Oregon

sites_addedPulmonary Arterial HypertensionSep 26Foresee Pharmaceuticals Co., Ltd.NCT06475781
Added 2 new sites: Banja Luka, Bosnia and Herzegovina, Sarajevo, Bosnia and Herzegovina

Added 2 new sites: Banja Luka, Bosnia and Herzegovina, Sarajevo, Bosnia and Herzegovina

Enrollment UpdatePulmonary Arterial HypertensionSep 26Chinese Pulmonary Vascular Disease Research GroupNCT04206852
Target enrollment increased from 200 to 1000

Target enrollment increased from 200 to 1000

sites_addedPulmonary Arterial HypertensionSep 26Regeneron PharmaceuticalsNCT07318597
Added 2 new sites: Brisbane, Australia, London, United Kingdom

Added 2 new sites: Brisbane, Australia, London, United Kingdom

sites_addedPulmonary Arterial HypertensionSep 26Merck Sharp & Dohme LLCNCT07218029
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

sites_addedPolycythemia VeraSep 26Novartis PharmaceuticalsNCT07357727
Added 20 new sites: Fort Myers, Florida, Miami, Florida, St. Petersburg, Florida and 17 more

Added 20 new sites: Fort Myers, Florida, Miami, Florida, St. Petersburg, Florida and 17 more

sites_addedPhelan-McDermid SyndromeSep 26Neuren Pharmaceuticals LimitedNCT07593391
Added 3 new sites: Birmingham, Alabama, Brookline, Massachusetts, New York, New York

Added 3 new sites: Birmingham, Alabama, Brookline, Massachusetts, New York, New York

sites_addedPhelan-McDermid SyndromeSep 26Neuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Washington D.C., District of Columbia, Atlanta, Georgia

Added 2 new sites: Washington D.C., District of Columbia, Atlanta, Georgia

sites_addedOsteogenesis ImperfectaSep 26Angitia Biopharmaceuticals Guangzhou LimitedNCT07557446
Added 1 new site: Changsha, China

Added 1 new site: Changsha, China

sites_removedNoonan SyndromeSep 26BioMarin PharmaceuticalNCT06668805
Reduced from 41 to 38 sites

Reduced from 41 to 38 sites

sites_addedNiemann-Pick Disease Type CSep 26Azafaros B.V.NCT07082543
Added 1 new site: Paris, France

Added 1 new site: Paris, France

sites_addedNiemann-Pick Disease Type CSep 26Azafaros B.V.NCT07054515
Added 1 new site: Paris, France

Added 1 new site: Paris, France

sites_addedNiemann-Pick DiseaseSep 26Azafaros B.V.NCT07054515
Added 1 new site: Paris, France

Added 1 new site: Paris, France

eligibility_changeNeuromyelitis OpticaSep 26Tongji HospitalNCT06620809
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeNeurofibromatosis Type 1Sep 26National Cancer Institute (NCI)NCT00924196
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyotonic DystrophySep 25Dyne TherapeuticsNCT07486934
Added 2 new sites: Rochester, New York, Göttingen, Germany

Added 2 new sites: Rochester, New York, Göttingen, Germany

sites_addedMyelofibrosisSep 25Novartis PharmaceuticalsNCT07357727
Added 6 new sites: Fort Myers, Florida, Dallas, Texas, Ostrava, Czechia and 3 more

Added 6 new sites: Fort Myers, Florida, Dallas, Texas, Ostrava, Czechia and 3 more

sites_addedMyelodysplastic SyndromesSep 25Janssen Research & Development, LLCNCT06788509
Added 2 new sites: Melbourne, Australia, Athens, Greece

Added 2 new sites: Melbourne, Australia, Athens, Greece

sites_addedMyasthenia GravisSep 25Janssen Research & Development, LLCNCT07217587
Added 2 new sites: Foxborough, Massachusetts, Austin, Texas

Added 2 new sites: Foxborough, Massachusetts, Austin, Texas

sites_addedMyasthenia GravisSep 25UCB Biopharma SRLNCT07463521
Added 3 new sites: Alicante, Spain, Barcelona, Spain, Madrid, Spain

Added 3 new sites: Alicante, Spain, Barcelona, Spain, Madrid, Spain

sites_addedMyasthenia GravisSep 25Dianthus TherapeuticsNCT07647510
Added 1 new site: Pasadena, California

Added 1 new site: Pasadena, California

sites_addedMesotheliomaSep 25IDEAYA BiosciencesNCT07277413
Added 1 new site: Goodyear, Arizona

Added 1 new site: Goodyear, Arizona

sites_addedMerkel Cell CarcinomaSep 25Weill Medical College of Cornell UniversityNCT05583708
Added 1 new site: Duarte, California

Added 1 new site: Duarte, California

sites_addedNarcolepsy Type 1Sep 25TakedaNCT07646678
Added 11 new sites: Chula Vista, California, Colorado Springs, Colorado, Winter Park, Florida and 8 more

Added 11 new sites: Chula Vista, California, Colorado Springs, Colorado, Winter Park, Florida and 8 more

sites_addedMyotonic DystrophySep 25SanofiNCT06844214
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

sites_addedMyotonic DystrophySep 25Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Nantes, France

Added 1 new site: Nantes, France

eligibility_changeMyelofibrosisSep 25Prelude Therapeutics IncorporatedNCT07469891
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisSep 25Sumitomo Pharma America, Inc.NCT04176198
Added 1 new site: Tokyo, Japan

Added 1 new site: Tokyo, Japan

sites_addedMyelofibrosisSep 25Novartis PharmaceuticalsNCT07357727
Added 15 new sites: Miami, Florida, St. Petersburg, Florida, Sankt Pölten, Austria and 12 more

Added 15 new sites: Miami, Florida, St. Petersburg, Florida, Sankt Pölten, Austria and 12 more

sites_addedMyelodysplastic SyndromesSep 25TakedaNCT07422480
Added 13 new sites: New Haven, Connecticut, Monroe, Louisiana, Shreveport, Louisiana and 10 more

Added 13 new sites: New Haven, Connecticut, Monroe, Louisiana, Shreveport, Louisiana and 10 more

sites_addedMyelodysplastic SyndromesSep 25National Cancer Institute (NCI)NCT05564390
Added 1 new site: Harvey, Illinois

Added 1 new site: Harvey, Illinois

sites_addedMyasthenia GravisSep 25TG Therapeutics, Inc.NCT07673744
Added 1 new site: Greenville, South Carolina

Added 1 new site: Greenville, South Carolina

sites_addedMyasthenia GravisSep 25argenxNCT07294170
Added 6 new sites: Las Vegas, Nevada, Chapel Hill, North Carolina, Cincinnati, Ohio and 3 more

Added 6 new sites: Las Vegas, Nevada, Chapel Hill, North Carolina, Cincinnati, Ohio and 3 more

sites_addedMyasthenia GravisSep 25argenxNCT07673627
Added 1 new site: Austin, Texas

Added 1 new site: Austin, Texas

sites_addedMyasthenia GravisSep 25argenxNCT07284420
Added 6 new sites: Las Vegas, Nevada, Chapel Hill, North Carolina, Cincinnati, Ohio and 3 more

Added 6 new sites: Las Vegas, Nevada, Chapel Hill, North Carolina, Cincinnati, Ohio and 3 more

sites_addedMyasthenia GravisSep 25argenxNCT06392386
Added 1 new site: Melbourne, Australia

Added 1 new site: Melbourne, Australia

eligibility_changeMultiple SclerosisSep 25Hoffmann-La RocheNCT04544449
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisSep 25Zenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Kansas City, Kansas, Katowice, Poland

Added 2 new sites: Kansas City, Kansas, Katowice, Poland

sites_addedMultiple SclerosisSep 25TG Therapeutics, Inc.NCT06433752
Added 2 new sites: Lincoln, Nebraska, London, United Kingdom

Added 2 new sites: Lincoln, Nebraska, London, United Kingdom

sites_addedMultiple SclerosisSep 25TG Therapeutics, Inc.NCT07220252
Added 1 new site: Warsaw, Poland

Added 1 new site: Warsaw, Poland

sites_addedMultiple SclerosisSep 25Tr1X, Inc.NCT07477639
Added 6 new sites: La Jolla, California, Rochester, Minnesota, New York, New York and 3 more

Added 6 new sites: La Jolla, California, Rochester, Minnesota, New York, New York and 3 more

sites_addedMultiple SclerosisSep 25Zenas BioPharma (USA), LLCNCT07299019
Added 2 new sites: Tampa, Florida, Poznan, Poland

Added 2 new sites: Tampa, Florida, Poznan, Poland

sites_removedMultiple SclerosisSep 25Hoffmann-La RocheNCT04586010
Reduced from 160 to 158 sites

Reduced from 160 to 158 sites

eligibility_changeMultiple SclerosisSep 25Hoffmann-La RocheNCT04586010
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMixed Connective Tissue DiseaseSep 25Paradigm HealthNCT06605404
Added 45 new sites: Birmingham, Alabama, Bullhead City, Arizona, Casa Grande, Arizona and 42 more

Added 45 new sites: Birmingham, Alabama, Bullhead City, Arizona, Casa Grande, Arizona and 42 more

sites_addedMixed Connective Tissue DiseaseSep 25National Cancer Institute (NCI)NCT04616560
Added 1 new site: Portland, Oregon

Added 1 new site: Portland, Oregon

eligibility_changeMesotheliomaSep 25Memorial Sloan Kettering Cancer CenterNCT06057935
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaSep 25IDEAYA BiosciencesNCT07277413
Added 3 new sites: Phoenix, Arizona, Jacksonville, Florida, Rochester, Minnesota

Added 3 new sites: Phoenix, Arizona, Jacksonville, Florida, Rochester, Minnesota

sites_addedMerkel Cell CarcinomaSep 25University of ArizonaNCT06814496
Added 1 new site: Gilbert, Arizona

Added 1 new site: Gilbert, Arizona

sites_addedMerkel Cell CarcinomaSep 25Novartis PharmaceuticalsNCT07006727
Added 1 new site: Chuo Ku, Japan

Added 1 new site: Chuo Ku, Japan

sites_addedMembranous NephropathySep 25Vertex Pharmaceuticals IncorporatedNCT07204275
Added 1 new site: Indianapolis, Indiana

Added 1 new site: Indianapolis, Indiana

sites_addedMedullary Thyroid CancerSep 25University of ArizonaNCT06814496
Added 1 new site: Gilbert, Arizona

Added 1 new site: Gilbert, Arizona

sites_addedMedullary Thyroid CancerSep 25Novartis PharmaceuticalsNCT07006727
Added 1 new site: Chuo Ku, Japan

Added 1 new site: Chuo Ku, Japan

sites_addedMastocytosisSep 25Blueprint Medicines CorporationNCT04910685
Added 1 new site: Santiago, Chile

Added 1 new site: Santiago, Chile

sites_addedIdiopathic Pulmonary FibrosisSep 24Contineum TherapeuticsNCT07284459
Added 3 new sites: Nice, France, Kaohsiung City, Taiwan, Taipei, Taiwan

Added 3 new sites: Nice, France, Kaohsiung City, Taiwan, Taipei, Taiwan

sites_addedHodgkin LymphomaSep 24Merck Sharp & Dohme LLCNCT07570173
Added 2 new sites: Odense C, Denmark, Bergamo, Italy

Added 2 new sites: Odense C, Denmark, Bergamo, Italy

sites_addedHodgkin LymphomaSep 24Memorial Sloan Kettering Cancer CenterNCT07223021
Added 1 new site: Utrecht, Netherlands

Added 1 new site: Utrecht, Netherlands

Enrollment UpdateHodgkin LymphomaSep 24Memorial Sloan Kettering Cancer CenterNCT07223021
Target enrollment increased from 65 to 130

Target enrollment increased from 65 to 130

sites_addedHodgkin LymphomaSep 24Autolus LimitedNCT03628612
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedHodgkin LymphomaSep 24Merck Sharp & Dohme LLCNCT07519772
Added 1 new site: CABA, Argentina

Added 1 new site: CABA, Argentina

eligibility_changeHodgkin LymphomaSep 24University of PennsylvaniaNCT07343934
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 24Merck Sharp & Dohme LLCNCT06890884
Added 4 new sites: Bruges, Belgium, Ulm, Germany, Dublin, Ireland and 1 more

Added 4 new sites: Bruges, Belgium, Ulm, Germany, Dublin, Ireland and 1 more

sites_addedHodgkin LymphomaSep 24National Cancer Institute (NCI)NCT05675410
Added 1 new site: Williamsville, New York

Added 1 new site: Williamsville, New York

sites_removedHodgkin LymphomaSep 24Acerta Pharma BVNCT02180711
Reduced from 32 to 29 sites

Reduced from 32 to 29 sites

sites_addedHodgkin LymphomaSep 24Alliance for Clinical Trials in OncologyNCT07215910
Added 24 new sites: Bakersfield, California, Coral Gables, Florida, Coral Springs, Florida and 21 more

Added 24 new sites: Bakersfield, California, Coral Gables, Florida, Coral Springs, Florida and 21 more

eligibility_changeImmune ThrombocytopeniaSep 24TakedaNCT06722235
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedImmune ThrombocytopeniaSep 24argenxNCT07194850
Added 1 new site: Osaka, Japan

Added 1 new site: Osaka, Japan

sites_removedImmune ThrombocytopeniaSep 24Eli Lilly and CompanyNCT06721013
Reduced from 45 to 43 sites

Reduced from 45 to 43 sites

sites_addedIgG4-Related DiseaseSep 24SanofiNCT07190196
Added 19 new sites: Sacramento, California, Detroit, Michigan, New York, New York and 16 more

Added 19 new sites: Sacramento, California, Detroit, Michigan, New York, New York and 16 more

sites_addedIgA NephropathySep 24Sitala Bio LTDNCT07606352
Added 5 new sites: Newcastle, Australia, Penrith, Australia, Sydney, Australia and 2 more

Added 5 new sites: Newcastle, Australia, Penrith, Australia, Sydney, Australia and 2 more

sites_addedIgA NephropathySep 24Novartis PharmaceuticalsNCT07498335
Added 3 new sites: Kanazawa, Japan, Barcelona, Spain, Taipei, Taiwan

Added 3 new sites: Kanazawa, Japan, Barcelona, Spain, Taipei, Taiwan

sites_addedIdiopathic Pulmonary FibrosisSep 24Chiesi Farmaceutici S.p.A.NCT07516951
Added 1 new site: Hannibal, Missouri

Added 1 new site: Hannibal, Missouri

sites_addedIdiopathic Pulmonary FibrosisSep 24AstraZenecaNCT07652658
Added 4 new sites: Camperdown, Australia, Amsterdam, Netherlands, Heerlen, Netherlands and 1 more

Added 4 new sites: Camperdown, Australia, Amsterdam, Netherlands, Heerlen, Netherlands and 1 more

sites_addedIdiopathic Pulmonary FibrosisSep 24Contineum TherapeuticsNCT07284459
Added 3 new sites: Lima, Peru, San Miguel, Peru, Bydgoszcz, Poland

Added 3 new sites: Lima, Peru, San Miguel, Peru, Bydgoszcz, Poland

sites_addedHuntington DiseaseSep 24Novartis PharmaceuticalsNCT07326709
Added 37 new sites: San Francisco, California, Miami, Florida, Chicago, Illinois and 34 more

Added 37 new sites: San Francisco, California, Miami, Florida, Chicago, Illinois and 34 more

sites_addedHuntington DiseaseSep 24Skyhawk Therapeutics, Inc.NCT07378644
Added 2 new sites: Taufkirchen, Germany, Madrid, Spain

Added 2 new sites: Taufkirchen, Germany, Madrid, Spain

sites_addedHodgkin LymphomaSep 24Merck Sharp & Dohme LLCNCT07634471
Added 6 new sites: Duarte, California, Lexington, Kentucky, Hackensack, New Jersey and 3 more

Added 6 new sites: Duarte, California, Lexington, Kentucky, Hackensack, New Jersey and 3 more

sites_addedHodgkin LymphomaSep 24National Cancer Institute (NCI)NCT06649812
Added 17 new sites: Crown Point, Indiana, Dyer, Indiana, Hobart, Indiana and 14 more

Added 17 new sites: Crown Point, Indiana, Dyer, Indiana, Hobart, Indiana and 14 more

Enrollment UpdateHodgkin LymphomaSep 24BioInvent International ABNCT04752826
Target enrollment increased from 176 to 250

Target enrollment increased from 176 to 250

eligibility_changeHodgkin LymphomaSep 24BioInvent International ABNCT04752826
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 24Novartis PharmaceuticalsNCT06784752
Added 3 new sites: Orlando, Florida, Atlanta, Georgia, Glasgow, United Kingdom

Added 3 new sites: Orlando, Florida, Atlanta, Georgia, Glasgow, United Kingdom

sites_addedHodgkin LymphomaSep 24AstraZenecaNCT06137144
Added 2 new sites: São Paulo, Brazil, São Paulo, Brazil

Added 2 new sites: São Paulo, Brazil, São Paulo, Brazil

eligibility_changeHodgkin LymphomaSep 24AstraZenecaNCT06137144
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaSep 24Memorial Sloan Kettering Cancer CenterNCT06756308
Target enrollment decreased from 25 to 2

Target enrollment decreased from 25 to 2

sites_addedHodgkin LymphomaSep 24Arvinas Inc.NCT06393738
Added 4 new sites: Miami, Florida, Charlottesville, Virginia, East Melbourne, Australia and 1 more

Added 4 new sites: Miami, Florida, Charlottesville, Virginia, East Melbourne, Australia and 1 more

Enrollment UpdateHodgkin LymphomaSep 24Arvinas Inc.NCT06393738
Target enrollment increased from 255 to 329

Target enrollment increased from 255 to 329

eligibility_changeHodgkin LymphomaSep 24Arvinas Inc.NCT06393738
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 24Canadian Cancer Trials GroupNCT07341191
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

eligibility_changeHereditary AngioedemaSep 24Pharvaris Netherlands B.V.NCT06679881
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHereditary AngioedemaSep 24Pharvaris Netherlands B.V.NCT07266805
Target enrollment increased from 32 to 48

Target enrollment increased from 32 to 48

eligibility_changeHereditary AngioedemaSep 24Pharvaris Netherlands B.V.NCT07266805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia ASep 24SanofiNCT06145373
Added 2 new sites: Flowood, Mississippi, Taipei, Taiwan

Added 2 new sites: Flowood, Mississippi, Taipei, Taiwan

sites_addedHemophilia ASep 24Hoffmann-La RocheNCT07416526
Added 5 new sites: Beijing, China, Lille, France, Zurich, Switzerland and 2 more

Added 5 new sites: Beijing, China, Lille, France, Zurich, Switzerland and 2 more

sites_addedFocal Segmental GlomerulosclerosisSep 23SanofiNCT06500702
Added 26 new sites: Birmingham, Alabama, Orange, California, San Francisco, California and 23 more

Added 26 new sites: Birmingham, Alabama, Orange, California, San Francisco, California and 23 more

sites_addedEssential ThrombocythemiaSep 23Novartis PharmaceuticalsNCT07357727
Added 12 new sites: Miami, Florida, St. Petersburg, Florida, Florianópolis, Brazil and 9 more

Added 12 new sites: Miami, Florida, St. Petersburg, Florida, Florianópolis, Brazil and 9 more

sites_addedGraves’ DiseaseSep 23Biohaven Therapeutics Ltd.NCT07661056
Added 6 new sites: Hollywood, Florida, Baton Rouge, Louisiana, Kansas City, Missouri and 3 more

Added 6 new sites: Hollywood, Florida, Baton Rouge, Louisiana, Kansas City, Missouri and 3 more

sites_addedGraves’ DiseaseSep 23argenxNCT07570316
Added 4 new sites: Jacksonville, Florida, Miami, Florida, Kansas City, Missouri and 1 more

Added 4 new sites: Jacksonville, Florida, Miami, Florida, Kansas City, Missouri and 1 more

sites_addedGraves’ DiseaseSep 23argenxNCT07596849
Added 17 new sites: Pazardzhik, Bulgaria, Sliven, Bulgaria, Varna, Bulgaria and 14 more

Added 17 new sites: Pazardzhik, Bulgaria, Sliven, Bulgaria, Varna, Bulgaria and 14 more

sites_addedGranulomatosis with PolyangiitisSep 23AstraZenecaNCT07444567
Expanded from 45 to 46 sites

Expanded from 45 to 46 sites

sites_addedGiant Cell ArteritisSep 23SanofiNCT07286214
Added 60 new sites: Avondale, Arizona, Phoenix, Arizona, Tucson, Arizona and 57 more

Added 60 new sites: Avondale, Arizona, Phoenix, Arizona, Tucson, Arizona and 57 more

sites_addedGeographic AtrophySep 23Regeneron PharmaceuticalsNCT06541704
Added 1 new site: Rouen, France

Added 1 new site: Rouen, France

sites_addedGeographic AtrophySep 23SanofiNCT07215234
Added 14 new sites: Beverly Hills, California, Encino, California, Sacramento, California and 11 more

Added 14 new sites: Beverly Hills, California, Encino, California, Sacramento, California and 11 more

sites_removedGeographic AtrophySep 23Astellas Pharma Global Development, Inc.NCT06779773
Reduced from 65 to 58 sites

Reduced from 65 to 58 sites

sites_addedFragile X SyndromeSep 23Mirum Pharmaceuticals, Inc.NCT07209462
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_removedFocal Segmental GlomerulosclerosisSep 23Boehringer IngelheimNCT07220083
Reduced from 309 to 307 sites

Reduced from 309 to 307 sites

eligibility_changeFanconi AnemiaSep 23National Heart, Lung, and Blood Institute (NHLBI)NCT03206086
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeFanconi AnemiaSep 23VA Office of Research and DevelopmentNCT04038502
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFanconi AnemiaSep 23Novartis PharmaceuticalsNCT06910813
Added 4 new sites: Phoenix, Arizona, San Diego, California, Stanford, California and 1 more

Added 4 new sites: Phoenix, Arizona, San Diego, California, Stanford, California and 1 more

Enrollment UpdateFabry DiseaseSep 23General University Hospital, PragueNCT07495410
Target enrollment increased from 15 to 50

Target enrollment increased from 15 to 50

eligibility_changeFabry DiseaseSep 23General University Hospital, PragueNCT07495410
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateEssential ThrombocythemiaSep 23Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Target enrollment increased from 76 to 256

Target enrollment increased from 76 to 256

eligibility_changeEssential ThrombocythemiaSep 23Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaSep 23Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Jinan, China, Padova, Italy, Bunkyo Ku, Japan and 1 more

Added 4 new sites: Jinan, China, Padova, Italy, Bunkyo Ku, Japan and 1 more

eligibility_changeEssential ThrombocythemiaSep 23Prelude Therapeutics IncorporatedNCT07469891
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEosinophilic Granulomatosis with PolyangiitisSep 23AstraZenecaNCT07444567
Expanded from 45 to 46 sites

Expanded from 45 to 46 sites

sites_addedEosinophilic EsophagitisSep 23SanofiNCT06695897
Added 14 new sites: Aschaffenburg, Germany, Augsburg, Germany, Erlangen, Germany and 11 more

Added 14 new sites: Aschaffenburg, Germany, Augsburg, Germany, Erlangen, Germany and 11 more

sites_addedDuchenne Muscular DystrophySep 22Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Nantes, France

Added 1 new site: Nantes, France

Enrollment UpdateCystic FibrosisSep 22Children's Hospital Medical Center, CincinnatiNCT05517655
Target enrollment decreased from 44 to 19

Target enrollment decreased from 44 to 19

sites_addedCystic FibrosisSep 22Anagram Therapeutics, Inc.NCT07450547
Added 1 new site: Minneapolis, Minnesota

Added 1 new site: Minneapolis, Minnesota

sites_removedChronic Inflammatory Demyelinating PolyneuropathySep 22SanofiNCT06290141
Reduced from 135 to 133 sites

Reduced from 135 to 133 sites

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 22argenxNCT06920004
Added 6 new sites: Nicholasville, Kentucky, São Paulo, Brazil, Calgary, Canada and 3 more

Added 6 new sites: Nicholasville, Kentucky, São Paulo, Brazil, Calgary, Canada and 3 more

sites_addedChronic Graft-versus-Host DiseaseSep 22SanofiNCT06290128
Added 7 new sites: Miami Lakes, Florida, New York, New York, Belo Horizonte, Brazil and 4 more

Added 7 new sites: Miami Lakes, Florida, New York, New York, Belo Horizonte, Brazil and 4 more

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22SanofiNCT06290128
Target enrollment decreased from 140 to 109

Target enrollment decreased from 140 to 109

eligibility_changeChronic Graft-versus-Host DiseaseSep 22University GhentNCT06892977
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 22Tr1X, Inc.NCT07477639
Added 6 new sites: La Jolla, California, Rochester, Minnesota, New York, New York and 3 more

Added 6 new sites: La Jolla, California, Rochester, Minnesota, New York, New York and 3 more

sites_addedChronic Graft-versus-Host DiseaseSep 22GigaGen, Inc.NCT07024641
Added 2 new sites: Songpa-dong, South Korea, Sanmin, Taiwan

Added 2 new sites: Songpa-dong, South Korea, Sanmin, Taiwan

sites_addedChronic Graft-versus-Host DiseaseSep 22argenxNCT07194850
Added 1 new site: Osaka, Japan

Added 1 new site: Osaka, Japan

sites_removedChronic Graft-versus-Host DiseaseSep 22Guangdong Provincial People's HospitalNCT04381143
Reduced from 139 to 127 sites

Reduced from 139 to 127 sites

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22Guangdong Provincial People's HospitalNCT04381143
Target enrollment increased from 9000 to 9082

Target enrollment increased from 9000 to 9082

sites_addedChronic Graft-versus-Host DiseaseSep 22National Cancer Institute (NCI)NCT04269902
Added 1 new site: Novi, Michigan

Added 1 new site: Novi, Michigan

sites_addedChronic Graft-versus-Host DiseaseSep 22Vironexis Biotherapeutics Inc.NCT06533579
Added 2 new sites: Jacksonville, Florida, Rochester, Minnesota

Added 2 new sites: Jacksonville, Florida, Rochester, Minnesota

sites_addedChronic Graft-versus-Host DiseaseSep 22Pulmonx CorporationNCT06035120
Added 2 new sites: Loveland, Colorado, Lexington, Kentucky

Added 2 new sites: Loveland, Colorado, Lexington, Kentucky

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22LG ChemNCT06206135
Target enrollment increased from 100 to 172

Target enrollment increased from 100 to 172

eligibility_changeChronic Graft-versus-Host DiseaseSep 22AstraZenecaNCT06087835
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedChronic Graft-versus-Host DiseaseSep 22BayerNCT05457283
Reduced from 179 to 151 sites

Reduced from 179 to 151 sites

sites_addedCholangiocarcinomaSep 22Astellas Pharma Global Development, Inc.NCT07488676
Added 5 new sites: Miami, Florida, Detroit, Michigan, New Brunswick, New Jersey and 2 more

Added 5 new sites: Miami, Florida, Detroit, Michigan, New Brunswick, New Jersey and 2 more

sites_addedDuchenne Muscular DystrophySep 22Sarepta Therapeutics, Inc.NCT04626674
Added 5 new sites: La Jolla, California, Los Angeles, California, Gainesville, Florida and 2 more

Added 5 new sites: La Jolla, California, Los Angeles, California, Gainesville, Florida and 2 more

eligibility_changeDuchenne Muscular DystrophySep 22Sarepta Therapeutics, Inc.NCT04626674
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDuchenne Muscular DystrophySep 22NS Pharma, Inc.NCT05996003
Added 5 new sites: Boston, Massachusetts, New York, New York, South Brisbane, Australia and 2 more

Added 5 new sites: Boston, Massachusetts, New York, New York, South Brisbane, Australia and 2 more

sites_addedDuchenne Muscular DystrophySep 22Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

sites_addedDuchenne Muscular DystrophySep 22Edgewise Therapeutics, Inc.NCT06066580
Added 18 new sites: Melbourne, Australia, Ghent, Belgium, Leuven, Belgium and 15 more

Added 18 new sites: Melbourne, Australia, Ghent, Belgium, Leuven, Belgium and 15 more

eligibility_changeDuchenne Muscular DystrophySep 22Edgewise Therapeutics, Inc.NCT06066580
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDuchenne Muscular DystrophySep 22Satellos Bioscience, Inc.NCT06867107
Added 3 new sites: Los Angeles, California, Columbus, Ohio, Memphis, Tennessee

Added 3 new sites: Los Angeles, California, Columbus, Ohio, Memphis, Tennessee

Enrollment UpdateDuchenne Muscular DystrophySep 22Satellos Bioscience, Inc.NCT06867107
Target enrollment increased from 10 to 30

Target enrollment increased from 10 to 30

eligibility_changeDuchenne Muscular DystrophySep 22Satellos Bioscience, Inc.NCT06867107
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeSep 22Longboard PharmaceuticalsNCT06908226
Added 53 new sites: Los Angeles, California, Palo Alto, California, Aurora, Colorado and 50 more

Added 53 new sites: Los Angeles, California, Palo Alto, California, Aurora, Colorado and 50 more

sites_addedDravet SyndromeSep 22Longboard PharmaceuticalsNCT06660394
Added 63 new sites: Los Angeles, California, Los Angeles, California, Palo Alto, California and 60 more

Added 63 new sites: Los Angeles, California, Los Angeles, California, Palo Alto, California and 60 more

eligibility_changeDermatomyositisSep 22National Cancer Institute (NCI)NCT03816345
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisSep 22University Hospital, LimogesNCT03582800
Added 3 new sites: Paris, France, Rennes, France, Toulouse, France

Added 3 new sites: Paris, France, Rennes, France, Toulouse, France

sites_removedDermatomyositisSep 22PfizerNCT05895786
Reduced from 146 to 138 sites

Reduced from 146 to 138 sites

sites_addedCystic FibrosisSep 22UCB Biopharma SRLNCT07720375
Added 1 new site: Brandon, Florida

Added 1 new site: Brandon, Florida

eligibility_changeCystic FibrosisSep 22UCB Biopharma SRLNCT07720375
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCystic FibrosisSep 22Paragonix TechnologiesNCT04930289
Target enrollment increased from 2000 to 3500

Target enrollment increased from 2000 to 3500

eligibility_changeCystic FibrosisSep 22Vertex Pharmaceuticals IncorporatedNCT07283770
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCystic FibrosisSep 22iSono Health, Inc.NCT07505797
Added 1 new site: Cypress, Texas

Added 1 new site: Cypress, Texas

sites_addedCongenital Adrenal HyperplasiaSep 22Neurocrine Switzerland GmbHNCT07536269
Added 1 new site: Aurora, Colorado

Added 1 new site: Aurora, Colorado

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 22argenxNCT06920004
Added 6 new sites: Cincinnati, Ohio, Córdoba, Argentina, Liverpool, Australia and 3 more

Added 6 new sites: Cincinnati, Ohio, Córdoba, Argentina, Liverpool, Australia and 3 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 22SanofiNCT06859099
Added 13 new sites: Scottsdale, Arizona, Brasília, Brazil, São Paulo, Brazil and 10 more

Added 13 new sites: Scottsdale, Arizona, Brasília, Brazil, São Paulo, Brazil and 10 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 22Kedrion S.p.A.NCT06752356
Added 2 new sites: Stamford, Connecticut, Miami, Florida

Added 2 new sites: Stamford, Connecticut, Miami, Florida

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 22argenxNCT07091630
Added 4 new sites: Knoxville, Tennessee, Guangzhou, China, Ramat Gan, Israel and 1 more

Added 4 new sites: Knoxville, Tennessee, Guangzhou, China, Ramat Gan, Israel and 1 more

sites_addedChronic Graft-versus-Host DiseaseSep 22Eli Lilly and CompanyNCT06672549
Added 14 new sites: Oxnard, California, Tainan, China, Taipei, China and 11 more

Added 14 new sites: Oxnard, California, Tainan, China, Taipei, China and 11 more

sites_addedChronic Graft-versus-Host DiseaseSep 22BeOne MedicinesNCT06973187
Added 5 new sites: New York, New York, Belo Horizonte, Brazil, Changchun, China and 2 more

Added 5 new sites: New York, New York, Belo Horizonte, Brazil, Changchun, China and 2 more

sites_addedChronic Graft-versus-Host DiseaseSep 22BeOne MedicinesNCT07277231
Added 24 new sites: Duarte, California, Orange, California, Aurora, Colorado and 21 more

Added 24 new sites: Duarte, California, Orange, California, Aurora, Colorado and 21 more

sites_addedChronic Graft-versus-Host DiseaseSep 22SanofiNCT07190222
Added 1 new site: Athens, Greece

Added 1 new site: Athens, Greece

sites_addedChronic Graft-versus-Host DiseaseSep 22Huahui HealthNCT07499544
Added 7 new sites: Garden Grove, California, Changchun, China, Kashgar, China and 4 more

Added 7 new sites: Garden Grove, California, Changchun, China, Kashgar, China and 4 more

sites_addedChronic Graft-versus-Host DiseaseSep 22Viz.ai, Inc.NCT07118306
Added 1 new site: Falls Church, Virginia

Added 1 new site: Falls Church, Virginia

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22M.D. Anderson Cancer CenterNCT06840262
Target enrollment increased from 45 to 54

Target enrollment increased from 45 to 54

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)NCT03756870
Target enrollment decreased from 82 to 48

Target enrollment decreased from 82 to 48

sites_addedChronic Graft-versus-Host DiseaseSep 22MedtronicNCT07507422
Added 3 new sites: San Diego, California, San Antonio, Texas, Lille, France

Added 3 new sites: San Diego, California, San Antonio, Texas, Lille, France

sites_removedChronic Graft-versus-Host DiseaseSep 22AstraZenecaNCT05637112
Reduced from 79 to 74 sites

Reduced from 79 to 74 sites

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22John Paul II Hospital, KrakowNCT05508035
Target enrollment decreased from 666 to 488

Target enrollment decreased from 666 to 488

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22iOMEDICO AGNCT05326308
Target enrollment increased from 705 to 845

Target enrollment increased from 705 to 845

sites_addedChronic Graft-versus-Host DiseaseSep 22Novartis PharmaceuticalsNCT07225504
Added 47 new sites: Phoenix, Arizona, Aurora, Colorado, Tampa, Florida and 44 more

Added 47 new sites: Phoenix, Arizona, Aurora, Colorado, Tampa, Florida and 44 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 22Novartis PharmaceuticalsNCT07225504
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 22Vifor Fresenius Medical Care Renal PharmaNCT06593392
Added 1 new site: Xi'an, China

Added 1 new site: Xi'an, China

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 22Northwestern UniversityNCT05719714
Target enrollment decreased from 60 to 18

Target enrollment decreased from 60 to 18

sites_addedChronic Graft-versus-Host DiseaseSep 22UCB Biopharma SRLNCT07725939
Added 4 new sites: Beijing, China, Chengdu, China, Hangzhou, China and 1 more

Added 4 new sites: Beijing, China, Chengdu, China, Hangzhou, China and 1 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 22University of PittsburghNCT07518719
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 22The University of QueenslandNCT03573089
Added 11 new sites: Ubá, Brazil, Winnipeg, Canada, North York, Canada and 8 more

Added 11 new sites: Ubá, Brazil, Winnipeg, Canada, North York, Canada and 8 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 22AstraZenecaNCT07024823
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 22Novartis PharmaceuticalsNCT07498335
Added 6 new sites: Beijing, China, Kanazawa, Japan, Chiba, Japan and 3 more

Added 6 new sites: Beijing, China, Kanazawa, Japan, Chiba, Japan and 3 more

sites_addedChronic Graft-versus-Host DiseaseSep 22Merz Therapeutics GmbHNCT07018713
Added 2 new sites: Rochester, Minnesota, Żory, Poland

Added 2 new sites: Rochester, Minnesota, Żory, Poland

Enrollment UpdateCholangiocarcinomaSep 22Arvinas Inc.NCT07023731
Target enrollment decreased from 159 to 76

Target enrollment decreased from 159 to 76

sites_addedAlagille SyndromeSep 21Digestive Care, Inc.NCT07335523
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

sites_addedBecker Muscular DystrophySep 21Edgewise Therapeutics, Inc.NCT06066580
Added 18 new sites: Melbourne, Australia, Ghent, Belgium, Leuven, Belgium and 15 more

Added 18 new sites: Melbourne, Australia, Ghent, Belgium, Leuven, Belgium and 15 more

eligibility_changeBecker Muscular DystrophySep 21Edgewise Therapeutics, Inc.NCT06066580
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeBecker Muscular DystrophySep 21West China HospitalNCT06114056
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedBecker Muscular DystrophySep 21Sarepta Therapeutics, Inc.NCT04626674
Added 5 new sites: La Jolla, California, Los Angeles, California, Gainesville, Florida and 2 more

Added 5 new sites: La Jolla, California, Los Angeles, California, Gainesville, Florida and 2 more

eligibility_changeBecker Muscular DystrophySep 21Sarepta Therapeutics, Inc.NCT04626674
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedBecker Muscular DystrophySep 21Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

sites_addedBecker Muscular DystrophySep 21NS Pharma, Inc.NCT05996003
Added 5 new sites: Boston, Massachusetts, New York, New York, South Brisbane, Australia and 2 more

Added 5 new sites: Boston, Massachusetts, New York, New York, South Brisbane, Australia and 2 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Sep 21BayerNCT07298044
Added 6 new sites: Ulm, Germany, Greifswald, Germany, Bonn, Germany and 3 more

Added 6 new sites: Ulm, Germany, Greifswald, Germany, Bonn, Germany and 3 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Sep 21Alnylam PharmaceuticalsNCT07052903
Added 12 new sites: Aurora, Colorado, Austin, Texas, Seattle, Washington and 9 more

Added 12 new sites: Aurora, Colorado, Austin, Texas, Seattle, Washington and 9 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Sep 21Alnylam PharmaceuticalsNCT07223203
Added 7 new sites: Rosario, Argentina, Rio de Janeiro, Brazil, Dublin, Ireland and 4 more

Added 7 new sites: Rosario, Argentina, Rio de Janeiro, Brazil, Dublin, Ireland and 4 more

eligibility_changeAplastic AnemiaSep 21National Heart, Lung, and Blood Institute (NHLBI)NCT03206086
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeAplastic AnemiaSep 21National Heart, Lung, and Blood Institute (NHLBI)NCT03173937
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisSep 21Nura BioNCT07369076
Added 4 new sites: Toronto, Canada, Utrecht, Netherlands, Krakow, Poland and 1 more

Added 4 new sites: Toronto, Canada, Utrecht, Netherlands, Krakow, Poland and 1 more

sites_addedAmyotrophic Lateral SclerosisSep 21Regeneron PharmaceuticalsNCT06351592
Added 2 new sites: Krakow, Poland, Huddinge, Sweden

Added 2 new sites: Krakow, Poland, Huddinge, Sweden

sites_removedAlport SyndromeSep 21BayerNCT07211685
Reduced from 60 to 58 sites

Reduced from 60 to 58 sites

eligibility_changeAlport SyndromeSep 21BayerNCT07211685
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAdrenocortical CarcinomaSep 21National Cancer Institute (NCI)NCT06900595
Added 1 new site: Fort Worth, Texas

Added 1 new site: Fort Worth, Texas

sites_addedWilson DiseaseSep 20Prime Medicine, Inc.NCT07748403
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedWild-Type ATTR Cardiac AmyloidosisSep 20Alnylam PharmaceuticalsNCT07052903
Added 12 new sites: Aurora, Colorado, Austin, Texas, Seattle, Washington and 9 more

Added 12 new sites: Aurora, Colorado, Austin, Texas, Seattle, Washington and 9 more

sites_addedWild-Type ATTR Cardiac AmyloidosisSep 20BayerNCT07298044
Added 6 new sites: Ulm, Germany, Greifswald, Germany, Bonn, Germany and 3 more

Added 6 new sites: Ulm, Germany, Greifswald, Germany, Bonn, Germany and 3 more

Enrollment UpdateWaldenström MacroglobulinemiaSep 19iOMEDICO AGNCT05326308
Target enrollment increased from 705 to 845

Target enrollment increased from 705 to 845

sites_addedWaldenström MacroglobulinemiaSep 19National Cancer Institute (NCI)NCT04840602
Added 6 new sites: Boise, Idaho, Caldwell, Idaho, Meridian, Idaho and 3 more

Added 6 new sites: Boise, Idaho, Caldwell, Idaho, Meridian, Idaho and 3 more

sites_addedVon Willebrand DiseaseSep 19TakedaNCT05582993
Added 2 new sites: Orlando, Florida, Oxford, United Kingdom

Added 2 new sites: Orlando, Florida, Oxford, United Kingdom

sites_addedVon Willebrand DiseaseSep 19Alnylam PharmaceuticalsNCT07575308
Added 8 new sites: San Diego, California, San Francisco, California, Pittsburgh, Pennsylvania and 5 more

Added 8 new sites: San Diego, California, San Francisco, California, Pittsburgh, Pennsylvania and 5 more

sites_addedVon Willebrand DiseaseSep 19Hoffmann-La RocheNCT06998524
Added 1 new site: Lexington, Kentucky

Added 1 new site: Lexington, Kentucky

sites_addedVon Willebrand DiseaseSep 19Vega Therapeutics, IncNCT07115004
Added 18 new sites: Palo Alto, California, New Haven, Connecticut, Ann Arbor, Michigan and 15 more

Added 18 new sites: Palo Alto, California, New Haven, Connecticut, Ann Arbor, Michigan and 15 more

sites_removedThalassemiaSep 19Bristol-Myers SquibbNCT05664737
Reduced from 36 to 31 sites

Reduced from 36 to 31 sites

sites_addedThalassemiaSep 19Novo Nordisk A/SNCT06609226
Expanded from 106 to 107 sites

Expanded from 106 to 107 sites

sites_removedSystemic Juvenile Idiopathic ArthritisSep 19AbbVieNCT05609630
Reduced from 48 to 45 sites

Reduced from 48 to 45 sites

Enrollment UpdateSystemic Juvenile Idiopathic ArthritisSep 19Changchun GeneScience Pharmaceutical Co., Ltd.NCT05925452
Target enrollment increased from 221 to 290

Target enrollment increased from 221 to 290

sites_addedStargardt DiseaseSep 19Astellas Institute for Regenerative MedicineNCT07734064
Added 2 new sites: Cincinnati, Ohio, Dallas, Texas

Added 2 new sites: Cincinnati, Ohio, Dallas, Texas

sites_addedStargardt DiseaseSep 19Alkeus Pharmaceuticals, Inc.NCT07419334
Added 4 new sites: Boston, Massachusetts, Philadelphia, Pennsylvania, Pittsburgh, Pennsylvania and 1 more

Added 4 new sites: Boston, Massachusetts, Philadelphia, Pennsylvania, Pittsburgh, Pennsylvania and 1 more

sites_removedSpinal Muscular AtrophySep 19Hoffmann-La RocheNCT05115110
Reduced from 35 to 30 sites

Reduced from 35 to 30 sites

Enrollment UpdateSpinal Muscular AtrophySep 19Hoffmann-La RocheNCT05115110
Target enrollment decreased from 259 to 97

Target enrollment decreased from 259 to 97

Enrollment UpdateHereditary Spastic Paraplegia Type 4Sep 19National Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
Target enrollment increased from 2323 to 2586

Target enrollment increased from 2323 to 2586

Enrollment UpdateSickle Cell DiseaseSep 19Novo Nordisk A/SNCT06612268
Target enrollment increased from 408 to 510

Target enrollment increased from 408 to 510

eligibility_changeSickle Cell DiseaseSep 19Novo Nordisk A/SNCT06612268
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateSickle Cell DiseaseSep 19National Heart, Lung, and Blood Institute (NHLBI)NCT05904093
Target enrollment increased from 25 to 35

Target enrollment increased from 25 to 35

sites_addedSickle Cell DiseaseSep 19Novartis PharmaceuticalsNCT06546670
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedSickle Cell DiseaseSep 19Novo Nordisk A/SNCT06609226
Expanded from 106 to 107 sites

Expanded from 106 to 107 sites

sites_addedSickle Cell DiseaseSep 19Nantes University HospitalNCT07274254
Added 1 new site: Bordeaux, France

Added 1 new site: Bordeaux, France

eligibility_changeSickle Cell DiseaseSep 19Nantes University HospitalNCT07274254
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSickle Cell DiseaseSep 19St. Jude Children's Research HospitalNCT07226219
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseSep 19Novartis PharmaceuticalsNCT06439082
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedSickle Cell DiseaseSep 19Disc Medicine, IncNCT07187973
Added 1 new site: Greenville, North Carolina

Added 1 new site: Greenville, North Carolina

sites_addedRett SyndromeSep 19UCB BIOSCIENCES, Inc.NCT07503444
Added 5 new sites: Miami, Florida, Paris, France, Naples, Italy and 2 more

Added 5 new sites: Miami, Florida, Paris, France, Naples, Italy and 2 more

Enrollment UpdatePulmonary Arterial HypertensionSep 18Paragonix TechnologiesNCT04930289
Target enrollment increased from 2000 to 3500

Target enrollment increased from 2000 to 3500

sites_addedPolycythemia VeraSep 18Novartis PharmaceuticalsNCT07357727
Added 1 new site: Jinan, China

Added 1 new site: Jinan, China

eligibility_changePolycythemia VeraSep 18Prelude Therapeutics IncorporatedNCT07469891
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeRett SyndromeSep 18Genecombio Ltd.NCT06739434
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateRetinitis PigmentosaSep 18Octant, Inc.NCT07408232
Target enrollment decreased from 32 to 18

Target enrollment decreased from 32 to 18

eligibility_changeRetinitis PigmentosaSep 18Octant, Inc.NCT07408232
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionSep 18Insmed IncorporatedNCT07481981
Added 6 new sites: Kansas City, Kansas, Omaha, Nebraska, Petah Tikva, Israel and 3 more

Added 6 new sites: Kansas City, Kansas, Omaha, Nebraska, Petah Tikva, Israel and 3 more

sites_addedPulmonary Arterial HypertensionSep 18Regeneron PharmaceuticalsNCT07318597
Added 3 new sites: Omaha, Nebraska, Cincinnati, Ohio, Strasbourg, France

Added 3 new sites: Omaha, Nebraska, Cincinnati, Ohio, Strasbourg, France

sites_addedProgressive Supranuclear PalsySep 18Adam BoxerNCT07173803
Added 4 new sites: Los Angeles, California, Aurora, Colorado, Cincinnati, Ohio and 1 more

Added 4 new sites: Los Angeles, California, Aurora, Colorado, Cincinnati, Ohio and 1 more

sites_addedPrimary Biliary CholangitisSep 18Gilead SciencesNCT06051617
Added 2 new sites: Creixomil, Portugal, Porto, Portugal

Added 2 new sites: Creixomil, Portugal, Porto, Portugal

Enrollment UpdatePrimary Biliary CholangitisSep 18Gilead SciencesNCT06051617
Target enrollment increased from 318 to 402

Target enrollment increased from 318 to 402

eligibility_changePrimary Biliary CholangitisSep 18Gilead SciencesNCT06051617
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraSep 18Novartis PharmaceuticalsNCT07357727
Added 6 new sites: Seattle, Washington, Aurangabad, India, Padova, Italy and 3 more

Added 6 new sites: Seattle, Washington, Aurangabad, India, Padova, Italy and 3 more

Enrollment UpdatePolycythemia VeraSep 18Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Target enrollment increased from 76 to 256

Target enrollment increased from 76 to 256

eligibility_changePolycythemia VeraSep 18Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraSep 18Disc Medicine, IncNCT05320198
Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

sites_addedParoxysmal Nocturnal HemoglobinuriaSep 18Regeneron PharmaceuticalsNCT05744921
Added 1 new site: Taichung, Taiwan

Added 1 new site: Taichung, Taiwan

sites_addedNiemann-Pick DiseaseSep 18SanofiNCT06192576
Added 2 new sites: Columbus, Ohio, St. George, Utah

Added 2 new sites: Columbus, Ohio, St. George, Utah

eligibility_changeNiemann-Pick DiseaseSep 18SanofiNCT06192576
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisSep 17Novartis PharmaceuticalsNCT07357727
Added 1 new site: Skorzewo, Poland

Added 1 new site: Skorzewo, Poland

Enrollment UpdateMyelofibrosisSep 17Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Target enrollment increased from 76 to 256

Target enrollment increased from 76 to 256

eligibility_changeMyelofibrosisSep 17Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesSep 17Regeneron PharmaceuticalsNCT05744921
Added 1 new site: Taichung, Taiwan

Added 1 new site: Taichung, Taiwan

sites_addedMyelodysplastic SyndromesSep 17Ryvu Therapeutics SANCT06987058
Added 7 new sites: Nice, France, Pessac, France, Chorzów, Poland and 4 more

Added 7 new sites: Nice, France, Pessac, France, Chorzów, Poland and 4 more

sites_addedMultiple SclerosisSep 17SanofiNCT07325292
Added 2 new sites: Baltimore, Maryland, Tokyo, Japan

Added 2 new sites: Baltimore, Maryland, Tokyo, Japan

eligibility_changeMultiple SclerosisSep 17Hoffmann-La RocheNCT04586023
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMultiple SclerosisSep 17Hoffmann-La RocheNCT04075266
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisSep 17Zenas BioPharma (USA), LLCNCT07299019
Added 5 new sites: Columbus, Ohio, Plovdiv, Bulgaria, Sofia, Bulgaria and 2 more

Added 5 new sites: Columbus, Ohio, Plovdiv, Bulgaria, Sofia, Bulgaria and 2 more

sites_addedMultiple SclerosisSep 17TG Therapeutics, Inc.NCT06433752
Added 17 new sites: Cullman, Alabama, Gainesville, Florida, Orlando, Florida and 14 more

Added 17 new sites: Cullman, Alabama, Gainesville, Florida, Orlando, Florida and 14 more

sites_addedMyotonic DystrophySep 17Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

sites_addedMyelofibrosisSep 17Disc Medicine, IncNCT05320198
Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

sites_addedMyelofibrosisSep 17Novartis PharmaceuticalsNCT07357727
Added 7 new sites: Seattle, Washington, Aurangabad, India, Padova, Italy and 4 more

Added 7 new sites: Seattle, Washington, Aurangabad, India, Padova, Italy and 4 more

sites_addedMyelodysplastic SyndromesSep 17BeOne MedicinesNCT04771130
Added 1 new site: Auckland, New Zealand

Added 1 new site: Auckland, New Zealand

sites_addedMyelodysplastic SyndromesSep 17Disc Medicine, IncNCT05320198
Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

Enrollment UpdateMyelodysplastic SyndromesSep 17Swedish Orphan BiovitrumNCT06782373
Target enrollment increased from 78 to 156

Target enrollment increased from 78 to 156

eligibility_changeMyelodysplastic SyndromesSep 17Swedish Orphan BiovitrumNCT06782373
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesSep 17Yale UniversityNCT07096297
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_addedMyasthenia GravisSep 17Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 5 new sites: Belgrade, Serbia, Niš, Serbia, Novi Sad, Serbia and 2 more

Added 5 new sites: Belgrade, Serbia, Niš, Serbia, Novi Sad, Serbia and 2 more

sites_addedMyasthenia GravisSep 17argenxNCT06392386
Added 1 new site: Randwick, Australia

Added 1 new site: Randwick, Australia

sites_addedMultiple System AtrophySep 17Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Marseille, France, Fuchū, Japan

Added 2 new sites: Marseille, France, Fuchū, Japan

eligibility_changeMultiple SclerosisSep 17University of CalgaryNCT06261489
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisSep 17National Institute of Allergy and Infectious Diseases (NIAID)NCT04047628
Added 1 new site: Detroit, Michigan

Added 1 new site: Detroit, Michigan

sites_addedMultiple SclerosisSep 17Zenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Tampa, Florida, Hradec Králové, Czechia

Added 2 new sites: Tampa, Florida, Hradec Králové, Czechia

sites_addedMultiple SclerosisSep 17Regeneron PharmaceuticalsNCT06351592
Added 2 new sites: Krakow, Poland, Huddinge, Sweden

Added 2 new sites: Krakow, Poland, Huddinge, Sweden

sites_removedMultiple SclerosisSep 17Hoffmann-La RocheNCT05704361
Reduced from 32 to 26 sites

Reduced from 32 to 26 sites

eligibility_changeMultiple SclerosisSep 17National Cancer Institute (NCI)NCT03816345
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMesotheliomaSep 17Memorial Sloan Kettering Cancer CenterNCT07532902
Eligibility criteria have been updated

Eligibility criteria have been updated

phase_changeMesotheliomaSep 17InSilico Medicine Hong Kong LimitedNCT06566079
Moved from Phase 1 to Phase 1, Phase 2

Moved from Phase 1 to Phase 1, Phase 2

Enrollment UpdateMesotheliomaSep 17InSilico Medicine Hong Kong LimitedNCT06566079
Target enrollment increased from 100 to 160

Target enrollment increased from 100 to 160

eligibility_changeMesotheliomaSep 17InSilico Medicine Hong Kong LimitedNCT06566079
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaSep 17SystImmune Inc.NCT07080242
Added 4 new sites: Glendale, California, Long Beach, California, New Orleans, Louisiana and 1 more

Added 4 new sites: Glendale, California, Long Beach, California, New Orleans, Louisiana and 1 more

eligibility_changeMerkel Cell CarcinomaSep 17SystImmune Inc.NCT07080242
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaSep 17Neonc Technologies, Inc.NCT06047379
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

Enrollment UpdateMedullary Thyroid CancerSep 17National Cancer Institute (NCI)NCT01660984
Target enrollment decreased from 259 to 105

Target enrollment decreased from 259 to 105

sites_addedImmune ThrombocytopeniaSep 16TakedaNCT06722235
Added 2 new sites: Madrid, Spain, Malmö, Sweden

Added 2 new sites: Madrid, Spain, Malmö, Sweden

sites_addedIgA NephropathySep 16TakedaNCT06963827
Added 2 new sites: Olomouc, Czechia, Birmingham, United Kingdom

Added 2 new sites: Olomouc, Czechia, Birmingham, United Kingdom

sites_addedIgA NephropathySep 16BiogenNCT06935357
Added 1 new site: La Boca, Argentina

Added 1 new site: La Boca, Argentina

sites_addedHodgkin LymphomaSep 16Novartis PharmaceuticalsNCT04094311
Expanded from 54 to 55 sites

Expanded from 54 to 55 sites

sites_addedHodgkin LymphomaSep 16Autolus LimitedNCT06173518
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_removedHodgkin LymphomaSep 16CelgeneNCT04884035
Reduced from 43 to 36 sites

Reduced from 43 to 36 sites

sites_addedHodgkin LymphomaSep 16National Cancer Institute (NCI)NCT04840602
Added 6 new sites: Boise, Idaho, Caldwell, Idaho, Meridian, Idaho and 3 more

Added 6 new sites: Boise, Idaho, Caldwell, Idaho, Meridian, Idaho and 3 more

sites_addedHodgkin LymphomaSep 16Fondazione Italiana Linfomi - ETSNCT05977673
Added 2 new sites: Novara, Italy, Reggio Emilia, Italy

Added 2 new sites: Novara, Italy, Reggio Emilia, Italy

sites_addedHodgkin LymphomaSep 16Regeneron PharmaceuticalsNCT06149286
Added 4 new sites: Essen, Germany, Bydgoszcz, Poland, Kaohsiung City, Taiwan and 1 more

Added 4 new sites: Essen, Germany, Bydgoszcz, Poland, Kaohsiung City, Taiwan and 1 more

Enrollment UpdateHodgkin LymphomaSep 16Regeneron PharmaceuticalsNCT06149286
Target enrollment increased from 470 to 580

Target enrollment increased from 470 to 580

eligibility_changeHodgkin LymphomaSep 16Regeneron PharmaceuticalsNCT06149286
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BSep 16CSL BehringNCT07080905
Added 1 new site: Brisbane, Australia

Added 1 new site: Brisbane, Australia

sites_addedLennox-Gastaut SyndromeSep 16Longboard PharmaceuticalsNCT06908226
Added 53 new sites: Los Angeles, California, Palo Alto, California, Aurora, Colorado and 50 more

Added 53 new sites: Los Angeles, California, Palo Alto, California, Aurora, Colorado and 50 more

sites_addedLennox-Gastaut SyndromeSep 16EpygenixNCT05066217
Added 3 new sites: Dallas, Texas, Genoa, Italy, Belgrade, Serbia

Added 3 new sites: Dallas, Texas, Genoa, Italy, Belgrade, Serbia

Enrollment UpdateIdiopathic Pulmonary FibrosisSep 16Mannkind CorporationNCT07679893
Target enrollment decreased from 210 to 153

Target enrollment decreased from 210 to 153

sites_addedIdiopathic Pulmonary FibrosisSep 16Trevi TherapeuticsNCT07671911
Added 4 new sites: Kissimmee, Florida, New Orleans, Louisiana, Winston-Salem, North Carolina and 1 more

Added 4 new sites: Kissimmee, Florida, New Orleans, Louisiana, Winston-Salem, North Carolina and 1 more

eligibility_changeIdiopathic Pulmonary FibrosisSep 16Trevi TherapeuticsNCT07671911
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaSep 16Northwestern UniversityNCT06242834
Target enrollment decreased from 32 to 11

Target enrollment decreased from 32 to 11

eligibility_changeHodgkin LymphomaSep 16Northwestern UniversityNCT06242834
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedHodgkin LymphomaSep 16Fondazione Italiana Linfomi - ETSNCT05300282
Reduced from 32 to 28 sites

Reduced from 32 to 28 sites

Enrollment UpdateHodgkin LymphomaSep 16Hoffmann-La RocheNCT06806033
Target enrollment increased from 100 to 130

Target enrollment increased from 100 to 130

eligibility_changeHodgkin LymphomaSep 16Hoffmann-La RocheNCT06806033
Eligibility criteria have been updated

Eligibility criteria have been updated

phase_changeHodgkin LymphomaSep 16M.D. Anderson Cancer CenterNCT07153796
Moved from Phase 2 to Phase 1, Phase 2

Moved from Phase 2 to Phase 1, Phase 2

sites_removedHodgkin LymphomaSep 16Lyell Immunopharma, Inc.NCT07188558
Reduced from 39 to 36 sites

Reduced from 39 to 36 sites

sites_addedHodgkin LymphomaSep 16Eastern Cooperative Oncology GroupNCT06724237
Added 7 new sites: Washington D.C., District of Columbia, Burr Ridge, Illinois, Homer Glen, Illinois and 4 more

Added 7 new sites: Washington D.C., District of Columbia, Burr Ridge, Illinois, Homer Glen, Illinois and 4 more

sites_addedHodgkin LymphomaSep 16National Cancer Institute (NCI)NCT07003295
Added 1 new site: Lexington, Kentucky

Added 1 new site: Lexington, Kentucky

sites_addedHodgkin LymphomaSep 16Eli Lilly and CompanyNCT07101328
Added 1 new site: Tokyo, Japan

Added 1 new site: Tokyo, Japan

sites_addedHodgkin LymphomaSep 16Fondazione Italiana Linfomi - ETSNCT05524298
Added 1 new site: Torino, Italy

Added 1 new site: Torino, Italy

sites_addedHodgkin LymphomaSep 16AstraZenecaNCT06564038
Expanded from 64 to 65 sites

Expanded from 64 to 65 sites

sites_removedHodgkin LymphomaSep 16BeOne MedicinesNCT05100862
Reduced from 266 to 264 sites

Reduced from 266 to 264 sites

sites_addedHodgkin LymphomaSep 16National Cancer Institute (NCI)NCT07332507
Added 3 new sites: Irvine, California, Los Angeles, California, Orange, California

Added 3 new sites: Irvine, California, Los Angeles, California, Orange, California

Enrollment UpdateHodgkin LymphomaSep 16M.D. Anderson Cancer CenterNCT06108232
Target enrollment increased from 33 to 83

Target enrollment increased from 33 to 83

sites_addedHodgkin LymphomaSep 16Merck Sharp & Dohme LLCNCT06717347
Added 5 new sites: Athens, Georgia, Reno, Nevada, Shirley, New York and 2 more

Added 5 new sites: Athens, Georgia, Reno, Nevada, Shirley, New York and 2 more

sites_addedHodgkin LymphomaSep 16Tempus AINCT07154823
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedHodgkin LymphomaSep 16Mission TherapeuticsNCT07630545
Added 13 new sites: Adelaide, Australia, Melbourne, Australia, Melbourne, Australia and 10 more

Added 13 new sites: Adelaide, Australia, Melbourne, Australia, Melbourne, Australia and 10 more

eligibility_changeHodgkin LymphomaSep 16Mission TherapeuticsNCT07630545
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 16Bristol-Myers SquibbNCT06090539
Added 4 new sites: Suzhou, China, Shenyang, China, Chuo-ku, Japan and 1 more

Added 4 new sites: Suzhou, China, Shenyang, China, Chuo-ku, Japan and 1 more

sites_addedHodgkin LymphomaSep 16Hoffmann-La RocheNCT07200375
Added 1 new site: Hong Kong, Hong Kong

Added 1 new site: Hong Kong, Hong Kong

Enrollment UpdateHodgkin LymphomaSep 16Fred Hutchinson Cancer CenterNCT03779854
Target enrollment decreased from 68 to 45

Target enrollment decreased from 68 to 45

sites_removedHodgkin LymphomaSep 16Fondazione Italiana Linfomi - ETSNCT06492837
Reduced from 24 to 20 sites

Reduced from 24 to 20 sites

sites_addedHodgkin LymphomaSep 16National Cancer Institute (NCI)NCT05675410
Added 5 new sites: Boise, Idaho, Caldwell, Idaho, Meridian, Idaho and 2 more

Added 5 new sites: Boise, Idaho, Caldwell, Idaho, Meridian, Idaho and 2 more

eligibility_changeFocal EpilepsySep 15NaviFUS CorporationNCT06388707
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaSep 15Novartis PharmaceuticalsNCT07357727
Added 3 new sites: Seattle, Washington, Aurangabad, India, Katowice, Poland

Added 3 new sites: Seattle, Washington, Aurangabad, India, Katowice, Poland

sites_addedEssential ThrombocythemiaSep 15Disc Medicine, IncNCT05320198
Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

Added 2 new sites: East Brunswick, New Jersey, Maumee, Ohio

phase_changeGeographic AtrophySep 15Janssen Research & Development, LLCNCT06635148
Moved from Phase 2 to Phase 2, Phase 3

Moved from Phase 2 to Phase 2, Phase 3

sites_removedEssential ThrombocythemiaSep 15Incyte CorporationNCT07623200
Reduced from 177 to 175 sites

Reduced from 177 to 175 sites

sites_addedEssential ThrombocythemiaSep 15Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Kobe, Japan, Fukuoka, Japan

Added 2 new sites: Kobe, Japan, Fukuoka, Japan

sites_addedPrimary HyperoxaluriaSep 14Novartis PharmaceuticalsNCT07681271
Added 3 new sites: Shanghai, China, Singapore, Singapore, Icheon, South Korea

Added 3 new sites: Shanghai, China, Singapore, Singapore, Icheon, South Korea

sites_addedPolycythemia VeraSep 14Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Kobe, Japan, Fukuoka, Japan

Added 2 new sites: Kobe, Japan, Fukuoka, Japan

Enrollment UpdatePheochromocytomaSep 14National Cancer Institute (NCI)NCT01660984
Target enrollment decreased from 259 to 105

Target enrollment decreased from 259 to 105

Enrollment UpdateParagangliomaSep 14National Cancer Institute (NCI)NCT01660984
Target enrollment decreased from 259 to 105

Target enrollment decreased from 259 to 105

sites_addedPulmonary Arterial HypertensionSep 14Merck Sharp & Dohme LLCNCT07646860
Added 2 new sites: Izumi, Japan, Suita, Japan

Added 2 new sites: Izumi, Japan, Suita, Japan

sites_addedPulmonary Arterial HypertensionSep 14Tenax Therapeutics, Inc.NCT07436689
Added 2 new sites: Boston, Massachusetts, Durham, North Carolina

Added 2 new sites: Boston, Massachusetts, Durham, North Carolina

sites_addedPulmonary Arterial HypertensionSep 14University of GiessenNCT06409624
Added 1 new site: Bad Nauheim, Germany

Added 1 new site: Bad Nauheim, Germany

Enrollment UpdatePulmonary Arterial HypertensionSep 14University of GiessenNCT06409624
Target enrollment increased from 350 to 400

Target enrollment increased from 350 to 400

eligibility_changePulmonary Arterial HypertensionSep 14University of GiessenNCT06409624
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePulmonary Arterial HypertensionSep 14National Institutes of Health Clinical Center (CC)NCT01712620
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionSep 1435Pharma IncNCT07143448
Added 2 new sites: Providence, Rhode Island, London, United Kingdom

Added 2 new sites: Providence, Rhode Island, London, United Kingdom

sites_addedPulmonary Arterial HypertensionSep 14Merck Sharp & Dohme LLCNCT07218029
Added 1 new site: Darlinghurst, Australia

Added 1 new site: Darlinghurst, Australia

sites_addedPulmonary Arterial HypertensionSep 14Pulmovant, Inc.NCT06635850
Added 85 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 82 more

Added 85 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 82 more

eligibility_changePulmonary Arterial HypertensionSep 14Pulmovant, Inc.NCT06635850
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPrimary HyperoxaluriaSep 14YolTech Therapeutics Co., LtdNCT07587021
Added 3 new sites: Aurora, Colorado, St Louis, Missouri, Philadelphia, Pennsylvania

Added 3 new sites: Aurora, Colorado, St Louis, Missouri, Philadelphia, Pennsylvania

sites_addedPolycythemia VeraSep 14Ono Pharmaceutical Co., Ltd.NCT07429266
Added 2 new sites: Macon, Georgia, Nagasaki, Japan

Added 2 new sites: Macon, Georgia, Nagasaki, Japan

sites_removedPolycythemia VeraSep 14Novartis PharmaceuticalsNCT07340138
Reduced from 7 to 3 sites

Reduced from 7 to 3 sites

Enrollment UpdatePolycythemia VeraSep 14Novartis PharmaceuticalsNCT07340138
Target enrollment decreased from 6 to 3

Target enrollment decreased from 6 to 3

sites_addedPolycythemia VeraSep 14Novartis PharmaceuticalsNCT07357727
Added 10 new sites: Indianapolis, Indiana, Albany, New York, Verona, Italy and 7 more

Added 10 new sites: Indianapolis, Indiana, Albany, New York, Verona, Italy and 7 more

eligibility_changeNeuromyelitis OpticaSep 13TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1Sep 13Centessa Pharmaceuticals (UK) LimitedNCT07598708
Added 5 new sites: Birmingham, Alabama, Brandon, Florida, Austin, Texas and 2 more

Added 5 new sites: Birmingham, Alabama, Brandon, Florida, Austin, Texas and 2 more

sites_addedMyotonic DystrophySep 13Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Oxford, United Kingdom

Added 1 new site: Oxford, United Kingdom

sites_removedMyelofibrosisSep 13Novartis PharmaceuticalsNCT07340138
Reduced from 7 to 3 sites

Reduced from 7 to 3 sites

Enrollment UpdateMyelofibrosisSep 13Novartis PharmaceuticalsNCT07340138
Target enrollment decreased from 6 to 3

Target enrollment decreased from 6 to 3

sites_addedMyelofibrosisSep 13Novartis PharmaceuticalsNCT07357727
Added 10 new sites: Indianapolis, Indiana, Albany, New York, Verona, Italy and 7 more

Added 10 new sites: Indianapolis, Indiana, Albany, New York, Verona, Italy and 7 more

eligibility_changeMyelodysplastic SyndromesSep 13National Heart, Lung, and Blood Institute (NHLBI)NCT03173937
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesSep 13National Cancer Institute (NCI)NCT01861106
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisSep 13AstraZenecaNCT07247279
Expanded from 7 to 8 sites

Expanded from 7 to 8 sites

sites_addedMyasthenia GravisSep 13UCB Biopharma SRLNCT07463521
Added 4 new sites: Chengdu, China, Nanchang, China, Suzhou, China and 1 more

Added 4 new sites: Chengdu, China, Nanchang, China, Suzhou, China and 1 more

eligibility_changeMyasthenia GravisSep 13TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisSep 13Vertex Pharmaceuticals IncorporatedNCT07501702
Added 1 new site: Kocaeli, Turkey (Türkiye)

Added 1 new site: Kocaeli, Turkey (Türkiye)

sites_removedMultiple System AtrophySep 13Hoffmann-La RocheNCT05115110
Reduced from 35 to 30 sites

Reduced from 35 to 30 sites

Enrollment UpdateMultiple System AtrophySep 13Hoffmann-La RocheNCT05115110
Target enrollment decreased from 259 to 97

Target enrollment decreased from 259 to 97

sites_addedMultiple SclerosisSep 13Zenas BioPharma (USA), LLCNCT07299019
Added 1 new site: Guaynabo, Puerto Rico

Added 1 new site: Guaynabo, Puerto Rico

sites_addedMultiple SclerosisSep 13Zenas BioPharma (USA), LLCNCT07067463
Added 8 new sites: Columbus, Ohio, Milwaukee, Wisconsin, Teplice, Czechia and 5 more

Added 8 new sites: Columbus, Ohio, Milwaukee, Wisconsin, Teplice, Czechia and 5 more

sites_addedMultiple SclerosisSep 13Genentech, Inc.NCT07609719
Added 2 new sites: Atlanta, Georgia, Cordova, Tennessee

Added 2 new sites: Atlanta, Georgia, Cordova, Tennessee

sites_addedMultiple SclerosisSep 13University of CalgaryNCT06261489
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

eligibility_changeMultiple SclerosisSep 13TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMultiple SclerosisSep 13Jung Diagnostics GmbHNCT07753928
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaSep 13AstraZenecaNCT06535607
Expanded from 97 to 98 sites

Expanded from 97 to 98 sites

sites_removedMembranous NephropathySep 13BeOne MedicinesNCT05707377
Reduced from 47 to 45 sites

Reduced from 47 to 45 sites

Enrollment UpdateLimb-Girdle Muscular DystrophySep 12ML Bio Solutions, Inc.NCT05775848
Target enrollment increased from 81 to 112

Target enrollment increased from 81 to 112

sites_addedLight Chain AmyloidosisSep 12Tianjin Medical UniversityNCT07232459
Added 1 new site: Qingdao, China

Added 1 new site: Qingdao, China

eligibility_changeIgA NephropathySep 12Purespring Therapeutics LimitedNCT07182227
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathySep 12Novartis PharmaceuticalsNCT06994845
Added 4 new sites: Madera, California, Gainesville, Florida, Córdoba, Argentina and 1 more

Added 4 new sites: Madera, California, Gainesville, Florida, Córdoba, Argentina and 1 more

sites_addedHereditary AngioedemaSep 12ADARx Pharmaceuticals, Inc.NCT06960213
Added 18 new sites: Scottsdale, Arizona, Santa Monica, California, Centennial, Colorado and 15 more

Added 18 new sites: Scottsdale, Arizona, Santa Monica, California, Centennial, Colorado and 15 more

sites_addedIdiopathic Pulmonary FibrosisSep 12Contineum TherapeuticsNCT07284459
Added 3 new sites: Buenos Aires, Argentina, Toronto, Canada, Trois-Rivières, Canada

Added 3 new sites: Buenos Aires, Argentina, Toronto, Canada, Trois-Rivières, Canada

sites_removedHunter SyndromeSep 12Denali Therapeutics Inc.NCT05371613
Reduced from 32 to 30 sites

Reduced from 32 to 30 sites

sites_removedHomozygous Familial HypercholesterolemiaSep 12CRISPR Therapeutics AGNCT07491172
Reduced from 19 to 17 sites

Reduced from 19 to 17 sites

eligibility_changeHodgkin LymphomaSep 12American Society of Clinical OncologyNCT02693535
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 12Yazeed SawalhaNCT06536049
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

Enrollment UpdateHodgkin LymphomaSep 12iOMEDICO AGNCT05326308
Target enrollment increased from 705 to 845

Target enrollment increased from 705 to 845

eligibility_changeHodgkin LymphomaSep 12AbbVieNCT07387471
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 12Merck Sharp & Dohme LLCNCT07519772
Added 4 new sites: Pilar, Argentina, Santiago, Chile, Giessen, Germany and 1 more

Added 4 new sites: Pilar, Argentina, Santiago, Chile, Giessen, Germany and 1 more

sites_removedHodgkin LymphomaSep 12Hoffmann-La RocheNCT02568267
Reduced from 130 to 120 sites

Reduced from 130 to 120 sites

sites_addedHodgkin LymphomaSep 12Novartis PharmaceuticalsNCT07422610
Added 2 new sites: Milan, Italy, Milan, Italy

Added 2 new sites: Milan, Italy, Milan, Italy

eligibility_changeHodgkin LymphomaSep 12Treeline Biosciences, Inc.NCT07082803
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 12National Cancer Institute (NCI)NCT03739814
Added 3 new sites: Evanston, Illinois, Glenview, Illinois, Highland Park, Illinois

Added 3 new sites: Evanston, Illinois, Glenview, Illinois, Highland Park, Illinois

sites_removedHodgkin LymphomaSep 12Imugene LimitedNCT03666000
Reduced from 23 to 15 sites

Reduced from 23 to 15 sites

eligibility_changeHodgkin LymphomaSep 12Imugene LimitedNCT03666000
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHereditary Spastic ParaplegiaSep 12National Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
Target enrollment increased from 2323 to 2586

Target enrollment increased from 2323 to 2586

sites_addedHemophilia BSep 11Regeneron PharmaceuticalsNCT06379789
Added 3 new sites: Vitória, Brazil, Campinas, Brazil, Rio de Janeiro, Brazil

Added 3 new sites: Vitória, Brazil, Campinas, Brazil, Rio de Janeiro, Brazil

sites_addedHemophilia ASep 11Regeneron PharmaceuticalsNCT06379789
Added 3 new sites: Vitória, Brazil, Campinas, Brazil, Rio de Janeiro, Brazil

Added 3 new sites: Vitória, Brazil, Campinas, Brazil, Rio de Janeiro, Brazil

eligibility_changeHemophilia ASep 11Hoffmann-La RocheNCT05987449
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsySep 11Xenon Pharmaceuticals Inc.NCT05718817
Added 1 new site: Heeze, Netherlands

Added 1 new site: Heeze, Netherlands

sites_addedHemophilia ASep 11Hoffmann-La RocheNCT07416526
Added 9 new sites: Godoy Cruz, Argentina, Ribeirão Preto, Brazil, Copenhaen, Denmark and 6 more

Added 9 new sites: Godoy Cruz, Argentina, Ribeirão Preto, Brazil, Copenhaen, Denmark and 6 more

sites_removedHemophilia ASep 11Novo Nordisk A/SNCT05685238
Reduced from 152 to 141 sites

Reduced from 152 to 141 sites

sites_addedGeographic AtrophySep 11Novartis PharmaceuticalsNCT07441642
Added 4 new sites: Barranquilla, Colombia, Kobe, Japan, Newcastle upon Tyne, United Kingdom and 1 more

Added 4 new sites: Barranquilla, Colombia, Kobe, Japan, Newcastle upon Tyne, United Kingdom and 1 more

sites_addedGeographic AtrophySep 11ONL TherapeuticsNCT06659445
Added 3 new sites: Chevy Chase, Maryland, Hagerstown, Maryland, San Antonio, Texas

Added 3 new sites: Chevy Chase, Maryland, Hagerstown, Maryland, San Antonio, Texas

sites_addedGastrointestinal Stromal TumorSep 11GlaxoSmithKlineNCT07218926
Added 34 new sites: Duarte, California, Upland, California, Aurora, Colorado and 31 more

Added 34 new sites: Duarte, California, Upland, California, Aurora, Colorado and 31 more

sites_addedFocal Segmental GlomerulosclerosisSep 11Boehringer IngelheimNCT07220083
Added 7 new sites: Kansas City, Kansas, Orangeburg, South Carolina, Conroe, Texas and 4 more

Added 7 new sites: Kansas City, Kansas, Orangeburg, South Carolina, Conroe, Texas and 4 more

sites_removedEssential ThrombocythemiaSep 11Novartis PharmaceuticalsNCT07340138
Reduced from 7 to 3 sites

Reduced from 7 to 3 sites

Enrollment UpdateEssential ThrombocythemiaSep 11Novartis PharmaceuticalsNCT07340138
Target enrollment decreased from 6 to 3

Target enrollment decreased from 6 to 3

sites_addedEssential ThrombocythemiaSep 11Novartis PharmaceuticalsNCT07357727
Added 10 new sites: Indianapolis, Indiana, Albany, New York, Verona, Italy and 7 more

Added 10 new sites: Indianapolis, Indiana, Albany, New York, Verona, Italy and 7 more

eligibility_changeDuchenne Muscular DystrophySep 10West China HospitalNCT06114056
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisSep 10Allogene TherapeuticsNCT07085104
Added 2 new sites: Baltimore, Maryland, Charlotte, North Carolina

Added 2 new sites: Baltimore, Maryland, Charlotte, North Carolina

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 10Immunovant Sciences GmbHNCT07032662
Added 1 new site: Buenos Aires, Argentina

Added 1 new site: Buenos Aires, Argentina

sites_addedChronic Graft-versus-Host DiseaseSep 10Gilead SciencesNCT07096193
Added 8 new sites: La Jolla, California, Hanover, Germany, Ankara, Turkey (Türkiye) and 5 more

Added 8 new sites: La Jolla, California, Hanover, Germany, Ankara, Turkey (Türkiye) and 5 more

sites_addedChronic Graft-versus-Host DiseaseSep 10Granular Therapeutics LimitedNCT07181369
Added 1 new site: Halle, Germany

Added 1 new site: Halle, Germany

sites_addedChronic Graft-versus-Host DiseaseSep 10Weill Medical College of Cornell UniversityNCT06168058
Added 1 new site: New Hyde Park, New York

Added 1 new site: New Hyde Park, New York

sites_addedChronic Graft-versus-Host DiseaseSep 10AstraZenecaNCT06564038
Expanded from 64 to 65 sites

Expanded from 64 to 65 sites

sites_addedChronic Graft-versus-Host DiseaseSep 10Airiver Medical, Inc.NCT07290140
Added 2 new sites: Charleston, South Carolina, Charleston, South Carolina

Added 2 new sites: Charleston, South Carolina, Charleston, South Carolina

eligibility_changeChronic Graft-versus-Host DiseaseSep 10University of California, Los AngelesNCT04556890
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaSep 10Francesco De CobelliNCT06375915
Added 3 new sites: Bergamo, Italy, Pisa, Italy, Naples, Italy

Added 3 new sites: Bergamo, Italy, Pisa, Italy, Naples, Italy

sites_addedDuchenne Muscular DystrophySep 10Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Oxford, United Kingdom

Added 1 new site: Oxford, United Kingdom

Enrollment UpdateDilated CardiomyopathySep 10AstraZenecaNCT07241104
Target enrollment decreased from 31 to 23

Target enrollment decreased from 31 to 23

eligibility_changeDilated CardiomyopathySep 10AstraZenecaNCT07241104
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCystic FibrosisSep 10Royal College of Surgeons, IrelandNCT04602468
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCystic FibrosisSep 10AstraZenecaNCT07245407
Target enrollment decreased from 320 to 251

Target enrollment decreased from 320 to 251

sites_addedCushing DiseaseSep 10Sparrow PharmaceuticalsNCT07296484
Added 3 new sites: Winter Park, Florida, Wichita, Kansas, Vienna, Virginia

Added 3 new sites: Winter Park, Florida, Wichita, Kansas, Vienna, Virginia

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 10Grifols Therapeutics LLCNCT07540221
Added 3 new sites: Hradec Králové, Czechia, Bydgoszcz, Poland, Katowice, Poland

Added 3 new sites: Hradec Králové, Czechia, Bydgoszcz, Poland, Katowice, Poland

eligibility_changeChronic Inflammatory Demyelinating PolyneuropathySep 10TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Granulomatous DiseaseSep 10National Institute of Allergy and Infectious Diseases (NIAID)NCT06253507
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 10Novartis PharmaceuticalsNCT06868212
Added 5 new sites: Palo Alto, California, Chicago, Illinois, Boston, Massachusetts and 2 more

Added 5 new sites: Palo Alto, California, Chicago, Illinois, Boston, Massachusetts and 2 more

sites_addedChronic Graft-versus-Host DiseaseSep 10AstraZenecaNCT06542250
Added 1 new site: Göttingen, Germany

Added 1 new site: Göttingen, Germany

sites_addedChronic Graft-versus-Host DiseaseSep 10AstraZenecaNCT07509151
Added 14 new sites: La Jolla, California, Denver, Colorado, New Haven, Connecticut and 11 more

Added 14 new sites: La Jolla, California, Denver, Colorado, New Haven, Connecticut and 11 more

sites_addedChronic Graft-versus-Host DiseaseSep 10Akebia TherapeuticsNCT07268638
Added 11 new sites: Boca Raton, Florida, Hialeah, Florida, Worcester, Massachusetts and 8 more

Added 11 new sites: Boca Raton, Florida, Hialeah, Florida, Worcester, Massachusetts and 8 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 10Akebia TherapeuticsNCT07268638
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 10University of PittsburghNCT06605807
Target enrollment decreased from 150 to 64

Target enrollment decreased from 150 to 64

sites_addedChronic Graft-versus-Host DiseaseSep 10CytokineticsNCT06736574
Added 27 new sites: Nanning, China, Chifeng, China, Xuzhou, China and 24 more

Added 27 new sites: Nanning, China, Chifeng, China, Xuzhou, China and 24 more

sites_addedChronic Graft-versus-Host DiseaseSep 10AstraZenecaNCT07662135
Added 2 new sites: Sun City, Arizona, Calicut, India

Added 2 new sites: Sun City, Arizona, Calicut, India

eligibility_changeChronic Graft-versus-Host DiseaseSep 10EnsomaNCT06876363
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseSep 10VA Office of Research and DevelopmentNCT04124029
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 10Newave Pharmaceutical IncNCT07342478
Added 1 new site: Skokie, Illinois

Added 1 new site: Skokie, Illinois

sites_addedChronic Graft-versus-Host DiseaseSep 10Viridian Therapeutics, Inc.NCT06625398
Added 24 new sites: San Diego, California, Berlin, Germany, Dresden, Germany and 21 more

Added 24 new sites: San Diego, California, Berlin, Germany, Dresden, Germany and 21 more

sites_addedChronic Graft-versus-Host DiseaseSep 10Merck Sharp & Dohme LLCNCT06136559
Added 2 new sites: Boise, Idaho, Iowa City, Iowa

Added 2 new sites: Boise, Idaho, Iowa City, Iowa

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 10Boston UniversityNCT05503173
Target enrollment decreased from 385 to 253

Target enrollment decreased from 385 to 253

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 10SanofiNCT06087627
Target enrollment decreased from 150 to 110

Target enrollment decreased from 150 to 110

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 10Washington University School of MedicineNCT07020273
Target enrollment decreased from 5000 to 3543

Target enrollment decreased from 5000 to 3543

eligibility_changeChronic Graft-versus-Host DiseaseSep 10Massachusetts General HospitalNCT06843902
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 10University of South CarolinaNCT06096701
Target enrollment decreased from 540 to 421

Target enrollment decreased from 540 to 421

sites_addedChronic Graft-versus-Host DiseaseSep 10BiocadNCT07119372
Added 14 new sites: Krasnodar, Russia, Krasnodar, Russia, Novosibirsk, Russia and 11 more

Added 14 new sites: Krasnodar, Russia, Krasnodar, Russia, Novosibirsk, Russia and 11 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 10BiocadNCT07119372
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 10SanofiNCT07190209
Added 55 new sites: Miami, Florida, Miami, Florida, Chicago, Illinois and 52 more

Added 55 new sites: Miami, Florida, Miami, Florida, Chicago, Illinois and 52 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Sep 9Alnylam PharmaceuticalsNCT07358078
Added 20 new sites: Tucson, Arizona, Beverly Hills, California, Kansas City, Kansas and 17 more

Added 20 new sites: Tucson, Arizona, Beverly Hills, California, Kansas City, Kansas and 17 more

eligibility_changeAmyotrophic Lateral SclerosisSep 9University of IowaNCT07224269
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisSep 9Trace Neuroscience, Inc.NCT07674667
Added 2 new sites: Rostock, Germany, Sheffield, United Kingdom

Added 2 new sites: Rostock, Germany, Sheffield, United Kingdom

sites_addedBecker Muscular DystrophySep 9Avidity Biosciences, Inc.NCT07587242
Added 1 new site: Oxford, United Kingdom

Added 1 new site: Oxford, United Kingdom

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Sep 9Alnylam PharmaceuticalsNCT05040373
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAngelman SyndromeSep 9OHB Pediatrics Ltd.NCT07605429
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedAlpha-1 Antitrypsin DeficiencySep 9Tessera Therapeutics, Inc.NCT07227207
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedSpinocerebellar AtaxiaSep 8IntraBio IncNCT07221292
Added 1 new site: Newcastle upon Tyne, United Kingdom

Added 1 new site: Newcastle upon Tyne, United Kingdom

sites_removedSpinal Muscular AtrophySep 8CelgeneNCT06764485
Reduced from 281 to 212 sites

Reduced from 281 to 212 sites

Enrollment UpdateSickle Cell DiseaseSep 8St. Jude Children's Research HospitalNCT06442761
Target enrollment increased from 15 to 20

Target enrollment increased from 15 to 20

eligibility_changeSickle Cell DiseaseSep 8St. Jude Children's Research HospitalNCT06442761
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedWilson DiseaseSep 8Prime Medicine, Inc.NCT07748403
Added 1 new site: San Antonio, Texas

Added 1 new site: San Antonio, Texas

eligibility_changeWaldenström MacroglobulinemiaSep 8AbbVieNCT07387471
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedTurner SyndromeSep 8Ascendis Pharma A/SNCT07221851
Added 3 new sites: Tübingen, Germany, Iași, Romania, Suwon, South Korea

Added 3 new sites: Tübingen, Germany, Iași, Romania, Suwon, South Korea

Enrollment UpdateSystemic MastocytosisSep 8Fred Hutchinson Cancer CenterNCT03779854
Target enrollment decreased from 68 to 45

Target enrollment decreased from 68 to 45

sites_addedSystemic MastocytosisSep 8Blueprint Medicines CorporationNCT07264959
Added 4 new sites: Aurora, Colorado, Normal, Illinois, St Louis, Missouri and 1 more

Added 4 new sites: Aurora, Colorado, Normal, Illinois, St Louis, Missouri and 1 more

Enrollment UpdateStargardt DiseaseSep 8VeonGen Therapeutics GmbHNCT07002398
Target enrollment increased from 15 to 18

Target enrollment increased from 15 to 18

eligibility_changeSpinocerebellar AtaxiaSep 8Lexeo TherapeuticsNCT06865482
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSpinal Muscular AtrophySep 8ORIC PharmaceuticalsNCT07723248
Added 5 new sites: Jeffersonville, Indiana, Canton, Ohio, Dallas, Texas and 2 more

Added 5 new sites: Jeffersonville, Indiana, Canton, Ohio, Dallas, Texas and 2 more

sites_addedSpinal Muscular AtrophySep 8BiogenNCT06555419
Added 2 new sites: Phoenix, Arizona, Orlando, Florida

Added 2 new sites: Phoenix, Arizona, Orlando, Florida

eligibility_changeSevere Combined ImmunodeficiencySep 7National Institute of Allergy and Infectious Diseases (NIAID)NCT06851767
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionSep 7Tempus AINCT06911632
Added 5 new sites: Riverside, California, Springfield, Missouri, New York, New York and 2 more

Added 5 new sites: Riverside, California, Springfield, Missouri, New York, New York and 2 more

sites_addedPulmonary Arterial HypertensionSep 7Insmed IncorporatedNCT07481981
Added 17 new sites: Los Angeles, California, Torrance, California, Rochester, New York and 14 more

Added 17 new sites: Los Angeles, California, Torrance, California, Rochester, New York and 14 more

sites_addedPulmonary Arterial HypertensionSep 7Pulmovant, Inc.NCT07333183
Added 13 new sites: Santa Barbara, California, Torrance, California, Kissimmee, Florida and 10 more

Added 13 new sites: Santa Barbara, California, Torrance, California, Kissimmee, Florida and 10 more

sites_addedPulmonary Arterial HypertensionSep 7Liquidia Technologies, Inc.NCT07285655
Added 20 new sites: Phoenix, Arizona, Los Angeles, California, San Francisco, California and 17 more

Added 20 new sites: Phoenix, Arizona, Los Angeles, California, San Francisco, California and 17 more

sites_addedPulmonary Arterial HypertensionSep 7Inhibikase TherapeuticsNCT07365332
Added 41 new sites: Scottsdale, Arizona, Beverly Hills, California, Santa Barbara, California and 38 more

Added 41 new sites: Scottsdale, Arizona, Beverly Hills, California, Santa Barbara, California and 38 more

eligibility_changePulmonary Arterial HypertensionSep 7University of PennsylvaniaNCT07132788
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraSep 7Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedPolycythemia VeraSep 7Novartis PharmaceuticalsNCT07357727
Added 1 new site: Adelaide, Australia

Added 1 new site: Adelaide, Australia

sites_addedPolycystic Kidney DiseaseSep 7Calico Life Sciences LLCNCT06902558
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedNoonan SyndromeSep 6Ascendis Pharma A/SNCT07221851
Added 3 new sites: Tübingen, Germany, Iași, Romania, Suwon, South Korea

Added 3 new sites: Tübingen, Germany, Iași, Romania, Suwon, South Korea

eligibility_changeNeurofibromatosis Type 1Sep 6National Cancer Institute (NCI)NCT06222203
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1Sep 6Alkermes, Inc.NCT07540897
Added 3 new sites: Brandon, Florida, Frisco, Texas, Krakow, Poland

Added 3 new sites: Brandon, Florida, Frisco, Texas, Krakow, Poland

sites_addedNarcolepsy Type 1Sep 6Alkermes, Inc.NCT07455383
Added 13 new sites: Novi, Michigan, Madison, Wisconsin, Bruges, Belgium and 10 more

Added 13 new sites: Novi, Michigan, Madison, Wisconsin, Bruges, Belgium and 10 more

Enrollment UpdateNarcolepsy Type 1Sep 6TakedaNCT07299097
Target enrollment increased from 100 to 402

Target enrollment increased from 100 to 402

sites_addedMyotonic DystrophySep 6Dyne TherapeuticsNCT07486934
Added 3 new sites: Palo Alto, California, Fitzroy, Australia, Aarhus, Denmark

Added 3 new sites: Palo Alto, California, Fitzroy, Australia, Aarhus, Denmark

sites_addedMyelofibrosisSep 6Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedMyelofibrosisSep 6Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Jupiter, Florida, Houston, Texas, Adelaide, Australia and 1 more

Added 4 new sites: Jupiter, Florida, Houston, Texas, Adelaide, Australia and 1 more

sites_addedMyelodysplastic SyndromesSep 6National Cancer Institute (NCI)NCT05564390
Added 1 new site: Ottawa, Canada

Added 1 new site: Ottawa, Canada

Enrollment UpdateMyelodysplastic SyndromesSep 6Auron Therapeutics, Inc.NCT06846606
Target enrollment increased from 34 to 69

Target enrollment increased from 34 to 69

eligibility_changeMyelodysplastic SyndromesSep 6Auron Therapeutics, Inc.NCT06846606
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesSep 6National Cancer Institute (NCI)NCT06577441
Added 1 new site: Hines, Illinois

Added 1 new site: Hines, Illinois

Enrollment UpdateMyelodysplastic SyndromesSep 6Fred Hutchinson Cancer CenterNCT03779854
Target enrollment decreased from 68 to 45

Target enrollment decreased from 68 to 45

eligibility_changeMyelodysplastic SyndromesSep 6National Cancer Institute (NCI)NCT00843882
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesSep 6Hannah Choe, MDNCT07249346
Added 2 new sites: Birmingham, Alabama, Boston, Massachusetts

Added 2 new sites: Birmingham, Alabama, Boston, Massachusetts

sites_addedMyelodysplastic SyndromesSep 6Therapeutic Advances in Childhood Leukemia ConsortiumNCT05849662
Added 2 new sites: Charlotte, North Carolina, Dallas, Texas

Added 2 new sites: Charlotte, North Carolina, Dallas, Texas

sites_addedMyasthenia GravisSep 6Centre Hospitalier Universitaire de NiceNCT07072988
Added 8 new sites: Créteil, France, Garches, France, Grenoble, France and 5 more

Added 8 new sites: Créteil, France, Garches, France, Grenoble, France and 5 more

sites_addedMyasthenia GravisSep 6AmgenNCT06987539
Added 6 new sites: Philadelphia, Pennsylvania, CABA, Argentina, Ciudad Autonoma de Buenos Aires, Argentina and 3 more

Added 6 new sites: Philadelphia, Pennsylvania, CABA, Argentina, Ciudad Autonoma de Buenos Aires, Argentina and 3 more

eligibility_changeMyasthenia GravisSep 6AmgenNCT06987539
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisSep 6Vertex Pharmaceuticals IncorporatedNCT07501702
Added 1 new site: Fatih, Turkey (Türkiye)

Added 1 new site: Fatih, Turkey (Türkiye)

eligibility_changeMyasthenia GravisSep 6Vertex Pharmaceuticals IncorporatedNCT07501702
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisSep 6Centre Hospitalier Universitaire de NiceNCT07570589
Added 10 new sites: Angers, France, Boulogne-sur-Mer, France, Créteil, France and 7 more

Added 10 new sites: Angers, France, Boulogne-sur-Mer, France, Créteil, France and 7 more

Enrollment UpdateMyasthenia GravisSep 6Cartesian TherapeuticsNCT06799247
Target enrollment increased from 100 to 128

Target enrollment increased from 100 to 128

sites_addedMyasthenia GravisSep 6Centre Hospitalier Universitaire de NiceNCT07411963
Added 7 new sites: Bron, France, Garches, France, Le Kremlin-Bicêtre, France and 4 more

Added 7 new sites: Bron, France, Garches, France, Le Kremlin-Bicêtre, France and 4 more

sites_addedMyasthenia GravisSep 6Candid TherapeuticsNCT07215650
Added 9 new sites: Fuzhou, China, Guangzhou, China, Guangzhou, China and 6 more

Added 9 new sites: Fuzhou, China, Guangzhou, China, Guangzhou, China and 6 more

eligibility_changeMultiple System AtrophySep 6Synusight Biotech (Shanghai) Co., Ltd.NCT07604116
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple System AtrophySep 6Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Kansas City, Kansas, Catania, Italy

Added 2 new sites: Kansas City, Kansas, Catania, Italy

sites_addedMultiple SclerosisSep 6Zenas BioPharma (USA), LLCNCT07299019
Added 1 new site: Pozzilli, Italy

Added 1 new site: Pozzilli, Italy

Enrollment UpdateMixed Connective Tissue DiseaseSep 6Fred Hutchinson Cancer CenterNCT03779854
Target enrollment decreased from 68 to 45

Target enrollment decreased from 68 to 45

Enrollment UpdateMesotheliomaSep 6A2 Biotherapeutics Inc.NCT04981119
Target enrollment increased from 200 to 500

Target enrollment increased from 200 to 500

sites_addedMembranous NephropathySep 6Vertex Pharmaceuticals IncorporatedNCT07204275
Added 3 new sites: Sacramento, California, Shreveport, Louisiana, Nijmegen, Netherlands

Added 3 new sites: Sacramento, California, Shreveport, Louisiana, Nijmegen, Netherlands

Enrollment UpdateMastocytosisSep 6National Institute of Allergy and Infectious Diseases (NIAID)NCT00044122
Target enrollment increased from 600 to 700

Target enrollment increased from 600 to 700

Enrollment UpdateMastocytosisSep 6Fred Hutchinson Cancer CenterNCT03779854
Target enrollment decreased from 68 to 45

Target enrollment decreased from 68 to 45

sites_addedMastocytosisSep 6Blueprint Medicines CorporationNCT07264959
Added 4 new sites: Aurora, Colorado, Normal, Illinois, St Louis, Missouri and 1 more

Added 4 new sites: Aurora, Colorado, Normal, Illinois, St Louis, Missouri and 1 more

sites_addedIgA NephropathySep 5ADARx Pharmaceuticals, Inc.NCT06989359
Added 3 new sites: Bologna, Italy, Kaohsiung City, Taiwan, Leicester, United Kingdom

Added 3 new sites: Bologna, Italy, Kaohsiung City, Taiwan, Leicester, United Kingdom

sites_addedIgA NephropathySep 5BiogenNCT06935357
Added 2 new sites: Shenyang, China, Bari, Italy

Added 2 new sites: Shenyang, China, Bari, Italy

sites_addedIdiopathic Pulmonary FibrosisSep 5AbbVieNCT07230288
Added 9 new sites: Box Hill, Australia, Montpellier, France, Rennes, France and 6 more

Added 9 new sites: Box Hill, Australia, Montpellier, France, Rennes, France and 6 more

sites_addedIdiopathic Pulmonary FibrosisSep 5Cumberland PharmaceuticalsNCT05571059
Added 1 new site: Farmington, Connecticut

Added 1 new site: Farmington, Connecticut

eligibility_changeIdiopathic Pulmonary FibrosisSep 5Cumberland PharmaceuticalsNCT05571059
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisSep 5Chiesi Farmaceutici S.p.A.NCT07516951
Added 1 new site: Denison, Texas

Added 1 new site: Denison, Texas

sites_addedIdiopathic Pulmonary FibrosisSep 5United TherapeuticsNCT04905693
Added 21 new sites: Buenos Aires, Argentina, Antwerp, Belgium, Liège, Belgium and 18 more

Added 21 new sites: Buenos Aires, Argentina, Antwerp, Belgium, Liège, Belgium and 18 more

sites_addedIdiopathic Pulmonary FibrosisSep 5Trevi TherapeuticsNCT07671911
Added 10 new sites: Newport Beach, California, Palm Springs, California, Redding, California and 7 more

Added 10 new sites: Newport Beach, California, Palm Springs, California, Redding, California and 7 more

sites_addedHypoparathyroidismSep 5MBX BiosciencesNCT07699471
Added 3 new sites: Metairie, Louisiana, Albany, New York, Summerville, South Carolina

Added 3 new sites: Metairie, Louisiana, Albany, New York, Summerville, South Carolina

sites_addedHuntington DiseaseSep 5Novartis PharmaceuticalsNCT07326709
Added 26 new sites: Birmingham, Alabama, Little Rock, Arkansas, Atlanta, Georgia and 23 more

Added 26 new sites: Birmingham, Alabama, Little Rock, Arkansas, Atlanta, Georgia and 23 more

Enrollment UpdateHuntington DiseaseSep 5Hoffmann-La RocheNCT07246941
Target enrollment decreased from 40 to 3

Target enrollment decreased from 40 to 3

sites_addedHuntington DiseaseSep 5Sarepta Therapeutics, Inc.NCT07536061
Added 3 new sites: Westmead, Australia, Gümligen, Switzerland, Cardiff, United Kingdom

Added 3 new sites: Westmead, Australia, Gümligen, Switzerland, Cardiff, United Kingdom

Enrollment UpdateHomozygous Familial HypercholesterolemiaSep 5First Affiliated Hospital Xi'an Jiaotong UniversityNCT06125847
Target enrollment increased from 12 to 21

Target enrollment increased from 12 to 21

eligibility_changeHomozygous Familial HypercholesterolemiaSep 5First Affiliated Hospital Xi'an Jiaotong UniversityNCT06125847
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaSep 5The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical SchoolNCT07083154
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaSep 5Memorial Sloan Kettering Cancer CenterNCT07223021
Target enrollment decreased from 130 to 65

Target enrollment decreased from 130 to 65

eligibility_changeHodgkin LymphomaSep 5BeOne MedicinesNCT06634589
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 5Merck Sharp & Dohme LLCNCT07570173
Added 1 new site: Athens, Greece

Added 1 new site: Athens, Greece

sites_addedHodgkin LymphomaSep 5National Cancer Institute (NCI)NCT04546399
Added 4 new sites: New Brunswick, New Jersey, Albuquerque, New Mexico, Tulsa, Oklahoma and 1 more

Added 4 new sites: New Brunswick, New Jersey, Albuquerque, New Mexico, Tulsa, Oklahoma and 1 more

sites_addedHodgkin LymphomaSep 5Merck Sharp & Dohme LLCNCT06395103
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

sites_addedHodgkin LymphomaSep 5AstraZenecaNCT06549595
Added 4 new sites: Chesterfield, Missouri, Wilmington, North Carolina, Cincinnati, Ohio and 1 more

Added 4 new sites: Chesterfield, Missouri, Wilmington, North Carolina, Cincinnati, Ohio and 1 more

sites_addedHodgkin LymphomaSep 5Medical College of WisconsinNCT07117136
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

eligibility_changeHodgkin LymphomaSep 5Medical College of WisconsinNCT07117136
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 5Janssen Research & Development, LLCNCT06788509
Added 2 new sites: Copenhagen, Denmark, Odense C, Denmark

Added 2 new sites: Copenhagen, Denmark, Odense C, Denmark

sites_addedHodgkin LymphomaSep 5Daiichi SankyoNCT07220616
Added 2 new sites: Villejuif, France, Bologna, Italy

Added 2 new sites: Villejuif, France, Bologna, Italy

sites_addedHodgkin LymphomaSep 5Allogene TherapeuticsNCT06500273
Added 2 new sites: Lone Tree, Colorado, Suwon, South Korea

Added 2 new sites: Lone Tree, Colorado, Suwon, South Korea

Enrollment UpdateHodgkin LymphomaSep 5National Cancer Institute (NCI)NCT04739813
Target enrollment decreased from 55 to 34

Target enrollment decreased from 55 to 34

Enrollment UpdateHodgkin LymphomaSep 5Baylor College of MedicineNCT04288726
Target enrollment increased from 18 to 27

Target enrollment increased from 18 to 27

eligibility_changeHodgkin LymphomaSep 5Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityNCT03198026
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaSep 5National Cancer Institute (NCI)NCT05675410
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedHereditary Hemorrhagic TelangiectasiaSep 5Atavistik Bio, IncNCT07601425
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedHereditary AngioedemaSep 5Ionis Pharmaceuticals, Inc.NCT07298447
Added 3 new sites: West Allis, Wisconsin, South Brisbane, Australia, Clayton, Australia

Added 3 new sites: West Allis, Wisconsin, South Brisbane, Australia, Clayton, Australia

sites_addedHereditary AngioedemaSep 5Pharvaris Netherlands B.V.NCT06679881
Added 8 new sites: Colorado Springs, Colorado, Wheaton, Illinois, Box Hill, Australia and 5 more

Added 8 new sites: Colorado Springs, Colorado, Wheaton, Illinois, Box Hill, Australia and 5 more

sites_addedHemophilia ASep 4Hoffmann-La RocheNCT07416604
Added 12 new sites: Godoy Cruz, Argentina, Rosario, Argentina, Brussels, Belgium and 9 more

Added 12 new sites: Godoy Cruz, Argentina, Rosario, Argentina, Brussels, Belgium and 9 more

sites_addedGraves’ DiseaseSep 4Yarrow Bioscience, Inc.NCT07682896
Added 3 new sites: Tucson, Arizona, Raleigh, North Carolina, Dallas, Texas

Added 3 new sites: Tucson, Arizona, Raleigh, North Carolina, Dallas, Texas

sites_addedGraves’ DiseaseSep 4BiogenNCT07722546
Added 3 new sites: Glendale, Arizona, Doral, Florida, Miami, Florida

Added 3 new sites: Glendale, Arizona, Doral, Florida, Miami, Florida

sites_addedGranulomatosis with PolyangiitisSep 4Novartis PharmaceuticalsNCT06868290
Added 6 new sites: Chicago, Illinois, Durham, North Carolina, Philadelphia, Pennsylvania and 3 more

Added 6 new sites: Chicago, Illinois, Durham, North Carolina, Philadelphia, Pennsylvania and 3 more

sites_addedEpidermolysis BullosaSep 4Paradigm TherapeuticsNCT07482787
Added 1 new site: Charleston, South Carolina

Added 1 new site: Charleston, South Carolina

sites_addedGraves’ DiseaseSep 4Biohaven Therapeutics Ltd.NCT07661056
Added 14 new sites: Tucson, Arizona, Oakland, California, Fort Myers, Florida and 11 more

Added 14 new sites: Tucson, Arizona, Oakland, California, Fort Myers, Florida and 11 more

eligibility_changeGraves’ DiseaseSep 4Biohaven Therapeutics Ltd.NCT07661056
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseSep 4Candid TherapeuticsNCT07597200
Added 13 new sites: Hefei, China, Beijing, China, Fuzhou, China and 10 more

Added 13 new sites: Hefei, China, Beijing, China, Fuzhou, China and 10 more

sites_addedGeographic AtrophySep 4Astellas Institute for Regenerative MedicineNCT03178149
Added 1 new site: Portland, Oregon

Added 1 new site: Portland, Oregon

sites_addedGeographic AtrophySep 4Complement TherapeuticsNCT07144137
Added 3 new sites: Belfast, United Kingdom, Frimley, United Kingdom, Manchester, United Kingdom

Added 3 new sites: Belfast, United Kingdom, Frimley, United Kingdom, Manchester, United Kingdom

sites_addedGeographic AtrophySep 4ADARx Pharmaceuticals, Inc.NCT06990269
Added 46 new sites: Riverside, California, Sutherland, Australia, Sydney, Australia and 43 more

Added 46 new sites: Riverside, California, Sutherland, Australia, Sydney, Australia and 43 more

eligibility_changeFriedreich AtaxiaSep 4Lexeo TherapeuticsNCT06865482
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsySep 4Rapport Therapeutics Inc.NCT07563881
Added 1 new site: Asheville, North Carolina

Added 1 new site: Asheville, North Carolina

sites_addedFocal EpilepsySep 4Rapport Therapeutics Inc.NCT07594119
Added 5 new sites: Huntsville, Alabama, Chicago, Illinois, New York, New York and 2 more

Added 5 new sites: Huntsville, Alabama, Chicago, Illinois, New York, New York and 2 more

sites_addedFanconi AnemiaSep 4Duke Street Bio LtdNCT06458712
Added 3 new sites: Sacramento, California, Lodz, Poland, Pamplona, Spain

Added 3 new sites: Sacramento, California, Lodz, Poland, Pamplona, Spain

sites_addedFabry DiseaseSep 4Amicus TherapeuticsNCT06904261
Added 1 new site: Sagamihara, Japan

Added 1 new site: Sagamihara, Japan

sites_addedEssential ThrombocythemiaSep 4Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_removedEssential ThrombocythemiaSep 4Incyte CorporationNCT07623200
Reduced from 180 to 177 sites

Reduced from 180 to 177 sites

sites_addedEssential ThrombocythemiaSep 4Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Jupiter, Florida, Houston, Texas, Adelaide, Australia and 1 more

Added 4 new sites: Jupiter, Florida, Houston, Texas, Adelaide, Australia and 1 more

sites_addedEpidermolysis BullosaSep 4Paradigm TherapeuticsNCT07482787
Added 1 new site: Redwood City, California

Added 1 new site: Redwood City, California

sites_addedDravet SyndromeSep 3Ionis Pharmaceuticals, Inc.NCT07531745
Added 2 new sites: Durham, North Carolina, Montreal, Canada

Added 2 new sites: Durham, North Carolina, Montreal, Canada

sites_addedCold Agglutinin DiseaseSep 3Lakefront Biotherapeutics West LLCNCT07083960
Added 4 new sites: Duarte, California, Washington D.C., District of Columbia, Ann Arbor, Michigan and 1 more

Added 4 new sites: Duarte, California, Washington D.C., District of Columbia, Ann Arbor, Michigan and 1 more

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 3Rush University Medical CenterNCT06044649
Target enrollment decreased from 460 to 408

Target enrollment decreased from 460 to 408

sites_addedChronic Graft-versus-Host DiseaseSep 3AmgenNCT07684235
Added 4 new sites: Skokie, Illinois, Winchester, Virginia, Minato-ku, Japan and 1 more

Added 4 new sites: Skokie, Illinois, Winchester, Virginia, Minato-ku, Japan and 1 more

sites_addedChronic Graft-versus-Host DiseaseSep 3Merck Sharp & Dohme LLCNCT05458297
Added 1 new site: Montreal, Canada

Added 1 new site: Montreal, Canada

sites_addedChronic Graft-versus-Host DiseaseSep 3TakedaNCT06948318
Added 7 new sites: Hot Springs, Arkansas, Indianapolis, Indiana, Kansas City, Missouri and 4 more

Added 7 new sites: Hot Springs, Arkansas, Indianapolis, Indiana, Kansas City, Missouri and 4 more

sites_addedChronic Graft-versus-Host DiseaseSep 3Novartis PharmaceuticalsNCT04877522
Added 2 new sites: Milwaukee, Wisconsin, Kobe, Japan

Added 2 new sites: Milwaukee, Wisconsin, Kobe, Japan

eligibility_changeChronic Graft-versus-Host DiseaseSep 3VA Office of Research and DevelopmentNCT04887688
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseSep 3National Institute of Allergy and Infectious Diseases (NIAID)NCT06476132
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 3SanofiNCT06290141
Added 13 new sites: Centennial, Colorado, Miami Lakes, Florida, New York, New York and 10 more

Added 13 new sites: Centennial, Colorado, Miami Lakes, Florida, New York, New York and 10 more

sites_addedChronic Graft-versus-Host DiseaseSep 3Novartis PharmaceuticalsNCT06675864
Added 3 new sites: Mainz, Germany, Barcelona, Spain, Madrid, Spain

Added 3 new sites: Mainz, Germany, Barcelona, Spain, Madrid, Spain

sites_addedChronic Graft-versus-Host DiseaseSep 3AbbVieNCT06603558
Added 32 new sites: Busan, South Korea, Busan, South Korea, Busan, South Korea and 29 more

Added 32 new sites: Busan, South Korea, Busan, South Korea, Busan, South Korea and 29 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 3HealthPartners InstituteNCT07491861
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEosinophilic Granulomatosis with PolyangiitisSep 3University of PennsylvaniaNCT00315380
Added 2 new sites: Cambridge, United Kingdom, London, United Kingdom

Added 2 new sites: Cambridge, United Kingdom, London, United Kingdom

sites_removedEosinophilic EsophagitisSep 3Dr. Falk Pharma GmbHNCT06596252
Reduced from 27 to 21 sites

Reduced from 27 to 21 sites

sites_addedDilated CardiomyopathySep 3Affinia TherapeuticsNCT07426419
Added 1 new site: Montreal, Canada

Added 1 new site: Montreal, Canada

phase_changeDesmoplastic Small Round Cell TumorSep 3National Cancer Institute (NCI)NCT04616560
Moved from Phase 1, Phase 2 to Phase 2

Moved from Phase 1, Phase 2 to Phase 2

sites_addedCongenital Adrenal HyperplasiaSep 3Crinetics Pharmaceuticals Inc.NCT07144163
Added 5 new sites: Gainesville, Florida, Graz, Austria, Hiroshima, Japan and 2 more

Added 5 new sites: Gainesville, Florida, Graz, Austria, Hiroshima, Japan and 2 more

sites_addedCongenital Adrenal HyperplasiaSep 3Assistance Publique - Hôpitaux de ParisNCT06900153
Added 3 new sites: Bordeaux, France, Créteil, France, Le Kremlin-Bicêtre, France

Added 3 new sites: Bordeaux, France, Créteil, France, Le Kremlin-Bicêtre, France

sites_addedCongenital Adrenal HyperplasiaSep 3Crinetics Pharmaceuticals Inc.NCT07159841
Added 4 new sites: Santiago, Chile, Cologne, Germany, Bialystok, Poland and 1 more

Added 4 new sites: Santiago, Chile, Cologne, Germany, Bialystok, Poland and 1 more

eligibility_changeCommon Variable ImmunodeficiencySep 3Memorial Sloan Kettering Cancer CenterNCT06976476
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 3Janssen Research & Development, LLCNCT05327114
Added 1 new site: Augusta, Georgia

Added 1 new site: Augusta, Georgia

sites_addedChronic Inflammatory Demyelinating PolyneuropathySep 3Dianthus TherapeuticsNCT06858579
Added 4 new sites: Serdang, Malaysia, Ankara, Turkey (Türkiye), Hanoi, Vietnam and 1 more

Added 4 new sites: Serdang, Malaysia, Ankara, Turkey (Türkiye), Hanoi, Vietnam and 1 more

sites_addedChronic Graft-versus-Host DiseaseSep 3Janssen Research & Development, LLCNCT05963074
Added 1 new site: El Paso, Texas

Added 1 new site: El Paso, Texas

sites_addedChronic Graft-versus-Host DiseaseSep 3PfizerNCT07363694
Added 32 new sites: Tampa, Florida, Kansas City, Kansas, Fall River, Massachusetts and 29 more

Added 32 new sites: Tampa, Florida, Kansas City, Kansas, Fall River, Massachusetts and 29 more

sites_addedChronic Graft-versus-Host DiseaseSep 3LMU KlinikumNCT05554835
Added 18 new sites: Munich, Germany, Halle, Germany, München, Germany and 15 more

Added 18 new sites: Munich, Germany, Halle, Germany, München, Germany and 15 more

sites_addedChronic Graft-versus-Host DiseaseSep 3SanofiNCT07116031
Added 4 new sites: Seattle, Washington, Vancouver, Canada, Turin, Italy and 1 more

Added 4 new sites: Seattle, Washington, Vancouver, Canada, Turin, Italy and 1 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 3SanofiNCT07116031
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseSep 3Centre Hospitalier Universitaire de Saint EtienneNCT05780762
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseSep 3Lomond Therapeutics Holdings, Inc.NCT06708897
Added 1 new site: Dallas, Texas

Added 1 new site: Dallas, Texas

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 3Lomond Therapeutics Holdings, Inc.NCT06708897
Target enrollment increased from 66 to 84

Target enrollment increased from 66 to 84

eligibility_changeChronic Graft-versus-Host DiseaseSep 3Lomond Therapeutics Holdings, Inc.NCT06708897
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseSep 3VA Office of Research and DevelopmentNCT06004804
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseSep 3St. Olavs HospitalNCT05372822
Target enrollment decreased from 87 to 40

Target enrollment decreased from 87 to 40

sites_addedChronic Graft-versus-Host DiseaseSep 3Fondazione Don Carlo Gnocchi ETSNCT06547827
Added 14 new sites: Bergamo, Italy, Catania, Italy, Catanzaro, Italy and 11 more

Added 14 new sites: Bergamo, Italy, Catania, Italy, Catanzaro, Italy and 11 more

eligibility_changeChronic Graft-versus-Host DiseaseSep 3University of MinnesotaNCT06808048
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaSep 3UNICANCERNCT06529718
Added 1 new site: Grenoble, France

Added 1 new site: Grenoble, France

Enrollment UpdateAmyotrophic Lateral SclerosisSep 2Dallas VA Medical CenterNCT04244630
Target enrollment decreased from 60 to 21

Target enrollment decreased from 60 to 21

sites_addedAplastic AnemiaSep 2Medical College of WisconsinNCT07227155
Added 8 new sites: Duarte, California, Atlanta, Georgia, Chicago, Illinois and 5 more

Added 8 new sites: Duarte, California, Atlanta, Georgia, Chicago, Illinois and 5 more

sites_addedAmyotrophic Lateral SclerosisSep 2Zydus Therapeutics Inc.NCT07023835
Added 32 new sites: Caulfield South, Australia, Sydney, Australia, Southport, Australia and 29 more

Added 32 new sites: Caulfield South, Australia, Sydney, Australia, Southport, Australia and 29 more

sites_addedAmyotrophic Lateral SclerosisSep 2Insmed Gene Therapy LLCNCT07290062
Added 2 new sites: Baltimore, Maryland, Worcester, Massachusetts

Added 2 new sites: Baltimore, Maryland, Worcester, Massachusetts

sites_addedAdrenocortical CarcinomaSep 2National Cancer Institute (NCI)NCT06900595
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedAchondroplasiaSep 2Tyra Biosciences, IncNCT06842355
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

eligibility_changeThalassemiaSep 1National Heart, Lung, and Blood Institute (NHLBI)NCT05904093
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSickle Cell DiseaseSep 1National Heart, Lung, and Blood Institute (NHLBI)NCT05904093
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedUrea Cycle DisordersSep 1iECURE, Inc.NCT06805695
Added 2 new sites: Aurora, Colorado, New York, New York

Added 2 new sites: Aurora, Colorado, New York, New York

sites_addedThalassemiaSep 1Agios Pharmaceuticals, Inc.NCT07506863
Added 2 new sites: New York, New York, Philadelphia, Pennsylvania

Added 2 new sites: New York, New York, Philadelphia, Pennsylvania

sites_addedThalassemiaSep 1Agios Pharmaceuticals, Inc.NCT07517133
Added 3 new sites: Palo Alto, California, New York, New York, Philadelphia, Pennsylvania

Added 3 new sites: Palo Alto, California, New York, New York, Philadelphia, Pennsylvania

sites_addedStargardt DiseaseSep 1Alkeus Pharmaceuticals, Inc.NCT07419334
Added 5 new sites: Aurora, Colorado, Grand Rapids, Michigan, Columbus, Ohio and 2 more

Added 5 new sites: Aurora, Colorado, Grand Rapids, Michigan, Columbus, Ohio and 2 more