Rare Disease Trial Updates: May 2026

In May 2026, Trial Friend tracked 771 rare disease clinical trial updates across 116 conditions, including 7 FDA-related actions and 107 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 ChangeMultiple SclerosisMay 31SandozNCT06847724
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 31NYU Langone HealthNCT06816004
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaMay 31University of Wisconsin, MadisonNCT06608511
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaMay 31H. Lee Moffitt Cancer Center and Research InstituteNCT04975152
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeLight Chain AmyloidosisMay 30Lantheus Germany GmbHNCT05184088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 30Karyopharm Therapeutics IncNCT04442022
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 30City of Hope Medical CenterNCT05157971
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 30AbbVieNCT05618028
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 30OHSU Knight Cancer InstituteNCT04872790
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 30M.D. Anderson Cancer CenterNCT07153796
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 30Regeneron PharmaceuticalsNCT06230224
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia AMay 29University Hospital, Clermont-FerrandNCT05202951
No longer enrolling new participants

No longer enrolling new participants

FDA SafetyMay 29
FDA Issues Draft Guidance to Cut Unnecessary Animal Testing for Cancer Drugs

The U.S. Food and Drug Administration today issued a draft guidance to reduce unnecessary animal testing in nonclinical safety assessments for certain cancer drugs.

Status ChangeGranulomatosis with PolyangiitisMay 29NS Pharma, Inc.NCT06046222
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFragile X SyndromeMay 29Zynerba Pharmaceuticals, Inc.NCT03802799
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 28Vertex Pharmaceuticals IncorporatedNCT05422222
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 28UConn HealthNCT05705193
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic Granulomatosis with PolyangiitisMay 28NS Pharma, Inc.NCT06046222
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 28Vertex Pharmaceuticals IncorporatedNCT05668741
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 28Vertex Pharmaceuticals IncorporatedNCT06154447
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 28Massachusetts General HospitalNCT05747534
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 28Arthrosi TherapeuticsNCT07116746
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 28University of Mississippi Medical CenterNCT05687058
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCharcot-Marie-Tooth DiseaseMay 28University Hospital, Basel, SwitzerlandNCT05336864
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)May 27PfizerNCT07494656
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)May 27Lantheus Germany GmbHNCT05184088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAplastic AnemiaMay 27Fred Hutchinson Cancer CenterNCT06752694
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWaldenström MacroglobulinemiaMay 26Shayna Sarosiek, MDNCT05190705
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaMay 25Octant, Inc.NCT07408232
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionMay 25Gradient Denervation TechnologiesNCT06052072
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeParoxysmal Nocturnal HemoglobinuriaMay 25Novartis PharmaceuticalsNCT06606314
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 24Novartis PharmaceuticalsNCT06606314
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMixed Connective Tissue DiseaseMay 24Jose Roberto CortelliNCT07036302
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisMay 23Alnylam PharmaceuticalsNCT06360289
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 23New York Medical CollegeNCT07013565
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHodgkin LymphomaMay 23University Medical Center GroningenNCT06180733
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 23Regeneron PharmaceuticalsNCT06091865
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

FDA ApprovalMay 22
FDA Approves First Treatment for Chronic Hepatitis Delta Virus (HDV) Infection

Today, the U.S. Food and Drug Administration approved Hepcludex (bulevirtide-gmod) injection to treat chronic hepatitis delta virus (HDV) infection in adults without cirrhosis (advanced liver scarring) or with compensated cirrhosis.

Status ChangeCushing DiseaseMay 21Assistance Publique - Hôpitaux de ParisNCT04840693
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 21Taizhou Mabtech Pharmaceutical Co.,LtdNCT06365879
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)May 20Alnylam PharmaceuticalsNCT06360289
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaMay 18SparingVisionNCT05748873
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionMay 18University of PennsylvaniaNCT07132788
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePrimary Biliary CholangitisMay 18Zydus Therapeutics Inc.NCT06427395
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNoonan SyndromeMay 17Day One Biopharmaceuticals, Inc.NCT05566795
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 17Stanford UniversityNCT05088356
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyMay 17Washington University School of MedicineNCT05826457
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMultiple SclerosisMay 17SanofiNCT06141473
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 17SanofiNCT06141486
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 17TG Therapeutics, Inc.NCT07211633
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMixed Connective Tissue DiseaseMay 17Valent Technologies, LLCNCT04337177
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 16Children's Oncology GroupNCT05602194
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 16Institute of Hematology & Blood Diseases Hospital, ChinaNCT06504199
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 16Canadian Cancer Trials GroupNCT04161248
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 16Karyopharm Therapeutics IncNCT02227251
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 16University of UtahNCT05723055
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary AngioedemaMay 16CSL BehringNCT06806657
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFacioscapulohumeral Muscular DystrophyMay 15Avidity Biosciences, Inc.NCT06547216
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFriedreich AtaxiaMay 15BiogenNCT06054893
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal Segmental GlomerulosclerosisMay 15The Hospital for Sick ChildrenNCT06635720
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 14University of AlbertaNCT03672877
No longer enrolling new participants

No longer enrolling new participants

Status ChangeArrhythmogenic CardiomyopathyMay 13St. George's Hospital, LondonNCT04257994
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeAmyotrophic Lateral SclerosisMay 13University of AlbertaNCT03362658
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWilson DiseaseMay 12Prime Medicine, Inc.NCT07226622
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseMay 12City of Hope Medical CenterNCT03279094
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

FDA DesignationChronic Graft-versus-Host DiseaseMay 12
FDA grants orphan drug designation to rituximab for Chronic Graft-versus-Host Disease

The FDA granted orphan drug designation to rituximab, developed by ODDIFACT SAS, on 2026-05-12 for the treatment of chronic graft-versus-host disease. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.

Status ChangeSpinocerebellar AtaxiaMay 12BiogenNCT06054893
No longer enrolling new participants

No longer enrolling new participants

FDA NewsMay 11
FDA Advances Drug Repurposing to Address Unmet Medical Needs

FDA announced it is soliciting input on efforts with respect to drug repurposing to help address unmet medical needs across a range of diseases and conditions.

Status ChangeRetinitis PigmentosaMay 11University Hospital TuebingenNCT06323772
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Langerhans Cell HistiocytosisMay 11Novartis PharmaceuticalsNCT03975829
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePulmonary Arterial HypertensionMay 11Saglik Bilimleri UniversitesiNCT07193173
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraMay 11John MascarenhasNCT05467800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeurofibromatosis Type 1May 10Novartis PharmaceuticalsNCT03975829
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyelofibrosisMay 10John MascarenhasNCT05467800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 10M.D. Anderson Cancer CenterNCT04493138
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 10H. Lee Moffitt Cancer Center and Research InstituteNCT05732961
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 10Bristol-Myers SquibbNCT05949684
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 10University of ExeterNCT06274671
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 10ProJenXNCT05279755
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisMay 9Attralus, Inc.NCT05951049
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisMay 9National Cancer Institute (NCI)NCT04847453
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseMay 9Universitaire Ziekenhuizen KU LeuvenNCT06626412
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomocystinuriaMay 9Travere Therapeutics, Inc.NCT06247085
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHodgkin LymphomaMay 9Poseida Therapeutics, Inc.NCT06014762
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 9Kite, A Gilead CompanyNCT06539338
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 9Miltenyi Biomedicine GmbHNCT04844866
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHereditary Spastic ParaplegiaMay 9National Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalCholangiocarcinomaMay 8
FDA Grants Seventh Approval under the National Priority Voucher Pilot Program

FDA issued an approval for Bizengri (zenocutuzumab-zbco), a drug that treats NRG1 fusion-positive cholangiocarcinoma, an ultra-rare, aggressive cancer that forms in the bile ducts.

FDA ApprovalMay 8
FDA Issues Guidance to Improve Collection of Pregnancy Safety Data for Drugs and Biologics

The U.S. Food and Drug Administration today issued a final guidance for industry, Postapproval Pregnancy Safety Studies, with recommendations on different methodologies that can be used in the postapproval setting to study the safety of drugs and biological products when used during pregnancy.

Status ChangeGeographic AtrophyMay 8Hoffmann-La RocheNCT06961370
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaMay 8John MascarenhasNCT05467800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 7Centre Hospitalier Universitaire de la RéunionNCT06371261
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 7Sionna Therapeutics Inc.NCT07108153
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseMay 7AbbVieNCT06125795
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlpha-1 Antitrypsin DeficiencyMay 6BioMarin PharmaceuticalNCT06738017
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAdrenocortical CarcinomaMay 6Columbia UniversityNCT03127774
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic Paraplegia Type 4May 5National Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionMay 4Tempus AINCT06911632
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePulmonary Arterial HypertensionMay 4United TherapeuticsNCT05776225
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraMay 4Swedish Orphan BiovitrumNCT03165734
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParoxysmal Nocturnal HemoglobinuriaMay 4Regeneron PharmaceuticalsNCT05133531
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisMay 3Swedish Orphan BiovitrumNCT03165734
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 3Regeneron PharmaceuticalsNCT05133531
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 3University of VirginiaNCT04593082
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMixed Connective Tissue DiseaseMay 3National Cancer Institute (NCI)NCT04704661
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisMay 2Calluna Pharma ASNCT06736990
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseMay 2University of IowaNCT05707663
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseMay 2Skyhawk Therapeutics, Inc.NCT06873334
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomozygous Familial HypercholesterolemiaMay 2Novartis PharmaceuticalsNCT05682378
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeGeographic AtrophyMay 1ClinOmicsAINCT07298291
No longer enrolling new participants

No longer enrolling new participants

FDA NewsMay 1
FDA Permits Expanded Access for Investigational Pancreatic Cancer Drug

FDA is announcing that it issued a “safe to proceed” letter to Revolution Medicines, allowing the sponsor to initiate an expanded access treatment protocol (EAP) for its experimental pancreatic cancer drug, daraxonrasib.

Status ChangeGraves’ DiseaseMay 1The First Affiliated Hospital of Xiamen UniversityNCT07547930
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeGranulomatosis with PolyangiitisMay 1Imperial College LondonNCT06230354
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFabry DiseaseMay 1Universität MünsterNCT06095713
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedNoonan SyndromeMay 31Ascendis Pharma A/SNCT07221851
Added 1 new site: Sejong, South Korea

Added 1 new site: Sejong, South Korea

sites_addedNeurofibromatosis Type 1May 31University of Alabama at BirminghamNCT06188741
Added 2 new sites: Palo Alto, California, St Louis, Missouri

Added 2 new sites: Palo Alto, California, St Louis, Missouri

sites_addedMyelofibrosisMay 31Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedMyelofibrosisMay 31Incyte CorporationNCT07441694
Added 3 new sites: Montreal, Canada, Kashiwa Chiba, Japan, Toon, Ehime, Japan

Added 3 new sites: Montreal, Canada, Kashiwa Chiba, Japan, Toon, Ehime, Japan

Enrollment UpdateMyelodysplastic SyndromesMay 31Mayo ClinicNCT03017820
Target enrollment decreased from 127 to 99

Target enrollment decreased from 127 to 99

sites_addedMyasthenia GravisMay 31Novartis PharmaceuticalsNCT06744920
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedMyasthenia GravisMay 31AmgenNCT06987539
Added 2 new sites: Paris, France, Valencia, Spain

Added 2 new sites: Paris, France, Valencia, Spain

sites_addedMyasthenia GravisMay 31argenxNCT07284420
Added 1 new site: Bergamo, Italy

Added 1 new site: Bergamo, Italy

sites_addedMyasthenia GravisMay 31Novartis PharmaceuticalsNCT06517758
Added 7 new sites: Boston, Massachusetts, Detroit, Michigan, CABA, Argentina and 4 more

Added 7 new sites: Boston, Massachusetts, Detroit, Michigan, CABA, Argentina and 4 more

sites_addedMultiple System AtrophyMay 31Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Tel Aviv, Israel, Milan, Italy

Added 2 new sites: Tel Aviv, Israel, Milan, Italy

eligibility_changeMultiple SclerosisMay 31Hoffmann-La RocheNCT04586010
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 31SanofiNCT07325292
Added 8 new sites: Phoenix, Arizona, Northbrook, Illinois, Teaneck, New Jersey and 5 more

Added 8 new sites: Phoenix, Arizona, Northbrook, Illinois, Teaneck, New Jersey and 5 more

eligibility_changeMultiple SclerosisMay 31Hoffmann-La RocheNCT05119569
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 31AmgenNCT06700343
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_removedMultiple SclerosisMay 31Novartis PharmaceuticalsNCT05156281
Reduced from 225 to 223 sites

Reduced from 225 to 223 sites

sites_addedMultiple SclerosisMay 31Novartis PharmaceuticalsNCT07225504
Added 11 new sites: Chicago, Illinois, Rosario, Argentina, Murdoch, Australia and 8 more

Added 11 new sites: Chicago, Illinois, Rosario, Argentina, Murdoch, Australia and 8 more

sites_addedMultiple SclerosisMay 31Hoffmann-La RocheNCT06780150
Added 6 new sites: Arnsdorf, Germany, Berg, Germany, Frankfurt, Germany and 3 more

Added 6 new sites: Arnsdorf, Germany, Berg, Germany, Frankfurt, Germany and 3 more

sites_addedMultiple SclerosisMay 31Zenas BioPharma (USA), LLCNCT07299019
Added 3 new sites: Bradenton, Florida, Rochester Hills, Michigan, St Louis, Missouri

Added 3 new sites: Bradenton, Florida, Rochester Hills, Michigan, St Louis, Missouri

sites_addedMultiple SclerosisMay 31Novartis PharmaceuticalsNCT06846281
Added 2 new sites: Irvine, California, Hradec Králové, Czechia

Added 2 new sites: Irvine, California, Hradec Králové, Czechia

sites_addedMultiple SclerosisMay 31TG Therapeutics, Inc.NCT07211633
Added 3 new sites: Tulza, Bosnia and Herzegovina, Kragujevac, Serbia, Niš, Serbia

Added 3 new sites: Tulza, Bosnia and Herzegovina, Kragujevac, Serbia, Niš, Serbia

sites_addedMultiple SclerosisMay 31TG Therapeutics, Inc.NCT05877963
Added 1 new site: Savannah, Georgia

Added 1 new site: Savannah, Georgia

sites_addedMultiple SclerosisMay 31TG Therapeutics, Inc.NCT06433752
Added 3 new sites: Billings, Montana, Norman, Oklahoma, Ogden, Utah

Added 3 new sites: Billings, Montana, Norman, Oklahoma, Ogden, Utah

sites_addedMultiple SclerosisMay 31Zenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Pheonix, Arizona, West Hollywood, California

Added 2 new sites: Pheonix, Arizona, West Hollywood, California

sites_addedMesotheliomaMay 31AstraZenecaNCT06535607
Added 31 new sites: New York, New York, Philadelphia, Pennsylvania, Clayton, Australia and 28 more

Added 31 new sites: New York, New York, Philadelphia, Pennsylvania, Clayton, Australia and 28 more

Enrollment UpdateMesotheliomaMay 31GenmabNCT05579366
Target enrollment increased from 764 to 884

Target enrollment increased from 764 to 884

eligibility_changeMesotheliomaMay 31GenmabNCT05579366
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaMay 31Merck Sharp & Dohme LLCNCT07302347
Added 1 new site: Chūō, Japan

Added 1 new site: Chūō, Japan

sites_addedMedullary Thyroid CancerMay 31University of Southern CaliforniaNCT07259304
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedLambert-Eaton Myasthenic SyndromeMay 30argenxNCT06078553
Added 3 new sites: Boston, Massachusetts, Columbus, Ohio, Philadelphia, Pennsylvania

Added 3 new sites: Boston, Massachusetts, Columbus, Ohio, Philadelphia, Pennsylvania

sites_addedIgA NephropathyMay 30BiogenNCT06935357
Added 4 new sites: Los Angeles, California, New York, New York, Shanghai, China and 1 more

Added 4 new sites: Los Angeles, California, New York, New York, Shanghai, China and 1 more

sites_addedIgA NephropathyMay 30TakedaNCT06963827
Added 5 new sites: Jacksonville, Florida, Cincinnati, Ohio, Arlington, Virginia and 2 more

Added 5 new sites: Jacksonville, Florida, Cincinnati, Ohio, Arlington, Virginia and 2 more

sites_addedIgA NephropathyMay 30Novartis PharmaceuticalsNCT07146906
Added 2 new sites: Opole, Poland, Seoul, South Korea

Added 2 new sites: Opole, Poland, Seoul, South Korea

eligibility_changeIgA NephropathyMay 30Novartis PharmaceuticalsNCT07146906
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyMay 30Haisco Pharmaceutical Group Co., Ltd.NCT07390123
Added 8 new sites: Huangshan City, China, Wuhu, China, Zhanjiang, China and 5 more

Added 8 new sites: Huangshan City, China, Wuhu, China, Zhanjiang, China and 5 more

sites_addedHuntington DiseaseMay 30Hoffmann-La RocheNCT07246941
Added 1 new site: Parkville, Australia

Added 1 new site: Parkville, Australia

sites_addedHuntington DiseaseMay 30Novartis PharmaceuticalsNCT07326709
Added 2 new sites: Georgetown, Texas, Southampton, United Kingdom

Added 2 new sites: Georgetown, Texas, Southampton, United Kingdom

sites_addedHomozygous Familial HypercholesterolemiaMay 30Arrowhead PharmaceuticalsNCT07037771
Added 7 new sites: Innsbruck, Austria, Linz, Austria, Vienna, Austria and 4 more

Added 7 new sites: Innsbruck, Austria, Linz, Austria, Vienna, Austria and 4 more

sites_addedHomocystinuriaMay 30Travere Therapeutics, Inc.NCT06431893
Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

eligibility_changeHomocystinuriaMay 30Travere Therapeutics, Inc.NCT06431893
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaMay 30Memorial Sloan Kettering Cancer CenterNCT07001384
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 30Merck Sharp & Dohme LLCNCT05139017
Added 8 new sites: Horsham, Pennsylvania, Knoxville, Tennessee, Aarhus, Denmark and 5 more

Added 8 new sites: Horsham, Pennsylvania, Knoxville, Tennessee, Aarhus, Denmark and 5 more

sites_removedHodgkin LymphomaMay 30Karyopharm Therapeutics IncNCT04442022
Reduced from 57 to 38 sites

Reduced from 57 to 38 sites

sites_addedHodgkin LymphomaMay 30American Society of Clinical OncologyNCT02693535
Added 7 new sites: Cayey, Puerto Rico, Dorado, Puerto Rico, Manati, Puerto Rico and 4 more

Added 7 new sites: Cayey, Puerto Rico, Dorado, Puerto Rico, Manati, Puerto Rico and 4 more

eligibility_changeHodgkin LymphomaMay 30Children's Oncology GroupNCT02981628
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 30University of Southern CaliforniaNCT07259304
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedHodgkin LymphomaMay 30BeOne MedicinesNCT06742996
Added 6 new sites: Tucson, Arizona, Irvine, California, Nashville, Tennessee and 3 more

Added 6 new sites: Tucson, Arizona, Irvine, California, Nashville, Tennessee and 3 more

Enrollment UpdateHodgkin LymphomaMay 30AbbVieNCT05618028
Target enrollment decreased from 150 to 78

Target enrollment decreased from 150 to 78

sites_addedHodgkin LymphomaMay 30National Cancer Institute (NCI)NCT05886036
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_removedHodgkin LymphomaMay 30Shanghai Unicar-Therapy Bio-medicine Technology Co.,LtdNCT07093073
Reduced from 3 to 1 sites

Reduced from 3 to 1 sites

sites_addedHodgkin LymphomaMay 30National Cancer Institute (NCI)NCT05675410
Added 4 new sites: Los Angeles, California, Jacksonville, Florida, Miami Beach, Florida and 1 more

Added 4 new sites: Los Angeles, California, Jacksonville, Florida, Miami Beach, Florida and 1 more

sites_removedHodgkin LymphomaMay 30Hoffmann-La RocheNCT04077723
Reduced from 44 to 35 sites

Reduced from 44 to 35 sites

eligibility_changeHodgkin LymphomaMay 30Umoja BiopharmaNCT06528301
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 30Hoffmann-La RocheNCT06806033
Added 1 new site: Grand Rapids, Michigan

Added 1 new site: Grand Rapids, Michigan

eligibility_changeHodgkin LymphomaMay 30Mayo ClinicNCT07285044
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 30AbbVieNCT07387471
Added 10 new sites: Nagoya, Japan, Fukuoka, Japan, Hiroshima, Japan and 7 more

Added 10 new sites: Nagoya, Japan, Fukuoka, Japan, Hiroshima, Japan and 7 more

sites_addedHodgkin LymphomaMay 30Merck Sharp & Dohme LLCNCT07570173
Added 2 new sites: Haifa, Israel, Ramat Gan, Israel

Added 2 new sites: Haifa, Israel, Ramat Gan, Israel

eligibility_changeHodgkin LymphomaMay 30Memorial Sloan Kettering Cancer CenterNCT07223021
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaMay 30M.D. Anderson Cancer CenterNCT07153796
Target enrollment decreased from 60 to 3

Target enrollment decreased from 60 to 3

sites_removedHodgkin LymphomaMay 30Regeneron PharmaceuticalsNCT06230224
Reduced from 101 to 81 sites

Reduced from 101 to 81 sites

sites_addedHereditary AngioedemaMay 30Ionis Pharmaceuticals, Inc.NCT07298447
Added 2 new sites: Padova, Italy, San Donato Milanese, Italy

Added 2 new sites: Padova, Italy, San Donato Milanese, Italy

eligibility_changeGiant Cell ArteritisMay 29University of AarhusNCT07552636
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFacioscapulohumeral Muscular DystrophyMay 29Avidity Biosciences, Inc.NCT07038200
Added 24 new sites: Aarhus, Denmark, Copenhagen, Denmark, Marseille, France and 21 more

Added 24 new sites: Aarhus, Denmark, Copenhagen, Denmark, Marseille, France and 21 more

sites_addedFocal Segmental GlomerulosclerosisMay 29Boehringer IngelheimNCT07220083
Added 15 new sites: Fortaleza, Brazil, Porto Alegre, Brazil, São Paulo, Brazil and 12 more

Added 15 new sites: Fortaleza, Brazil, Porto Alegre, Brazil, São Paulo, Brazil and 12 more

sites_addedEssential ThrombocythemiaMay 29Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedEssential ThrombocythemiaMay 29Incyte CorporationNCT07441694
Added 3 new sites: Montreal, Canada, Kashiwa Chiba, Japan, Toon, Ehime, Japan

Added 3 new sites: Montreal, Canada, Kashiwa Chiba, Japan, Toon, Ehime, Japan

sites_removedHemophilia BMay 29CSL BehringNCT07080905
Reduced from 11 to 2 sites

Reduced from 11 to 2 sites

eligibility_changeHemophilia BMay 29CSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BMay 29SanofiNCT07285460
Added 3 new sites: Bengaluru, India, Vellore, India, Riyadh, Saudi Arabia

Added 3 new sites: Bengaluru, India, Vellore, India, Riyadh, Saudi Arabia

sites_addedHemophilia BMay 29PfizerNCT05568719
Added 1 new site: Taichung, Taiwan

Added 1 new site: Taichung, Taiwan

sites_addedHemophilia AMay 29SanofiNCT07285460
Added 3 new sites: Bengaluru, India, Vellore, India, Riyadh, Saudi Arabia

Added 3 new sites: Bengaluru, India, Vellore, India, Riyadh, Saudi Arabia

sites_addedHemophilia AMay 29PfizerNCT05568719
Added 1 new site: Taichung, Taiwan

Added 1 new site: Taichung, Taiwan

sites_addedGuillain-Barré SyndromeMay 29Annexon, Inc.NCT07020819
Added 4 new sites: Phoenix, Arizona, Carlsbad, California, Jacksonville, Florida and 1 more

Added 4 new sites: Phoenix, Arizona, Carlsbad, California, Jacksonville, Florida and 1 more

sites_addedGraves’ DiseaseMay 29Merida BiosciencesNCT07305818
Added 1 new site: Wesley Chapel, Florida

Added 1 new site: Wesley Chapel, Florida

sites_addedGeographic AtrophyMay 29Regeneron PharmaceuticalsNCT07230834
Added 2 new sites: Bucharest, Romania, Cluj-Napoca, Romania

Added 2 new sites: Bucharest, Romania, Cluj-Napoca, Romania

sites_addedGeographic AtrophyMay 29Novartis PharmaceuticalsNCT07441642
Added 3 new sites: Pécs, Hungary, Bydgoszcz, Poland, Krakow, Poland

Added 3 new sites: Pécs, Hungary, Bydgoszcz, Poland, Krakow, Poland

sites_addedGeographic AtrophyMay 29Complement TherapeuticsNCT07144137
Added 2 new sites: London, United Kingdom, Southampton, United Kingdom

Added 2 new sites: London, United Kingdom, Southampton, United Kingdom

eligibility_changeGastrointestinal Stromal TumorMay 29University of California, San DiegoNCT07171203
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGastrointestinal Stromal TumorMay 29GlaxoSmithKlineNCT07218926
Added 33 new sites: La Jolla, California, Los Angeles, California, San Francisco, California and 30 more

Added 33 new sites: La Jolla, California, Los Angeles, California, San Francisco, California and 30 more

Enrollment UpdateFragile X SyndromeMay 29Zynerba Pharmaceuticals, Inc.NCT03802799
Target enrollment decreased from 550 to 476

Target enrollment decreased from 550 to 476

eligibility_changeFocal EpilepsyMay 29National Institute of Neurological Disorders and Stroke (NINDS)NCT03478852
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyMay 29Rapport Therapeutics Inc.NCT07563881
Added 1 new site: Fort Wayne, Indiana

Added 1 new site: Fort Wayne, Indiana

sites_addedDravet SyndromeMay 28Encoded TherapeuticsNCT05419492
Added 1 new site: Ann Arbor, Michigan

Added 1 new site: Ann Arbor, Michigan

sites_addedDermatomyositisMay 28AstraZenecaNCT06455449
Added 1 new site: Amarillo, Texas

Added 1 new site: Amarillo, Texas

sites_addedDermatomyositisMay 28argenxNCT05979441
Added 1 new site: Nanjing, China

Added 1 new site: Nanjing, China

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28argenxNCT07091630
Added 13 new sites: Linz, Austria, Sofia, Bulgaria, Fuzhou, China and 10 more

Added 13 new sites: Linz, Austria, Sofia, Bulgaria, Fuzhou, China and 10 more

sites_addedChronic Graft-versus-Host DiseaseMay 28Blueprint Medicines CorporationNCT07143669
Added 1 new site: Kansas City, Kansas

Added 1 new site: Kansas City, Kansas

sites_addedChronic Graft-versus-Host DiseaseMay 28London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph'sNCT06032208
Added 2 new sites: Winnipeg, Canada, Halifax, Canada

Added 2 new sites: Winnipeg, Canada, Halifax, Canada

sites_addedChronic Graft-versus-Host DiseaseMay 28Zenas BioPharma (USA), LLCNCT07067463
Added 7 new sites: Scottsdale, Arizona, Bradenton, Florida, St Louis, Missouri and 4 more

Added 7 new sites: Scottsdale, Arizona, Bradenton, Florida, St Louis, Missouri and 4 more

eligibility_changeEosinophilic EsophagitisMay 28National Institute of Allergy and Infectious Diseases (NIAID)NCT02504853
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 28Encoded TherapeuticsNCT05419492
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_addedDermatomyositisMay 28argenxNCT05979441
Added 1 new site: Baltimore, Maryland

Added 1 new site: Baltimore, Maryland

sites_addedDermatomyositisMay 28AstraZenecaNCT06916806
Added 2 new sites: Omaha, Nebraska, Pok Fu Lam, Hong Kong

Added 2 new sites: Omaha, Nebraska, Pok Fu Lam, Hong Kong

sites_removedCystic FibrosisMay 28Vertex Pharmaceuticals IncorporatedNCT05668741
Reduced from 43 to 26 sites

Reduced from 43 to 26 sites

Enrollment UpdateCystic FibrosisMay 28Vertex Pharmaceuticals IncorporatedNCT05668741
Target enrollment decreased from 39 to 26

Target enrollment decreased from 39 to 26

Enrollment UpdateCystic FibrosisMay 28Vertex Pharmaceuticals IncorporatedNCT06154447
Target enrollment decreased from 255 to 165

Target enrollment decreased from 255 to 165

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28SanofiNCT06290128
Added 5 new sites: Centennial, Colorado, Bradenton, Florida, Kansas City, Kansas and 2 more

Added 5 new sites: Centennial, Colorado, Bradenton, Florida, Kansas City, Kansas and 2 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28SanofiNCT06859099
Added 2 new sites: Kansas City, Kansas, Philadelphia, Pennsylvania

Added 2 new sites: Kansas City, Kansas, Philadelphia, Pennsylvania

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28SanofiNCT06290141
Added 1 new site: Budapest, Hungary

Added 1 new site: Budapest, Hungary

sites_addedChronic Graft-versus-Host DiseaseMay 28Saluda Medical Pty LtdNCT04662905
Added 2 new sites: Robina, Australia, Doncaster East, Australia

Added 2 new sites: Robina, Australia, Doncaster East, Australia

sites_addedChronic Graft-versus-Host DiseaseMay 28BeOne MedicinesNCT07277231
Added 23 new sites: Blacktown, Australia, Malvern, Australia, Brasília, Brazil and 20 more

Added 23 new sites: Blacktown, Australia, Malvern, Australia, Brasília, Brazil and 20 more

Enrollment UpdateChronic Graft-versus-Host DiseaseMay 28Arthrosi TherapeuticsNCT07116746
Target enrollment decreased from 25 to 15

Target enrollment decreased from 25 to 15

eligibility_changeChronic Graft-versus-Host DiseaseMay 28National Taiwan University HospitalNCT07034521
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseMay 28Massachusetts General HospitalNCT05747534
Target enrollment decreased from 150 to 107

Target enrollment decreased from 150 to 107

sites_removedCholangiocarcinomaMay 28Eli Lilly and CompanyNCT06607185
Reduced from 56 to 47 sites

Reduced from 56 to 47 sites

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 27Novo Nordisk A/SNCT07207811
Added 4 new sites: Cleveland, Ohio, Adelaide, Australia, Roeselare, Belgium and 1 more

Added 4 new sites: Cleveland, Ohio, Adelaide, Australia, Roeselare, Belgium and 1 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 27AstraZenecaNCT06465810
Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

Enrollment UpdateAplastic AnemiaMay 27Fred Hutchinson Cancer CenterNCT06752694
Target enrollment decreased from 20 to 1

Target enrollment decreased from 20 to 1

sites_addedAmyotrophic Lateral SclerosisMay 27Nura BioNCT07369076
Added 2 new sites: Brisbane, Australia, Fredericton, Canada

Added 2 new sites: Brisbane, Australia, Fredericton, Canada

sites_addedAdrenocortical CarcinomaMay 27National Cancer Institute (NCI)NCT06900595
Added 1 new site: Kansas City, Missouri

Added 1 new site: Kansas City, Missouri

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 26AstraZenecaNCT06465810
Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

sites_addedWaldenström MacroglobulinemiaMay 26National Cancer Institute (NCI)NCT04840602
Added 2 new sites: Saint Louis Park, Minnesota, Appleton, Wisconsin

Added 2 new sites: Saint Louis Park, Minnesota, Appleton, Wisconsin

sites_addedSystemic Juvenile Idiopathic ArthritisMay 26AbbVieNCT05609630
Added 14 new sites: Little Rock, Arkansas, Hollywood, Florida, Columbus, Ohio and 11 more

Added 14 new sites: Little Rock, Arkansas, Hollywood, Florida, Columbus, Ohio and 11 more

sites_addedStickler SyndromeMay 26Helen Keller Eye Research FoundationNCT07146516
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

eligibility_changeStickler SyndromeMay 26Helen Keller Eye Research FoundationNCT07146516
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedWaldenström MacroglobulinemiaMay 26National Cancer Institute (NCI)NCT04840602
Added 1 new site: Nampa, Idaho

Added 1 new site: Nampa, Idaho

Enrollment UpdateWaldenström MacroglobulinemiaMay 26Shayna Sarosiek, MDNCT05190705
Target enrollment decreased from 36 to 21

Target enrollment decreased from 36 to 21

sites_addedTurner SyndromeMay 26Ascendis Pharma A/SNCT07221851
Added 1 new site: Hanover, Germany

Added 1 new site: Hanover, Germany

sites_addedSystemic MastocytosisMay 26Blueprint Medicines CorporationNCT04910685
Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

sites_addedSystemic Juvenile Idiopathic ArthritisMay 26SanofiNCT02991469
Added 1 new site: Lisbon, Portugal

Added 1 new site: Lisbon, Portugal

eligibility_changeStargardt DiseaseMay 26National Eye Institute (NEI)NCT01145196
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedSpinal Muscular AtrophyMay 26BiogenNCT05067790
Reduced from 44 to 22 sites

Reduced from 44 to 22 sites

sites_addedSpinal Muscular AtrophyMay 26Massachusetts General HospitalNCT05966038
Added 2 new sites: Grand Rapids, Michigan, Philadelphia, Pennsylvania

Added 2 new sites: Grand Rapids, Michigan, Philadelphia, Pennsylvania

sites_addedSjögren's SyndromeMay 26Artiva Biotherapeutics, Inc.NCT06991114
Added 27 new sites: Tucson, Arizona, Chula Vista, California, Los Angeles, California and 24 more

Added 27 new sites: Tucson, Arizona, Chula Vista, California, Los Angeles, California and 24 more

sites_addedSickle Cell DiseaseMay 26Disc Medicine, IncNCT07187973
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

Enrollment UpdateRetinitis PigmentosaMay 25Octant, Inc.NCT07408232
Target enrollment decreased from 50 to 32

Target enrollment decreased from 50 to 32

Enrollment UpdatePulmonary Arterial HypertensionMay 25OrphAI TherapeuticsNCT05798923
Target enrollment increased from 10 to 85

Target enrollment increased from 10 to 85

eligibility_changePulmonary Arterial HypertensionMay 25OrphAI TherapeuticsNCT05798923
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 25PfizerNCT06137742
Added 1 new site: Edmonton, Canada

Added 1 new site: Edmonton, Canada

eligibility_changePulmonary Arterial HypertensionMay 25Gradient Denervation TechnologiesNCT06052072
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 25Merck Sharp & Dohme LLCNCT07218029
Added 5 new sites: Orange, California, Dallas, Texas, Blumenau, Brazil and 2 more

Added 5 new sites: Orange, California, Dallas, Texas, Blumenau, Brazil and 2 more

sites_addedPulmonary Arterial HypertensionMay 25PfizerNCT07073820
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedProgressive Supranuclear PalsyMay 25Novartis PharmaceuticalsNCT07498426
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedPrader-Willi SyndromeMay 25Soleno Therapeutics, Inc.NCT07450664
Added 4 new sites: Chicago, Illinois, Ann Arbor, Michigan, Chapel Hill, North Carolina and 1 more

Added 4 new sites: Chicago, Illinois, Ann Arbor, Michigan, Chapel Hill, North Carolina and 1 more

Enrollment UpdateParoxysmal Nocturnal HemoglobinuriaMay 25Novartis PharmaceuticalsNCT06606314
Target enrollment increased from 100 to 132

Target enrollment increased from 100 to 132

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 25AstraZenecaNCT07457151
Expanded from 4 to 5 sites

Expanded from 4 to 5 sites

sites_addedNoonan SyndromeMay 24Ascendis Pharma A/SNCT07221851
Added 3 new sites: Le Kremlin-Bicêtre, France, Hanover, Germany, Seoul, South Korea

Added 3 new sites: Le Kremlin-Bicêtre, France, Hanover, Germany, Seoul, South Korea

Enrollment UpdateNeuromyelitis OpticaMay 24TG Therapeutics, Inc.NCT06680037
Target enrollment increased from 32 to 100

Target enrollment increased from 32 to 100

eligibility_changeNeuromyelitis OpticaMay 24TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNeurofibromatosis Type 1May 24Healx LimitedNCT06541847
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

sites_addedMyelodysplastic SyndromesMay 24Blueprint Medicines CorporationNCT07143669
Added 1 new site: Kansas City, Kansas

Added 1 new site: Kansas City, Kansas

Enrollment UpdateMyelodysplastic SyndromesMay 24Novartis PharmaceuticalsNCT06606314
Target enrollment increased from 100 to 132

Target enrollment increased from 100 to 132

sites_addedMyelodysplastic SyndromesMay 24AstraZenecaNCT07457151
Expanded from 4 to 5 sites

Expanded from 4 to 5 sites

eligibility_changeMyelodysplastic SyndromesMay 24Mayo ClinicNCT07285044
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesMay 24City of Hope Medical CenterNCT06287944
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesMay 24Novartis PharmaceuticalsNCT07229235
Added 2 new sites: Milan, Italy, Torino, Italy

Added 2 new sites: Milan, Italy, Torino, Italy

sites_addedMyasthenia GravisMay 24UCB Biopharma SRLNCT07246564
Added 1 new site: Changchun, China

Added 1 new site: Changchun, China

sites_addedMyasthenia GravisMay 24Vertex Pharmaceuticals IncorporatedNCT07501702
Added 3 new sites: Boca Raton, Florida, Houston, Texas, Waxahachie, Texas

Added 3 new sites: Boca Raton, Florida, Houston, Texas, Waxahachie, Texas

sites_addedMyasthenia GravisMay 24Immunovant Sciences GmbHNCT07039916
Added 2 new sites: Huntsville, Alabama, Rosario, Argentina

Added 2 new sites: Huntsville, Alabama, Rosario, Argentina

sites_addedMyasthenia GravisMay 24Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 5 new sites: Farmington, Connecticut, Cincinnati, Ohio, Camperdown, Australia and 2 more

Added 5 new sites: Farmington, Connecticut, Cincinnati, Ohio, Camperdown, Australia and 2 more

eligibility_changeMyasthenia GravisMay 24Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyasthenia GravisMay 24Novartis PharmaceuticalsNCT06744920
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMyasthenia GravisMay 24TG Therapeutics, Inc.NCT06680037
Target enrollment increased from 32 to 100

Target enrollment increased from 32 to 100

eligibility_changeMyasthenia GravisMay 24TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple System AtrophyMay 24Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: La Jolla, California, Roma, Italy

Added 2 new sites: La Jolla, California, Roma, Italy

sites_addedMultiple SclerosisMay 24Novartis PharmaceuticalsNCT06655896
Added 6 new sites: Ghent, Belgium, Salvador, Brazil, Barretos, Brazil and 3 more

Added 6 new sites: Ghent, Belgium, Salvador, Brazil, Barretos, Brazil and 3 more

sites_addedMultiple SclerosisMay 24Hoffmann-La RocheNCT07282574
Added 26 new sites: Heidelberg, Australia, Nîmes, France, Saint-Herblain, France and 23 more

Added 26 new sites: Heidelberg, Australia, Nîmes, France, Saint-Herblain, France and 23 more

sites_addedMultiple SclerosisMay 24Novartis PharmaceuticalsNCT07225504
Added 5 new sites: Hradec Králové, Czechia, Tallinn, Estonia, 's-Hertogenbosch, Netherlands and 2 more

Added 5 new sites: Hradec Králové, Czechia, Tallinn, Estonia, 's-Hertogenbosch, Netherlands and 2 more

Enrollment UpdateMultiple SclerosisMay 24Daishi TianNCT07303790
Target enrollment decreased from 15 to 9

Target enrollment decreased from 15 to 9

eligibility_changeMultiple SclerosisMay 24Daishi TianNCT07303790
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 24Novartis PharmaceuticalsNCT06869785
Added 3 new sites: Miami, Florida, Weston, Florida, Columbus, Ohio

Added 3 new sites: Miami, Florida, Weston, Florida, Columbus, Ohio

sites_addedMultiple SclerosisMay 24SetPoint Medical CorporationNCT06796504
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedMultiple SclerosisMay 24Zenas BioPharma (USA), LLCNCT07067463
Added 7 new sites: Scottsdale, Arizona, Bradenton, Florida, St Louis, Missouri and 4 more

Added 7 new sites: Scottsdale, Arizona, Bradenton, Florida, St Louis, Missouri and 4 more

Enrollment UpdateMultiple SclerosisMay 24TG Therapeutics, Inc.NCT06680037
Target enrollment increased from 32 to 100

Target enrollment increased from 32 to 100

eligibility_changeMultiple SclerosisMay 24TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaMay 24A2 Biotherapeutics Inc.NCT06885424
Added 1 new site: Gilbert, Arizona

Added 1 new site: Gilbert, Arizona

Enrollment UpdateMesotheliomaMay 24National Cancer Institute (NCI)NCT04431024
Target enrollment decreased from 800 to 300

Target enrollment decreased from 800 to 300

eligibility_changeMesotheliomaMay 24National Cancer Institute (NCI)NCT04431024
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaMay 24Mayo ClinicNCT07285044
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaMay 24SystImmune Inc.NCT07080242
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyMay 24BiogenNCT06962800
Added 4 new sites: Stanford, California, Nashville, Tennessee, Lucknow, India and 1 more

Added 4 new sites: Stanford, California, Nashville, Tennessee, Lucknow, India and 1 more

sites_addedMembranous NephropathyMay 24Nkarta, Inc.NCT06557265
Added 1 new site: Parkville, Australia

Added 1 new site: Parkville, Australia

Enrollment UpdateMembranous NephropathyMay 24Nkarta, Inc.NCT06557265
Target enrollment increased from 96 to 120

Target enrollment increased from 96 to 120

eligibility_changeMembranous NephropathyMay 24Nkarta, Inc.NCT06557265
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMastocytosisMay 24Blueprint Medicines CorporationNCT07143669
Added 1 new site: Kansas City, Kansas

Added 1 new site: Kansas City, Kansas

sites_addedMastocytosisMay 24Blueprint Medicines CorporationNCT04910685
Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

Enrollment UpdateLight Chain AmyloidosisMay 23Alnylam PharmaceuticalsNCT06360289
Target enrollment decreased from 500 to 346

Target enrollment decreased from 500 to 346

sites_addedLennox-Gastaut SyndromeMay 23Longboard PharmaceuticalsNCT06719141
Added 10 new sites: Edegem, Belgium, Porto Alegre, Brazil, Florence, Italy and 7 more

Added 10 new sites: Edegem, Belgium, Porto Alegre, Brazil, Florence, Italy and 7 more

sites_addedIgG4-Related DiseaseMay 23SanofiNCT07190196
Added 8 new sites: Fontana, California, Miami, Florida, Atlanta, Georgia and 5 more

Added 8 new sites: Fontana, California, Miami, Florida, Atlanta, Georgia and 5 more

sites_addedIgA NephropathyMay 23TakedaNCT06963827
Added 4 new sites: Melbourne, Florida, Rockford, Illinois, Media, Pennsylvania and 1 more

Added 4 new sites: Melbourne, Florida, Rockford, Illinois, Media, Pennsylvania and 1 more

sites_addedIgA NephropathyMay 23BiogenNCT06935357
Added 2 new sites: Hong Kong, China, Sheffield, United Kingdom

Added 2 new sites: Hong Kong, China, Sheffield, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisMay 23Contineum TherapeuticsNCT07284459
Added 9 new sites: Midland, Australia, Spearwood, Australia, Trois-Rivières, Canada and 6 more

Added 9 new sites: Midland, Australia, Spearwood, Australia, Trois-Rivières, Canada and 6 more

eligibility_changeHypoparathyroidismMay 23Peking Union Medical College HospitalNCT07197450
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseMay 23Novartis PharmaceuticalsNCT07326709
Added 2 new sites: Philadelphia, Pennsylvania, Montreal, Canada

Added 2 new sites: Philadelphia, Pennsylvania, Montreal, Canada

eligibility_changeHomozygous Familial HypercholesterolemiaMay 23Eddingpharm (Zhuhai) Co., Ltd.NCT07489209
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHomozygous Familial HypercholesterolemiaMay 23Novartis PharmaceuticalsNCT06275724
Added 1 new site: Yamagata, Japan

Added 1 new site: Yamagata, Japan

sites_addedHodgkin LymphomaMay 23Janssen Research & Development, LLCNCT01804686
Added 2 new sites: Melbourne, Australia, Warsaw, Poland

Added 2 new sites: Melbourne, Australia, Warsaw, Poland

eligibility_changeHodgkin LymphomaMay 23New York Medical CollegeNCT07013565
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 23Reid Merryman, MDNCT05783609
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedHodgkin LymphomaMay 23Allogene TherapeuticsNCT06500273
Added 4 new sites: Auchenflower, Australia, Fitzroy, Australia, Heidelberg, Australia and 1 more

Added 4 new sites: Auchenflower, Australia, Fitzroy, Australia, Heidelberg, Australia and 1 more

sites_addedHodgkin LymphomaMay 23Alliance for Clinical Trials in OncologyNCT05976763
Added 5 new sites: Los Angeles, California, Albany, Georgia, Crown Point, Indiana and 2 more

Added 5 new sites: Los Angeles, California, Albany, Georgia, Crown Point, Indiana and 2 more

eligibility_changeHodgkin LymphomaMay 23Dipenkumar ModiNCT05852717
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 23Regeneron PharmaceuticalsNCT06091865
Added 7 new sites: Baltimore, Maryland, Greenville, North Carolina, Dublin, Ireland and 4 more

Added 7 new sites: Baltimore, Maryland, Greenville, North Carolina, Dublin, Ireland and 4 more

sites_addedHodgkin LymphomaMay 23National Cancer Institute (NCI)NCT07003295
Added 1 new site: Pittsburgh, Pennsylvania

Added 1 new site: Pittsburgh, Pennsylvania

sites_addedHodgkin LymphomaMay 23National Cancer Institute (NCI)NCT06317662
Added 1 new site: Fargo, North Dakota

Added 1 new site: Fargo, North Dakota

sites_addedHodgkin LymphomaMay 23Merck Sharp & Dohme LLCNCT06890884
Added 3 new sites: Goodyear, Arizona, Mons, Belgium, Stuttgart, Germany

Added 3 new sites: Goodyear, Arizona, Mons, Belgium, Stuttgart, Germany

eligibility_changeHodgkin LymphomaMay 23Fred Hutchinson Cancer CenterNCT07225985
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 23National Cancer Institute (NCI)NCT04840602
Added 1 new site: Nampa, Idaho

Added 1 new site: Nampa, Idaho

sites_addedHodgkin LymphomaMay 23AstraZenecaNCT06549595
Added 32 new sites: Huntsville, Alabama, Newark, Delaware, Evanston, Illinois and 29 more

Added 32 new sites: Huntsville, Alabama, Newark, Delaware, Evanston, Illinois and 29 more

sites_addedHereditary AngioedemaMay 23ADARx Pharmaceuticals, Inc.NCT06960213
Added 1 new site: Krakow, Poland

Added 1 new site: Krakow, Poland

sites_addedHereditary AngioedemaMay 23Astria Therapeutics, Inc.NCT07204938
Added 9 new sites: Little Rock, Arkansas, San Diego, California, Santa Monica, California and 6 more

Added 9 new sites: Little Rock, Arkansas, San Diego, California, Santa Monica, California and 6 more

sites_addedHereditary AngioedemaMay 23Ionis Pharmaceuticals, Inc.NCT07298447
Added 2 new sites: Miami, Florida, Hershey, Pennsylvania

Added 2 new sites: Miami, Florida, Hershey, Pennsylvania

sites_addedGraves’ DiseaseMay 22Immunovant Sciences GmbHNCT06727604
Added 1 new site: Cambridge, United Kingdom

Added 1 new site: Cambridge, United Kingdom

sites_addedGraves’ DiseaseMay 22Immunovant Sciences GmbHNCT07018323
Added 10 new sites: Fortaleza, Brazil, Brasília, Brazil, Curitiba, Brazil and 7 more

Added 10 new sites: Fortaleza, Brazil, Brasília, Brazil, Curitiba, Brazil and 7 more

sites_addedGaucher DiseaseMay 22Prevail TherapeuticsNCT05487599
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

eligibility_changeGastrointestinal Stromal TumorMay 22Cogent Biosciences, Inc.NCT05208047
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeGastrointestinal Stromal TumorMay 22Affiliated Hospital to Academy of Military Medical SciencesNCT07581977
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedFibrodysplasia Ossificans ProgressivaMay 22Incyte CorporationNCT05090891
Reduced from 27 to 24 sites

Reduced from 27 to 24 sites

eligibility_changeFibrodysplasia Ossificans ProgressivaMay 22Incyte CorporationNCT05090891
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BMay 22Regeneron PharmaceuticalsNCT06379789
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedHemophilia BMay 22PfizerNCT05568719
Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

sites_addedHemophilia AMay 22Regeneron PharmaceuticalsNCT06379789
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedHemophilia AMay 22PfizerNCT05568719
Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

sites_addedHemophilia AMay 22TakedaNCT06550882
Added 3 new sites: Seoul, South Korea, Seoul, South Korea, Ulsan, South Korea

Added 3 new sites: Seoul, South Korea, Seoul, South Korea, Ulsan, South Korea

sites_addedHemophilia AMay 22PfizerNCT04370054
Added 10 new sites: Oakland, California, Redwood City, California, San Francisco, California and 7 more

Added 10 new sites: Oakland, California, Redwood City, California, San Francisco, California and 7 more

sites_addedGranulomatosis with PolyangiitisMay 22Novartis PharmaceuticalsNCT06868290
Added 5 new sites: Ann Arbor, Michigan, Minneapolis, Minnesota, Dallas, Texas and 2 more

Added 5 new sites: Ann Arbor, Michigan, Minneapolis, Minnesota, Dallas, Texas and 2 more

sites_addedGastrointestinal Stromal TumorMay 22Kumquat Biosciences Inc.NCT07406633
Added 7 new sites: Los Angeles, California, Jacksonville, Florida, Tampa, Florida and 4 more

Added 7 new sites: Los Angeles, California, Jacksonville, Florida, Tampa, Florida and 4 more

sites_addedEpidermolysis BullosaMay 22Xinnate ABNCT06594393
Added 3 new sites: Rome, Italy, Barcelona, Spain, Uppsala, Sweden

Added 3 new sites: Rome, Italy, Barcelona, Spain, Uppsala, Sweden

sites_addedEosinophilic EsophagitisMay 21Regeneron PharmaceuticalsNCT07112378
Added 2 new sites: Boston, Massachusetts, Philadelphia, Pennsylvania

Added 2 new sites: Boston, Massachusetts, Philadelphia, Pennsylvania

sites_removedDuchenne Muscular DystrophyMay 21Hoffmann-La RocheNCT06450639
Reduced from 22 to 19 sites

Reduced from 22 to 19 sites

eligibility_changeDuchenne Muscular DystrophyMay 21Hoffmann-La RocheNCT06450639
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 21Longboard PharmaceuticalsNCT06660394
Added 12 new sites: Washington D.C., District of Columbia, Edegem, Belgium, Porto Alegre, Brazil and 9 more

Added 12 new sites: Washington D.C., District of Columbia, Edegem, Belgium, Porto Alegre, Brazil and 9 more

sites_addedDravet SyndromeMay 21Ionis Pharmaceuticals, Inc.NCT07531745
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_removedDravet SyndromeMay 21University of Colorado, DenverNCT06598449
Reduced from 3 to 1 sites

Reduced from 3 to 1 sites

Enrollment UpdateDravet SyndromeMay 21University of Colorado, DenverNCT06598449
Target enrollment decreased from 12 to 5

Target enrollment decreased from 12 to 5

eligibility_changeDravet SyndromeMay 21University of Colorado, DenverNCT06598449
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 21Encoded TherapeuticsNCT05419492
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedDermatomyositisMay 21Novartis PharmaceuticalsNCT06665256
Added 13 new sites: Pittsburgh, Pennsylvania, Dallas, Texas, Houston, Texas and 10 more

Added 13 new sites: Pittsburgh, Pennsylvania, Dallas, Texas, Houston, Texas and 10 more

sites_addedDermatomyositisMay 21PfizerNCT06698796
Added 17 new sites: Allen, Texas, Beckley, West Virginia, SAN M. de Tucuman, Argentina and 14 more

Added 17 new sites: Allen, Texas, Beckley, West Virginia, SAN M. de Tucuman, Argentina and 14 more

sites_addedDermatomyositisMay 21PfizerNCT05895786
Added 79 new sites: Glendale, Arizona, Phoenix, Arizona, Scottsdale, Arizona and 76 more

Added 79 new sites: Glendale, Arizona, Phoenix, Arizona, Scottsdale, Arizona and 76 more

eligibility_changeDermatomyositisMay 21Novartis PharmaceuticalsNCT06665256
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCushing DiseaseMay 21Assistance Publique - Hôpitaux de ParisNCT04840693
Target enrollment decreased from 540 to 31

Target enrollment decreased from 540 to 31

sites_addedCongenital Adrenal HyperplasiaMay 21Crinetics Pharmaceuticals Inc.NCT07144163
Added 10 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Dallas, Texas and 7 more

Added 10 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Dallas, Texas and 7 more

Enrollment UpdateChronic Inflammatory Demyelinating PolyneuropathyMay 21TG Therapeutics, Inc.NCT06680037
Target enrollment increased from 32 to 100

Target enrollment increased from 32 to 100

eligibility_changeChronic Inflammatory Demyelinating PolyneuropathyMay 21TG Therapeutics, Inc.NCT06680037
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 21SanofiNCT06290141
Added 4 new sites: Hershey, Pennsylvania, Houston, Texas, Belo Horizonte, Brazil and 1 more

Added 4 new sites: Hershey, Pennsylvania, Houston, Texas, Belo Horizonte, Brazil and 1 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 21Grifols Therapeutics LLCNCT07540221
Added 3 new sites: Rancho Mirage, California, New York, New York, Sherman, Texas

Added 3 new sites: Rancho Mirage, California, New York, New York, Sherman, Texas

sites_addedChronic Graft-versus-Host DiseaseMay 21National Cancer Institute (NCI)NCT07061951
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedChronic Graft-versus-Host DiseaseMay 21BeOne MedicinesNCT05006716
Added 1 new site: Shenyang, China

Added 1 new site: Shenyang, China

Enrollment UpdateCholangiocarcinomaMay 21New Phase Ltd.NCT07224464
Target enrollment increased from 9 to 12

Target enrollment increased from 9 to 12

eligibility_changeCholangiocarcinomaMay 21New Phase Ltd.NCT07224464
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaMay 21UNICANCERNCT06529718
Added 38 new sites: Amiens, France, Angers, France, Avignon, France and 35 more

Added 38 new sites: Amiens, France, Angers, France, Avignon, France and 35 more

sites_addedCholangiocarcinomaMay 21Bold Therapeutics, Inc.NCT04421820
Added 6 new sites: Milan, Italy, Naples, Italy, Pisa, Italy and 3 more

Added 6 new sites: Milan, Italy, Naples, Italy, Pisa, Italy and 3 more

sites_addedCholangiocarcinomaMay 21Myeloid TherapeuticsNCT07334119
Added 1 new site: Darlinghurst, Australia

Added 1 new site: Darlinghurst, Australia

eligibility_changeCentral Diabetes InsipidusMay 21Massachusetts General HospitalNCT07568509
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateATTR Amyloidosis (Transthyretin Amyloidosis)May 20Alnylam PharmaceuticalsNCT06360289
Target enrollment decreased from 500 to 346

Target enrollment decreased from 500 to 346

sites_addedAmyotrophic Lateral SclerosisMay 20Massachusetts General HospitalNCT05966038
Added 2 new sites: Grand Rapids, Michigan, Philadelphia, Pennsylvania

Added 2 new sites: Grand Rapids, Michigan, Philadelphia, Pennsylvania

sites_removedBecker Muscular DystrophyMay 20Satellos Bioscience, Inc.NCT07287189
Reduced from 25 to 21 sites

Reduced from 25 to 21 sites

sites_removedBecker Muscular DystrophyMay 20Hoffmann-La RocheNCT06450639
Reduced from 22 to 19 sites

Reduced from 22 to 19 sites

eligibility_changeBecker Muscular DystrophyMay 20Hoffmann-La RocheNCT06450639
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 20Alnylam PharmaceuticalsNCT07223203
Added 11 new sites: Baltimore, Maryland, São Paulo, Brazil, Le Kremlin-Bicêtre, France and 8 more

Added 11 new sites: Baltimore, Maryland, São Paulo, Brazil, Le Kremlin-Bicêtre, France and 8 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 20Alnylam PharmaceuticalsNCT07052903
Added 15 new sites: Evanston, Illinois, Portland, Oregon, Charleston, South Carolina and 12 more

Added 15 new sites: Evanston, Illinois, Portland, Oregon, Charleston, South Carolina and 12 more

sites_removedAmyotrophic Lateral SclerosisMay 20Novartis PharmaceuticalsNCT06643481
Reduced from 86 to 74 sites

Reduced from 86 to 74 sites

eligibility_changeAmyotrophic Lateral SclerosisMay 20QurAlis CorporationNCT05633459
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAdrenocortical CarcinomaMay 20National Cancer Institute (NCI)NCT06900595
Added 1 new site: San Antonio, Texas

Added 1 new site: San Antonio, Texas

sites_addedWaldenström MacroglobulinemiaMay 19AbbVieNCT07387471
Added 5 new sites: Nagoya, Japan, Fukuoka, Japan, Koto-ku, Japan and 2 more

Added 5 new sites: Nagoya, Japan, Fukuoka, Japan, Koto-ku, Japan and 2 more

sites_addedTurner SyndromeMay 19Ascendis Pharma A/SNCT07221851
Added 2 new sites: Le Kremlin-Bicêtre, France, Seoul, South Korea

Added 2 new sites: Le Kremlin-Bicêtre, France, Seoul, South Korea

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 19Alnylam PharmaceuticalsNCT07052903
Added 15 new sites: Evanston, Illinois, Portland, Oregon, Charleston, South Carolina and 12 more

Added 15 new sites: Evanston, Illinois, Portland, Oregon, Charleston, South Carolina and 12 more

sites_addedWaldenström MacroglobulinemiaMay 19AbbVieNCT07387471
Added 4 new sites: Kyoto, Japan, Sakai-shi, Japan, Chuo-shi, Japan and 1 more

Added 4 new sites: Kyoto, Japan, Sakai-shi, Japan, Chuo-shi, Japan and 1 more

sites_addedStargardt DiseaseMay 19Astellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Southaven, Mississippi

Added 1 new site: Southaven, Mississippi

eligibility_changeSpinal Muscular AtrophyMay 19Columbia UniversityNCT06955897
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 19Novartis PharmaceuticalsNCT06546670
Added 1 new site: Providence, Rhode Island

Added 1 new site: Providence, Rhode Island

sites_addedSickle Cell DiseaseMay 19Novartis PharmaceuticalsNCT06439082
Added 1 new site: Orange, California

Added 1 new site: Orange, California

eligibility_changeSickle Cell DiseaseMay 19New York UniversityNCT06318143
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 19CSL BehringNCT06699849
Added 2 new sites: Louisville, Kentucky, Greenville, North Carolina

Added 2 new sites: Louisville, Kentucky, Greenville, North Carolina

eligibility_changeSevere Combined ImmunodeficiencyMay 18Fondazione TelethonNCT03478670
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePrimary Sclerosing CholangitisMay 18National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT04685200
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePrimary Biliary CholangitisMay 18National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT04685200
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 18Novartis PharmaceuticalsNCT07229235
Added 2 new sites: Milan, Italy, Torino, Italy

Added 2 new sites: Milan, Italy, Torino, Italy

eligibility_changeRetinitis PigmentosaMay 18SparingVisionNCT05748873
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePulmonary Arterial HypertensionMay 18VA Office of Research and DevelopmentNCT05937854
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 18PfizerNCT07073820
Added 25 new sites: San Francisco, California, Louisville, Kentucky, Louisville, Kentucky and 22 more

Added 25 new sites: San Francisco, California, Louisville, Kentucky, Louisville, Kentucky and 22 more

sites_addedPulmonary Arterial HypertensionMay 18PfizerNCT06137742
Added 36 new sites: Costa Mesa, California, Orange, California, Sacramento, California and 33 more

Added 36 new sites: Costa Mesa, California, Orange, California, Sacramento, California and 33 more

eligibility_changePulmonary Arterial HypertensionMay 18Nantes University HospitalNCT06735560
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 18Merck Sharp & Dohme LLCNCT07218029
Added 6 new sites: Houston, Texas, Calgary, Canada, Hanover, Germany and 3 more

Added 6 new sites: Houston, Texas, Calgary, Canada, Hanover, Germany and 3 more

sites_addedPulmonary Arterial HypertensionMay 18Apollo Therapeutics LtdNCT06846554
Added 1 new site: Cambridge, United Kingdom

Added 1 new site: Cambridge, United Kingdom

sites_addedProgressive Supranuclear PalsyMay 18Novartis PharmaceuticalsNCT07498426
Added 1 new site: Ulm, Germany

Added 1 new site: Ulm, Germany

sites_addedPrimary Biliary CholangitisMay 18Umecrine Cognition ABNCT07304843
Added 4 new sites: Balçova, Turkey (Türkiye), Rize, Turkey (Türkiye), Sanliurfa, Turkey (Türkiye) and 1 more

Added 4 new sites: Balçova, Turkey (Türkiye), Rize, Turkey (Türkiye), Sanliurfa, Turkey (Türkiye) and 1 more

Enrollment UpdatePrimary Biliary CholangitisMay 18Zydus Therapeutics Inc.NCT06427395
Target enrollment decreased from 150 to 102

Target enrollment decreased from 150 to 102

sites_addedPorphyria Cutanea TardaMay 18The American Porphyrias Expert CollaborativeNCT01561157
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedPolycythemia VeraMay 18Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

sites_addedPolycythemia VeraMay 18Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

sites_addedPolycythemia VeraMay 18Disc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedPolycythemia VeraMay 18Novartis PharmaceuticalsNCT07357727
Added 1 new site: Genolier, Switzerland

Added 1 new site: Genolier, Switzerland

sites_addedPolycythemia VeraMay 18National Cancer Institute (NCI)NCT06661915
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedPolycystic Kidney DiseaseMay 18Vertex Pharmaceuticals IncorporatedNCT07161037
Added 2 new sites: Nashville, Tennessee, Groningen, Netherlands

Added 2 new sites: Nashville, Tennessee, Groningen, Netherlands

sites_addedPolycystic Kidney DiseaseMay 18PYC TherapeuticsNCT06714006
Added 1 new site: Concord, Australia

Added 1 new site: Concord, Australia

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 18Hoffmann-La RocheNCT04654468
Added 1 new site: Guangzhou, China

Added 1 new site: Guangzhou, China

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 18Regeneron PharmaceuticalsNCT07154745
Added 3 new sites: Seoul, South Korea, Murcia, Spain, Salamanca, Spain

Added 3 new sites: Seoul, South Korea, Murcia, Spain, Salamanca, Spain

sites_addedNoonan SyndromeMay 17BioMarin PharmaceuticalNCT06668805
Added 12 new sites: Los Angeles, California, Atlanta, Georgia, Chicago, Illinois and 9 more

Added 12 new sites: Los Angeles, California, Atlanta, Georgia, Chicago, Illinois and 9 more

Enrollment UpdateNoonan SyndromeMay 17BioMarin PharmaceuticalNCT06668805
Target enrollment decreased from 54 to 30

Target enrollment decreased from 54 to 30

phase_changeNeuromyelitis OpticaMay 17Tianjin Medical University General HospitalNCT07420296
Moved from Phase 4 to Phase 3

Moved from Phase 4 to Phase 3

sites_addedMyotonic DystrophyMay 17Dyne TherapeuticsNCT05481879
Added 3 new sites: Stanford, California, Gainesville, Florida, St Louis, Missouri

Added 3 new sites: Stanford, California, Gainesville, Florida, St Louis, Missouri

sites_addedMyelofibrosisMay 17Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

sites_addedMyelofibrosisMay 17Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

sites_addedMyelofibrosisMay 17Disc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedMyelofibrosisMay 17Novartis PharmaceuticalsNCT07357727
Added 1 new site: Genolier, Switzerland

Added 1 new site: Genolier, Switzerland

sites_addedMyelofibrosisMay 17National Cancer Institute (NCI)NCT06661915
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_removedMyelodysplastic SyndromesMay 17Memorial Sloan Kettering Cancer CenterNCT07566377
Reduced from 7 to 1 sites

Reduced from 7 to 1 sites

eligibility_changeMyelodysplastic SyndromesMay 17GCP-Service International West GmbHNCT05583552
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesMay 17National Cancer Institute (NCI)NCT05564390
Added 2 new sites: Los Angeles, California, Stony Brook, New York

Added 2 new sites: Los Angeles, California, Stony Brook, New York

sites_addedMyelodysplastic SyndromesMay 17Disc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedMyelodysplastic SyndromesMay 17Hoffmann-La RocheNCT04654468
Added 1 new site: Guangzhou, China

Added 1 new site: Guangzhou, China

sites_addedMyelodysplastic SyndromesMay 17National Cancer Institute (NCI)NCT06577441
Added 1 new site: Stony Brook, New York

Added 1 new site: Stony Brook, New York

eligibility_changeMyelodysplastic SyndromesMay 17Fred Hutchinson Cancer CenterNCT06928662
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesMay 17Regeneron PharmaceuticalsNCT07154745
Added 3 new sites: Seoul, South Korea, Murcia, Spain, Salamanca, Spain

Added 3 new sites: Seoul, South Korea, Murcia, Spain, Salamanca, Spain

sites_addedMyelodysplastic SyndromesMay 17TakedaNCT06499285
Added 26 new sites: Kansas City, Missouri, Lake Success, New York, Winston-Salem, North Carolina and 23 more

Added 26 new sites: Kansas City, Missouri, Lake Success, New York, Winston-Salem, North Carolina and 23 more

sites_addedMyasthenia GravisMay 17AstraZenecaNCT07247279
Added 1 new site: Saint Petersburg, Russia

Added 1 new site: Saint Petersburg, Russia

sites_addedMyasthenia GravisMay 17Kyverna TherapeuticsNCT06193889
Added 2 new sites: Columbia, Missouri, Munich, Germany

Added 2 new sites: Columbia, Missouri, Munich, Germany

sites_addedMyasthenia GravisMay 17Novartis PharmaceuticalsNCT06517758
Added 1 new site: Porto Alegre, Brazil

Added 1 new site: Porto Alegre, Brazil

sites_addedMultiple System AtrophyMay 17Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 1 new site: Düsseldorf, Germany

Added 1 new site: Düsseldorf, Germany

sites_addedMultiple System AtrophyMay 17Dyne TherapeuticsNCT05481879
Added 3 new sites: Stanford, California, Gainesville, Florida, St Louis, Missouri

Added 3 new sites: Stanford, California, Gainesville, Florida, St Louis, Missouri

sites_removedMultiple SclerosisMay 17SanofiNCT06141473
Reduced from 384 to 382 sites

Reduced from 384 to 382 sites

Enrollment UpdateMultiple SclerosisMay 17SanofiNCT06141473
Target enrollment increased from 1600 to 1655

Target enrollment increased from 1600 to 1655

eligibility_changeMultiple SclerosisMay 17QurAlis CorporationNCT05633459
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMultiple SclerosisMay 17TG Therapeutics, Inc.NCT06433752
Target enrollment increased from 500 to 2000

Target enrollment increased from 500 to 2000

sites_addedMultiple SclerosisMay 17Novartis PharmaceuticalsNCT07225504
Added 15 new sites: Homestead, Florida, Foxborough, Massachusetts, Greenville, South Carolina and 12 more

Added 15 new sites: Homestead, Florida, Foxborough, Massachusetts, Greenville, South Carolina and 12 more

sites_removedMultiple SclerosisMay 17SanofiNCT06141486
Reduced from 338 to 311 sites

Reduced from 338 to 311 sites

sites_addedMultiple SclerosisMay 17Novartis PharmaceuticalsNCT06846281
Added 3 new sites: Buffalo, New York, São Paulo, Brazil, Bochum, Germany

Added 3 new sites: Buffalo, New York, São Paulo, Brazil, Bochum, Germany

sites_addedMultiple SclerosisMay 17National Tuberous Sclerosis AssociationNCT05676099
Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

sites_addedMultiple SclerosisMay 17SanofiNCT07325292
Added 6 new sites: Fort Collins, Colorado, Ghent, Belgium, Chengdu, China and 3 more

Added 6 new sites: Fort Collins, Colorado, Ghent, Belgium, Chengdu, China and 3 more

sites_addedMultiple SclerosisMay 17AmgenNCT06700343
Added 1 new site: Pensacola, Florida

Added 1 new site: Pensacola, Florida

sites_addedMixed Connective Tissue DiseaseMay 17Valent Technologies, LLCNCT04337177
Added 3 new sites: Phoenix, Arizona, Atlanta, Georgia, St Louis, Missouri

Added 3 new sites: Phoenix, Arizona, Atlanta, Georgia, St Louis, Missouri

sites_addedMesotheliomaMay 17Verismo TherapeuticsNCT05568680
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

sites_addedMembranous NephropathyMay 17Nkarta, Inc.NCT06557265
Added 2 new sites: Tustin, California, Summit, New Jersey

Added 2 new sites: Tustin, California, Summit, New Jersey

sites_addedMembranous NephropathyMay 17Vertex Pharmaceuticals IncorporatedNCT07204275
Added 7 new sites: Belo Horizonte, Brazil, Harbin, China, Shijiazhuang, China and 4 more

Added 7 new sites: Belo Horizonte, Brazil, Harbin, China, Shijiazhuang, China and 4 more

sites_addedLight Chain AmyloidosisMay 16AbbVieNCT06158854
Added 2 new sites: Nashville, Tennessee, Pavia, Italy

Added 2 new sites: Nashville, Tennessee, Pavia, Italy

sites_addedIgA NephropathyMay 16Hoffmann-La RocheNCT05797610
Added 2 new sites: Doral, Florida, Cleveland, Ohio

Added 2 new sites: Doral, Florida, Cleveland, Ohio

sites_addedIgA NephropathyMay 16TakedaNCT06963827
Added 1 new site: Prague, Czechia

Added 1 new site: Prague, Czechia

Enrollment UpdateIdiopathic Pulmonary FibrosisMay 16Assiut UniversityNCT05842681
Target enrollment increased from 30 to 1000

Target enrollment increased from 30 to 1000

eligibility_changeIdiopathic Pulmonary FibrosisMay 16Assiut UniversityNCT05842681
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisMay 16Boehringer IngelheimNCT06238622
Added 3 new sites: The Woodlands, Texas, Vienna, Austria, Exeter, United Kingdom

Added 3 new sites: The Woodlands, Texas, Vienna, Austria, Exeter, United Kingdom

sites_addedHunter SyndromeMay 16Denali Therapeutics Inc.NCT06075537
Added 1 new site: Manchester, United Kingdom

Added 1 new site: Manchester, United Kingdom

sites_addedHodgkin LymphomaMay 16GenmabNCT05283720
Added 15 new sites: Shanghai, China, Paris, France, Budapest, Hungary and 12 more

Added 15 new sites: Shanghai, China, Paris, France, Budapest, Hungary and 12 more

phase_changeHodgkin LymphomaMay 16RenJi HospitalNCT06852638
Moved from N/A to Phase 2

Moved from N/A to Phase 2

eligibility_changeHodgkin LymphomaMay 16T. John Winhusen, PhDNCT06651177
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 16Merck Sharp & Dohme LLCNCT06395103
Added 1 new site: Melbourne, Australia

Added 1 new site: Melbourne, Australia

sites_addedHodgkin LymphomaMay 16Merck Sharp & Dohme LLCNCT06717347
Added 5 new sites: Detroit, Michigan, Fargo, North Dakota, Hamilton, Canada and 2 more

Added 5 new sites: Detroit, Michigan, Fargo, North Dakota, Hamilton, Canada and 2 more

sites_addedHodgkin LymphomaMay 16Lyell Immunopharma, Inc.NCT05826535
Added 1 new site: Hackensack, New Jersey

Added 1 new site: Hackensack, New Jersey

sites_removedHodgkin LymphomaMay 16Memorial Sloan Kettering Cancer CenterNCT07566377
Reduced from 7 to 1 sites

Reduced from 7 to 1 sites

Enrollment UpdateHodgkin LymphomaMay 16Canadian Cancer Trials GroupNCT04161248
Target enrollment increased from 18 to 40

Target enrollment increased from 18 to 40

sites_addedHodgkin LymphomaMay 16AstraZenecaNCT06137118
Added 2 new sites: Kiel, Germany, Taoyuan, Taiwan

Added 2 new sites: Kiel, Germany, Taoyuan, Taiwan

eligibility_changeHodgkin LymphomaMay 16University of UtahNCT05723055
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHereditary AngioedemaMay 16Intellia TherapeuticsNCT06634420
Target enrollment increased from 60 to 80

Target enrollment increased from 60 to 80

Enrollment UpdateHereditary AngioedemaMay 16CSL BehringNCT06806657
Target enrollment decreased from 30 to 18

Target enrollment decreased from 30 to 18

sites_addedGraves’ DiseaseMay 15AmgenNCT06401044
Added 4 new sites: Thessaloniki, Greece, Miyazaki, Japan, Poznan, Poland and 1 more

Added 4 new sites: Thessaloniki, Greece, Miyazaki, Japan, Poznan, Poland and 1 more

Enrollment UpdateFragile X SyndromeMay 15Hospices Civils de LyonNCT06868979
Target enrollment increased from 88 to 118

Target enrollment increased from 88 to 118

eligibility_changeFragile X SyndromeMay 15Hospices Civils de LyonNCT06868979
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BMay 15CSL BehringNCT07080905
Added 1 new site: Madrid, Spain

Added 1 new site: Madrid, Spain

sites_addedHemophilia BMay 15PfizerNCT03861273
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedHemophilia BMay 15PfizerNCT05568719
Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

sites_addedHemophilia BMay 15PfizerNCT03307980
Added 13 new sites: Sacramento, California, Sacramento, California, Metairie, Louisiana and 10 more

Added 13 new sites: Sacramento, California, Sacramento, California, Metairie, Louisiana and 10 more

sites_addedHemophilia AMay 15PfizerNCT03861273
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedHemophilia AMay 15OctapharmaNCT05936580
Added 1 new site: Stasbourg, France

Added 1 new site: Stasbourg, France

sites_addedHemophilia AMay 15PfizerNCT05568719
Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

sites_addedHemophilia AMay 15PfizerNCT03307980
Added 13 new sites: Sacramento, California, Sacramento, California, Metairie, Louisiana and 10 more

Added 13 new sites: Sacramento, California, Sacramento, California, Metairie, Louisiana and 10 more

sites_addedGraves’ DiseaseMay 15Biohaven Therapeutics Ltd.NCT06980649
Added 4 new sites: Greenville, North Carolina, Canton, Ohio, Memphis, Tennessee and 1 more

Added 4 new sites: Greenville, North Carolina, Canton, Ohio, Memphis, Tennessee and 1 more

sites_addedGeographic AtrophyMay 15Novartis PharmaceuticalsNCT07441642
Added 2 new sites: Erie, Pennsylvania, Bellaire, Texas

Added 2 new sites: Erie, Pennsylvania, Bellaire, Texas

sites_addedFocal Segmental GlomerulosclerosisMay 15Boehringer IngelheimNCT07220083
Added 10 new sites: Brunswick, Georgia, Ahmedabad, India, Dehradun, India and 7 more

Added 10 new sites: Brunswick, Georgia, Ahmedabad, India, Dehradun, India and 7 more

sites_addedFocal EpilepsyMay 15Xenon Pharmaceuticals Inc.NCT05718817
Added 3 new sites: Buenos Aires, Argentina, San Luis, Argentina, Kuopio, Finland

Added 3 new sites: Buenos Aires, Argentina, San Luis, Argentina, Kuopio, Finland

sites_addedFocal EpilepsyMay 15Xenon Pharmaceuticals Inc.NCT05716100
Added 1 new site: Portland, Oregon

Added 1 new site: Portland, Oregon

sites_addedEssential ThrombocythemiaMay 15National Cancer Institute (NCI)NCT06661915
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedEssential ThrombocythemiaMay 15Merck Sharp & Dohme LLCNCT06456346
Added 3 new sites: Amarillo, Texas, Dallas, Texas, Webster, Texas

Added 3 new sites: Amarillo, Texas, Dallas, Texas, Webster, Texas

sites_addedEssential ThrombocythemiaMay 15Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

sites_addedEssential ThrombocythemiaMay 15Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

sites_addedEssential ThrombocythemiaMay 15Novartis PharmaceuticalsNCT07357727
Added 1 new site: Genolier, Switzerland

Added 1 new site: Genolier, Switzerland

sites_addedEssential ThrombocythemiaMay 15Disc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_removedDuchenne Muscular DystrophyMay 14Satellos Bioscience, Inc.NCT07287189
Reduced from 25 to 21 sites

Reduced from 25 to 21 sites

sites_addedDravet SyndromeMay 14EpygenixNCT04462770
Added 13 new sites: Birmingham, Alabama, Little Rock, Arkansas, Morrisville, North Carolina and 10 more

Added 13 new sites: Birmingham, Alabama, Little Rock, Arkansas, Morrisville, North Carolina and 10 more

sites_addedCystic FibrosisMay 14SpliSense Ltd.NCT06429176
Added 2 new sites: Los Angeles, California, Denver, Colorado

Added 2 new sites: Los Angeles, California, Denver, Colorado

Enrollment UpdateCystic FibrosisMay 14SpliSense Ltd.NCT06429176
Target enrollment increased from 24 to 64

Target enrollment increased from 24 to 64

eligibility_changeCystic FibrosisMay 14SpliSense Ltd.NCT06429176
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseMay 14BiogenNCT06044337
Added 1 new site: Porto Alegre, Brazil

Added 1 new site: Porto Alegre, Brazil

sites_addedChronic Graft-versus-Host DiseaseMay 14National Cancer Institute (NCI)NCT07061951
Added 4 new sites: Duarte, California, Irvine, California, Boston, Massachusetts and 1 more

Added 4 new sites: Duarte, California, Irvine, California, Boston, Massachusetts and 1 more

Enrollment UpdateChronic Graft-versus-Host DiseaseMay 14Novartis PharmaceuticalsNCT04925479
Target enrollment decreased from 44 to 34

Target enrollment decreased from 44 to 34

sites_addedCholangiocarcinomaMay 14J-Pharma Co., Ltd.NCT07265674
Added 7 new sites: Miami Beach, Florida, Tampa, Florida, Zion, Illinois and 4 more

Added 7 new sites: Miami Beach, Florida, Tampa, Florida, Zion, Illinois and 4 more

sites_addedCholangiocarcinomaMay 14Eli Lilly and CompanyNCT07213791
Added 1 new site: Chiba, Japan

Added 1 new site: Chiba, Japan

Enrollment UpdateEhlers-Danlos SyndromeMay 14Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

Enrollment UpdateDuchenne Muscular DystrophyMay 14Shanghai Siponuoyin Biotechnology Co LtdNCT07188012
Target enrollment increased from 6 to 9

Target enrollment increased from 6 to 9

eligibility_changeDuchenne Muscular DystrophyMay 14Shanghai Siponuoyin Biotechnology Co LtdNCT07188012
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateDilated CardiomyopathyMay 14Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_removedDermatomyositisMay 14PfizerNCT05895786
Reduced from 147 to 66 sites

Reduced from 147 to 66 sites

sites_removedDermatomyositisMay 14PfizerNCT06698796
Reduced from 24 to 7 sites

Reduced from 24 to 7 sites

sites_addedCystic FibrosisMay 14Krystal Biotech, Inc.NCT05504837
Added 1 new site: Boise, Idaho

Added 1 new site: Boise, Idaho

Enrollment UpdateCystic FibrosisMay 14Krystal Biotech, Inc.NCT05504837
Target enrollment increased from 12 to 20

Target enrollment increased from 12 to 20

eligibility_changeCystic FibrosisMay 14Krystal Biotech, Inc.NCT05504837
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCystic FibrosisMay 14Arcturus Therapeutics, Inc.NCT06747858
Added 6 new sites: Washington D.C., District of Columbia, St Louis, Missouri, Cleveland, Ohio and 3 more

Added 6 new sites: Washington D.C., District of Columbia, St Louis, Missouri, Cleveland, Ohio and 3 more

Enrollment UpdateCystic FibrosisMay 14Arcturus Therapeutics, Inc.NCT06747858
Target enrollment increased from 12 to 33

Target enrollment increased from 12 to 33

eligibility_changeCystic FibrosisMay 14Arcturus Therapeutics, Inc.NCT06747858
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCushing DiseaseMay 14Sparrow PharmaceuticalsNCT07296484
Added 5 new sites: New Port Richey, Florida, Port Charlotte, Florida, Edgewood, Kentucky and 2 more

Added 5 new sites: New Port Richey, Florida, Port Charlotte, Florida, Edgewood, Kentucky and 2 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14Dianthus TherapeuticsNCT06858579
Added 14 new sites: Nashville, Tennessee, Buenos Aires, Argentina, Liverpool, Australia and 11 more

Added 14 new sites: Nashville, Tennessee, Buenos Aires, Argentina, Liverpool, Australia and 11 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14Janssen Research & Development, LLCNCT05327114
Added 5 new sites: Marseille, France, Nîmes, France, Milan, Italy and 2 more

Added 5 new sites: Marseille, France, Nîmes, France, Milan, Italy and 2 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14argenxNCT06920004
Added 27 new sites: Tampa, Florida, Rosario, Argentina, Vienna, Austria and 24 more

Added 27 new sites: Tampa, Florida, Rosario, Argentina, Vienna, Austria and 24 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14argenxNCT07091630
Added 24 new sites: Rancho Mirage, California, Naples, Florida, Philadelphia, Pennsylvania and 21 more

Added 24 new sites: Rancho Mirage, California, Naples, Florida, Philadelphia, Pennsylvania and 21 more

sites_addedChronic Graft-versus-Host DiseaseMay 14Dianthus TherapeuticsNCT06858579
Added 14 new sites: Nashville, Tennessee, Buenos Aires, Argentina, Liverpool, Australia and 11 more

Added 14 new sites: Nashville, Tennessee, Buenos Aires, Argentina, Liverpool, Australia and 11 more

sites_addedChronic Graft-versus-Host DiseaseMay 14Janssen Research & Development, LLCNCT05327114
Added 5 new sites: Marseille, France, Nîmes, France, Milan, Italy and 2 more

Added 5 new sites: Marseille, France, Nîmes, France, Milan, Italy and 2 more

sites_addedChronic Graft-versus-Host DiseaseMay 14Mirum Pharmaceuticals, Inc.NCT07454837
Added 9 new sites: Brno, Czechia, Prague, Czechia, Prague, Czechia and 6 more

Added 9 new sites: Brno, Czechia, Prague, Czechia, Prague, Czechia and 6 more

eligibility_changeChronic Graft-versus-Host DiseaseMay 14University of Colorado, DenverNCT05896579
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseMay 14Alliance for Clinical Trials in OncologyNCT05334069
Added 2 new sites: Clinton, Maryland, Olney, Maryland

Added 2 new sites: Clinton, Maryland, Olney, Maryland

sites_addedCholangiocarcinomaMay 14Verismo TherapeuticsNCT05568680
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

sites_addedCholangiocarcinomaMay 14Kestrel Therapeutics, Inc.NCT07458347
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

eligibility_changeCholangiocarcinomaMay 14Nantes University HospitalNCT06735560
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaMay 14Institut de Recherches Internationales ServierNCT06501625
Added 1 new site: Los Alamitos, California

Added 1 new site: Los Alamitos, California

sites_addedCholangiocarcinomaMay 14Jazz PharmaceuticalsNCT06282575
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedCholangiocarcinomaMay 14BioNTech SENCT07186842
Added 2 new sites: Ulm, Germany, Barcelona, Spain

Added 2 new sites: Ulm, Germany, Barcelona, Spain

Enrollment UpdateCatecholaminergic Polymorphic Ventricular TachycardiaMay 14Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

eligibility_changeAlpha-1 Antitrypsin DeficiencyMay 13TakedaNCT05677971
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateBecker Muscular DystrophyMay 13Shanghai Siponuoyin Biotechnology Co LtdNCT07188012
Target enrollment increased from 6 to 9

Target enrollment increased from 6 to 9

eligibility_changeBecker Muscular DystrophyMay 13Shanghai Siponuoyin Biotechnology Co LtdNCT07188012
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAtypical Hemolytic Uremic SyndromeMay 13Novartis PharmaceuticalsNCT05795140
Added 2 new sites: Iruma-gun, Japan, Mersin, Turkey (Türkiye)

Added 2 new sites: Iruma-gun, Japan, Mersin, Turkey (Türkiye)

Enrollment UpdateAtypical Hemolytic Uremic SyndromeMay 13Novartis PharmaceuticalsNCT05795140
Target enrollment decreased from 125 to 65

Target enrollment decreased from 125 to 65

eligibility_changeAtypical Hemolytic Uremic SyndromeMay 13Novartis PharmaceuticalsNCT05795140
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 13Eidos Therapeutics, a BridgeBio companyNCT06563895
Added 3 new sites: Seattle, Washington, Murdoch, Australia, Woolloongabba, Australia

Added 3 new sites: Seattle, Washington, Murdoch, Australia, Woolloongabba, Australia

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)May 13Eidos Therapeutics, a BridgeBio companyNCT06563895
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateArrhythmogenic CardiomyopathyMay 13Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_removedAmyotrophic Lateral SclerosisMay 13University of AlbertaNCT03362658
Reduced from 9 to 7 sites

Reduced from 9 to 7 sites

Enrollment UpdateAmyotrophic Lateral SclerosisMay 13University of AlbertaNCT03362658
Target enrollment decreased from 700 to 119

Target enrollment decreased from 700 to 119

sites_addedAchondroplasiaMay 13BioMarin PharmaceuticalNCT07441876
Added 2 new sites: California City, California, Parkville, Australia

Added 2 new sites: California City, California, Parkville, Australia

sites_removedWaldenström MacroglobulinemiaMay 12Christian BuskeNCT04263480
Reduced from 20 to 17 sites

Reduced from 20 to 17 sites

sites_removedSystemic Juvenile Idiopathic ArthritisMay 12Eli Lilly and CompanyNCT03773965
Reduced from 82 to 80 sites

Reduced from 82 to 80 sites

sites_addedSpinal Muscular AtrophyMay 12Novartis PharmaceuticalsNCT07190300
Added 1 new site: Iowa City, Iowa

Added 1 new site: Iowa City, Iowa

sites_addedSickle Cell DiseaseMay 12Disc Medicine, IncNCT07187973
Added 2 new sites: Birmingham, Alabama, Augusta, Georgia

Added 2 new sites: Birmingham, Alabama, Augusta, Georgia

sites_addedSickle Cell DiseaseMay 12CSL BehringNCT07224360
Added 2 new sites: Little Rock, Arkansas, Bethesda, Maryland

Added 2 new sites: Little Rock, Arkansas, Bethesda, Maryland

sites_addedWaldenström MacroglobulinemiaMay 12BeOne MedicinesNCT05006716
Added 1 new site: Shenyang, China

Added 1 new site: Shenyang, China

sites_addedWaldenström MacroglobulinemiaMay 12Janssen Research & Development, LLCNCT01804686
Added 2 new sites: Melbourne, Australia, Warsaw, Poland

Added 2 new sites: Melbourne, Australia, Warsaw, Poland

Enrollment UpdateVascular Ehlers-Danlos SyndromeMay 12Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_addedTuberous SclerosisMay 12National Tuberous Sclerosis AssociationNCT05676099
Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

sites_addedStickler SyndromeMay 12Helen Keller Eye Research FoundationNCT07146516
Added 2 new sites: Royal Oak, Michigan, Westbury, New York

Added 2 new sites: Royal Oak, Michigan, Westbury, New York

sites_addedStargardt DiseaseMay 12Astellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedSpinal Muscular AtrophyMay 12CelgeneNCT06764485
Added 7 new sites: Anaheim, California, San Francisco, California, Santa Rosa, California and 4 more

Added 7 new sites: Anaheim, California, San Francisco, California, Santa Rosa, California and 4 more

sites_addedSpinal Muscular AtrophyMay 12Novartis PharmaceuticalsNCT07190300
Added 1 new site: Jena, Germany

Added 1 new site: Jena, Germany

eligibility_changeSpinal Muscular AtrophyMay 12Novartis PharmaceuticalsNCT07190300
Eligibility criteria have been updated

Eligibility criteria have been updated

phase_changeSickle Cell DiseaseMay 12Children's Hospital Medical Center, CincinnatiNCT06290401
Moved from Phase 2 to N/A

Moved from Phase 2 to N/A

Enrollment UpdateSickle Cell DiseaseMay 12St. Jude Children's Research HospitalNCT05810181
Target enrollment increased from 70 to 145

Target enrollment increased from 70 to 145

eligibility_changeSickle Cell DiseaseMay 12St. Jude Children's Research HospitalNCT05810181
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 12Disc Medicine, IncNCT07187973
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

eligibility_changeSickle Cell DiseaseMay 12Boston Children's HospitalNCT06555939
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedRetinitis PigmentosaMay 11Janssen Research & Development, LLCNCT04794101
Added 3 new sites: Bellaire, Texas, Milan, Italy, Rome, Italy

Added 3 new sites: Bellaire, Texas, Milan, Italy, Rome, Italy

sites_removedPulmonary Arterial HypertensionMay 11PfizerNCT06137742
Reduced from 67 to 31 sites

Reduced from 67 to 31 sites

sites_removedPulmonary Arterial HypertensionMay 11PfizerNCT07073820
Reduced from 44 to 22 sites

Reduced from 44 to 22 sites

eligibility_changePulmonary Arterial HypertensionMay 11University of Colorado, DenverNCT05896579
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPrimary Sclerosing CholangitisMay 11PSC Partners Seeking a CureNCT06297993
Added 2 new sites: Dallas, Texas, Oxford, United Kingdom

Added 2 new sites: Dallas, Texas, Oxford, United Kingdom

sites_addedPolycythemia VeraMay 11Novartis PharmaceuticalsNCT07357727
Added 1 new site: Berkeley Heights, New Jersey

Added 1 new site: Berkeley Heights, New Jersey

Enrollment UpdatePolycythemia VeraMay 11Icahn School of Medicine at Mount SinaiNCT05835466
Target enrollment decreased from 26 to 10

Target enrollment decreased from 26 to 10

sites_addedPhenylketonuriaMay 11PTC TherapeuticsNCT06302348
Added 3 new sites: Tours, France, Dublin, Ireland, Warsaw, Poland

Added 3 new sites: Tours, France, Dublin, Ireland, Warsaw, Poland

sites_addedPhelan-McDermid SyndromeMay 11Neuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Brookline, Massachusetts, Cincinnati, Ohio

Added 2 new sites: Brookline, Massachusetts, Cincinnati, Ohio

sites_addedOsteogenesis ImperfectaMay 11AmgenNCT07366086
Added 1 new site: Okayama, Japan

Added 1 new site: Okayama, Japan

sites_addedNarcolepsy Type 1May 10TakedaNCT07363720
Added 11 new sites: Middletown, New Jersey, Durham, North Carolina, Winston-Salem, North Carolina and 8 more

Added 11 new sites: Middletown, New Jersey, Durham, North Carolina, Winston-Salem, North Carolina and 8 more

sites_addedNarcolepsy Type 1May 10Alkermes, Inc.NCT07455383
Added 6 new sites: Brandon, Florida, Macon, Georgia, Lansing, Michigan and 3 more

Added 6 new sites: Brandon, Florida, Macon, Georgia, Lansing, Michigan and 3 more

sites_addedMyotonic DystrophyMay 10Dyne TherapeuticsNCT07486934
Added 1 new site: Toyonaka-Shi, Japan

Added 1 new site: Toyonaka-Shi, Japan

sites_addedMyelofibrosisMay 10Novartis PharmaceuticalsNCT07357727
Added 1 new site: Berkeley Heights, New Jersey

Added 1 new site: Berkeley Heights, New Jersey

Enrollment UpdateMyelofibrosisMay 10Icahn School of Medicine at Mount SinaiNCT05835466
Target enrollment decreased from 26 to 10

Target enrollment decreased from 26 to 10

sites_removedMyelofibrosisMay 10Bristol-Myers SquibbNCT04817007
Reduced from 53 to 49 sites

Reduced from 53 to 49 sites

Enrollment UpdateMyelodysplastic SyndromesMay 10M.D. Anderson Cancer CenterNCT04493138
Target enrollment decreased from 58 to 30

Target enrollment decreased from 58 to 30

Enrollment UpdateMyelodysplastic SyndromesMay 10H. Lee Moffitt Cancer Center and Research InstituteNCT05732961
Target enrollment decreased from 70 to 6

Target enrollment decreased from 70 to 6

sites_addedMyelodysplastic SyndromesMay 10National Cancer Institute (NCI)NCT05554406
Added 2 new sites: Los Angeles, California, Pembroke Pines, Florida

Added 2 new sites: Los Angeles, California, Pembroke Pines, Florida

sites_addedMyelodysplastic SyndromesMay 10GenmabNCT07384715
Added 9 new sites: Aarhus, Denmark, Copenhagen, Denmark, Berlin, Germany and 6 more

Added 9 new sites: Aarhus, Denmark, Copenhagen, Denmark, Berlin, Germany and 6 more

sites_addedMyelodysplastic SyndromesMay 10PedAL BCU, LLCNCT04726241
Added 1 new site: Winfield, Illinois

Added 1 new site: Winfield, Illinois

sites_removedMyelodysplastic SyndromesMay 10Bristol-Myers SquibbNCT05949684
Reduced from 169 to 144 sites

Reduced from 169 to 144 sites

phase_changeMyelodysplastic SyndromesMay 10University of VirginiaNCT04482894
Moved from Phase 2 to N/A

Moved from Phase 2 to N/A

sites_addedMyasthenia GravisMay 10Novartis PharmaceuticalsNCT06517758
Added 2 new sites: Córdoba, Argentina, Joinville, Brazil

Added 2 new sites: Córdoba, Argentina, Joinville, Brazil

sites_addedMyasthenia GravisMay 10Janssen Research & Development, LLCNCT07217587
Added 4 new sites: Pomona, California, Fort Collins, Colorado, Raleigh, North Carolina and 1 more

Added 4 new sites: Pomona, California, Fort Collins, Colorado, Raleigh, North Carolina and 1 more

sites_addedMyasthenia GravisMay 10Novartis PharmaceuticalsNCT06744920
Added 1 new site: Niš, Serbia

Added 1 new site: Niš, Serbia

sites_addedMyasthenia GravisMay 10UCB Biopharma SRLNCT06149559
Added 2 new sites: Denton, Texas, Ankara, Turkey (Türkiye)

Added 2 new sites: Denton, Texas, Ankara, Turkey (Türkiye)

sites_addedMyasthenia GravisMay 10Vertex Pharmaceuticals IncorporatedNCT07501702
Added 3 new sites: Boca Raton, Florida, Homestead, Florida, Las Vegas, Nevada

Added 3 new sites: Boca Raton, Florida, Homestead, Florida, Las Vegas, Nevada

sites_addedMyasthenia GravisMay 10Vor BiopharmaNCT06456580
Added 34 new sites: Tampa, Florida, Mendoza, Argentina, Liège, Belgium and 31 more

Added 34 new sites: Tampa, Florida, Mendoza, Argentina, Liège, Belgium and 31 more

sites_addedMultiple System AtrophyMay 10Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Salerno, Italy, Niigata, Japan

Added 2 new sites: Salerno, Italy, Niigata, Japan

Enrollment UpdateMultiple SclerosisMay 10NYU Langone HealthNCT07011914
Target enrollment decreased from 386 to 300

Target enrollment decreased from 386 to 300

Enrollment UpdateMultiple SclerosisMay 10Poitiers University HospitalNCT05491031
Target enrollment decreased from 250 to 50

Target enrollment decreased from 250 to 50

sites_addedMultiple SclerosisMay 10Novartis PharmaceuticalsNCT07225504
Added 22 new sites: Washington D.C., District of Columbia, Miami, Florida, Albuquerque, New Mexico and 19 more

Added 22 new sites: Washington D.C., District of Columbia, Miami, Florida, Albuquerque, New Mexico and 19 more

sites_addedMultiple SclerosisMay 10CelgeneNCT06782490
Added 4 new sites: Birmingham, Alabama, Southfield, Michigan, Hradec Králové, Czechia and 1 more

Added 4 new sites: Birmingham, Alabama, Southfield, Michigan, Hradec Králové, Czechia and 1 more

sites_addedMultiple SclerosisMay 10SandozNCT06847724
Added 1 new site: Lublin, Poland

Added 1 new site: Lublin, Poland

sites_addedMultiple SclerosisMay 10Reunion Neuroscience IncNCT07002034
Added 2 new sites: Kansas City, Kansas, Omaha, Nebraska

Added 2 new sites: Kansas City, Kansas, Omaha, Nebraska

sites_addedMultiple SclerosisMay 10Zenas BioPharma (USA), LLCNCT07299019
Added 4 new sites: Scottsdale, Arizona, Boston, Massachusetts, San Antonio, Texas and 1 more

Added 4 new sites: Scottsdale, Arizona, Boston, Massachusetts, San Antonio, Texas and 1 more

eligibility_changeMultiple SclerosisMay 10National Institute of Neurological Disorders and Stroke (NINDS)NCT01730131
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 10Novartis PharmaceuticalsNCT05344469
Added 4 new sites: Albstadt, Germany, Magdeburg, Germany, Schneverdingen, Germany and 1 more

Added 4 new sites: Albstadt, Germany, Magdeburg, Germany, Schneverdingen, Germany and 1 more

eligibility_changeMixed Connective Tissue DiseaseMay 10Memorial Sloan Kettering Cancer CenterNCT05985161
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMesotheliomaMay 10University of NebraskaNCT02012699
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedMembranous NephropathyMay 10BiogenNCT06962800
Added 2 new sites: St Albans, Australia, Chandigarh, India

Added 2 new sites: St Albans, Australia, Chandigarh, India

Enrollment UpdateMarfan SyndromeMay 9Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_addedLymphangioleiomyomatosisMay 9National Tuberous Sclerosis AssociationNCT05676099
Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

sites_addedLight Chain AmyloidosisMay 9Shanghai Chia Tai Tianqing Pharmaceutical Technology Development Co., Ltd.NCT07266116
Added 4 new sites: Guangzhou, China, Changchun, China, Changchun, China and 1 more

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

eligibility_changeLight Chain AmyloidosisMay 9Shanghai Chia Tai Tianqing Pharmaceutical Technology Development Co., Ltd.NCT07266116
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLight Chain AmyloidosisMay 9SWOG Cancer Research NetworkNCT06022939
Added 84 new sites: Goodyear, Arizona, Tucson, Arizona, Tucson, Arizona and 81 more

Added 84 new sites: Goodyear, Arizona, Tucson, Arizona, Tucson, Arizona and 81 more

sites_addedIgG4-Related DiseaseMay 9SanofiNCT07190196
Added 2 new sites: Hirakata, Japan, Riyadh, Saudi Arabia

Added 2 new sites: Hirakata, Japan, Riyadh, Saudi Arabia

sites_addedIgA NephropathyMay 9BiogenNCT06935357
Added 5 new sites: Philadelphia, Pennsylvania, Santa Fe, Argentina, Fontevivo, Italy and 2 more

Added 5 new sites: Philadelphia, Pennsylvania, Santa Fe, Argentina, Fontevivo, Italy and 2 more

sites_addedIgA NephropathyMay 9Otsuka Pharmaceutical Development & Commercialization, Inc.NCT05248659
Added 158 new sites: Birmingham, Alabama, Los Angeles, California, Palo Alto, California and 155 more

Added 158 new sites: Birmingham, Alabama, Los Angeles, California, Palo Alto, California and 155 more

eligibility_changeIgA NephropathyMay 9Otsuka Pharmaceutical Development & Commercialization, Inc.NCT05248659
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyMay 9Novartis PharmaceuticalsNCT06858319
Added 10 new sites: Orlando, Florida, New York, New York, Dallas, Texas and 7 more

Added 10 new sites: Orlando, Florida, New York, New York, Dallas, Texas and 7 more

eligibility_changeIgA NephropathyMay 9TakedaNCT06963827
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisMay 9Mediar TherapeuticsNCT06967805
Added 3 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Curitiba, Brazil

Added 3 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Curitiba, Brazil

sites_addedIdiopathic Pulmonary FibrosisMay 9Duke UniversityNCT01915511
Added 3 new sites: Aurora, Colorado, Houston, Texas, Salt Lake City, Utah

Added 3 new sites: Aurora, Colorado, Houston, Texas, Salt Lake City, Utah

sites_addedIdiopathic Pulmonary FibrosisMay 9Vejle HospitalNCT06714123
Added 3 new sites: Copenhagen, Denmark, Leicester, United Kingdom, Norwich, United Kingdom

Added 3 new sites: Copenhagen, Denmark, Leicester, United Kingdom, Norwich, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisMay 9Reunion Neuroscience IncNCT07002034
Added 2 new sites: Kansas City, Kansas, Omaha, Nebraska

Added 2 new sites: Kansas City, Kansas, Omaha, Nebraska

sites_addedIdiopathic Pulmonary FibrosisMay 9Rein TherapeuticsNCT06968845
Added 2 new sites: New Haven, Connecticut, Edinburgh, United Kingdom

Added 2 new sites: New Haven, Connecticut, Edinburgh, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisMay 9Mannkind CorporationNCT07344558
Added 1 new site: McKinney, Texas

Added 1 new site: McKinney, Texas

sites_addedIdiopathic Pulmonary FibrosisMay 9United TherapeuticsNCT04905693
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedHypoparathyroidismMay 9Ascendis Pharma Bone Diseases A/SNCT07081997
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedHomozygous Familial HypercholesterolemiaMay 9Arrowhead PharmaceuticalsNCT07037771
Added 15 new sites: La Louvière, Belgium, Leuven, Belgium, Cerqueira César, Brazil and 12 more

Added 15 new sites: La Louvière, Belgium, Leuven, Belgium, Cerqueira César, Brazil and 12 more

sites_addedHomocystinuriaMay 9Travere Therapeutics, Inc.NCT06247085
Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

eligibility_changeHomocystinuriaMay 9Travere Therapeutics, Inc.NCT06247085
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaMay 9BeOne MedicinesNCT06634589
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 9Allogene TherapeuticsNCT06500273
Added 3 new sites: Indianapolis, Indiana, Baltimore, Maryland, Montreal, Canada

Added 3 new sites: Indianapolis, Indiana, Baltimore, Maryland, Montreal, Canada

eligibility_changeHodgkin LymphomaMay 9University of WashingtonNCT07097363
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 9National Cancer Institute (NCI)NCT06649812
Added 2 new sites: Los Angeles, California, Durham, North Carolina

Added 2 new sites: Los Angeles, California, Durham, North Carolina

Enrollment UpdateHodgkin LymphomaMay 9Kite, A Gilead CompanyNCT06539338
Target enrollment decreased from 34 to 10

Target enrollment decreased from 34 to 10

sites_addedHodgkin LymphomaMay 9ModeX Therapeutics, An OPKO Health CompanyNCT07249905
Added 1 new site: Richmond, Australia

Added 1 new site: Richmond, Australia

sites_addedHodgkin LymphomaMay 9Merck Sharp & Dohme LLCNCT05139017
Added 3 new sites: Niles, Illinois, Sapporo, Japan, Bunkyo, Japan

Added 3 new sites: Niles, Illinois, Sapporo, Japan, Bunkyo, Japan

Enrollment UpdateHodgkin LymphomaMay 9Miltenyi Biomedicine GmbHNCT04844866
Target enrollment increased from 45 to 213

Target enrollment increased from 45 to 213

Enrollment UpdateHereditary Spastic ParaplegiaMay 9National Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
Target enrollment decreased from 9300 to 2323

Target enrollment decreased from 9300 to 2323

sites_addedHereditary AngioedemaMay 9Astria Therapeutics, Inc.NCT06842823
Added 2 new sites: Dallas, Texas, Milan, Italy

Added 2 new sites: Dallas, Texas, Milan, Italy

sites_addedHereditary AngioedemaMay 9Pharvaris Netherlands B.V.NCT07266805
Added 9 new sites: Sofia, Bulgaria, Paris, France, Berlin, Germany and 6 more

Added 9 new sites: Sofia, Bulgaria, Paris, France, Berlin, Germany and 6 more

sites_addedHereditary AngioedemaMay 9Pharvaris Netherlands B.V.NCT06679881
Added 37 new sites: Birmingham, Alabama, Scottsdale, Arizona, Little Rock, Arkansas and 34 more

Added 37 new sites: Birmingham, Alabama, Scottsdale, Arizona, Little Rock, Arkansas and 34 more

sites_addedGraves’ DiseaseMay 8Immunovant Sciences GmbHNCT06727604
Added 3 new sites: Metairie, Louisiana, Nashville, Tennessee, Lublin, Poland

Added 3 new sites: Metairie, Louisiana, Nashville, Tennessee, Lublin, Poland

sites_addedGraves’ DiseaseMay 8Immunovant Sciences GmbHNCT07018323
Added 6 new sites: Parkville, Australia, Campinas, Brazil, Rio de Janeiro, Brazil and 3 more

Added 6 new sites: Parkville, Australia, Campinas, Brazil, Rio de Janeiro, Brazil and 3 more

sites_addedGastrointestinal Stromal TumorMay 8Cancer Research UKNCT06380816
Added 2 new sites: London, United Kingdom, Newcastle upon Tyne, United Kingdom

Added 2 new sites: London, United Kingdom, Newcastle upon Tyne, United Kingdom

Enrollment UpdateFamilial Hypertrophic CardiomyopathyMay 8Hospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_addedEssential ThrombocythemiaMay 8Novartis PharmaceuticalsNCT07357727
Added 1 new site: Berkeley Heights, New Jersey

Added 1 new site: Berkeley Heights, New Jersey

sites_removedHemophilia BMay 8PfizerNCT03307980
Reduced from 25 to 12 sites

Reduced from 25 to 12 sites

sites_removedHemophilia BMay 8PfizerNCT05568719
Reduced from 17 to 8 sites

Reduced from 17 to 8 sites

sites_removedHemophilia BMay 8PfizerNCT05611801
Reduced from 66 to 34 sites

Reduced from 66 to 34 sites

sites_addedHemophilia BMay 8SanofiNCT07285460
Added 3 new sites: Ribeirão Preto, Brazil, Taichung, Taiwan, Taipei, Taiwan

Added 3 new sites: Ribeirão Preto, Brazil, Taichung, Taiwan, Taipei, Taiwan

sites_addedHemophilia AMay 8Hoffmann-La RocheNCT07416604
Added 3 new sites: Orange, California, Indianapolis, Indiana, Seattle, Washington

Added 3 new sites: Orange, California, Indianapolis, Indiana, Seattle, Washington

sites_removedHemophilia AMay 8PfizerNCT03307980
Reduced from 25 to 12 sites

Reduced from 25 to 12 sites

sites_removedHemophilia AMay 8PfizerNCT05568719
Reduced from 17 to 8 sites

Reduced from 17 to 8 sites

sites_removedHemophilia AMay 8PfizerNCT05611801
Reduced from 66 to 34 sites

Reduced from 66 to 34 sites

sites_addedHemophilia AMay 8SanofiNCT07285460
Added 3 new sites: Ribeirão Preto, Brazil, Taichung, Taiwan, Taipei, Taiwan

Added 3 new sites: Ribeirão Preto, Brazil, Taichung, Taiwan, Taipei, Taiwan

sites_addedGeographic AtrophyMay 8Novartis PharmaceuticalsNCT07441642
Added 7 new sites: Sacramento, California, Toms River, New Jersey, Houston, Texas and 4 more

Added 7 new sites: Sacramento, California, Toms River, New Jersey, Houston, Texas and 4 more

sites_addedGeographic AtrophyMay 8Complement TherapeuticsNCT07392255
Added 1 new site: La Crosse, Wisconsin

Added 1 new site: La Crosse, Wisconsin

Enrollment UpdateGeographic AtrophyMay 8Hoffmann-La RocheNCT06961370
Target enrollment decreased from 132 to 27

Target enrollment decreased from 132 to 27

sites_addedGeographic AtrophyMay 8Complement TherapeuticsNCT07144137
Added 2 new sites: Gloucester, United Kingdom, London, United Kingdom

Added 2 new sites: Gloucester, United Kingdom, London, United Kingdom

sites_addedGeographic AtrophyMay 8Annexon, Inc.NCT06510816
Added 18 new sites: Fullerton, California, Tampa, Florida, Elmhurst, Illinois and 15 more

Added 18 new sites: Fullerton, California, Tampa, Florida, Elmhurst, Illinois and 15 more

eligibility_changeGeographic AtrophyMay 8Annexon, Inc.NCT06510816
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyMay 8i-Lumen Scientific AUS PTY LTDNCT06662162
Added 4 new sites: Manchester, Connecticut, Fort Worth, Texas, Tyler, Texas and 1 more

Added 4 new sites: Manchester, Connecticut, Fort Worth, Texas, Tyler, Texas and 1 more

sites_addedGastrointestinal Stromal TumorMay 8IDRX, Inc., a wholly owned subsidiary of GSK, LLCNCT05489237
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

eligibility_changeGastrointestinal Stromal TumorMay 8IDRX, Inc., a wholly owned subsidiary of GSK, LLCNCT05489237
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedGastrointestinal Stromal TumorMay 8University of NebraskaNCT02012699
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedFacioscapulohumeral Muscular DystrophyMay 8University of Kansas Medical CenterNCT04635891
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedEssential ThrombocythemiaMay 8Merck Sharp & Dohme LLCNCT06079879
Added 3 new sites: Edmonton, Canada, Santiago de Compostela, Spain, Taoyuan, Taiwan

Added 3 new sites: Edmonton, Canada, Santiago de Compostela, Spain, Taoyuan, Taiwan

sites_addedEssential ThrombocythemiaMay 8Disc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

Enrollment UpdateEssential ThrombocythemiaMay 8Icahn School of Medicine at Mount SinaiNCT05835466
Target enrollment decreased from 26 to 10

Target enrollment decreased from 26 to 10

sites_addedEssential ThrombocythemiaMay 8Merck Sharp & Dohme LLCNCT06456346
Added 3 new sites: Eugene, Oregon, L'Hospitalet de Llobregat, Spain, Taoyuan, Taiwan

Added 3 new sites: Eugene, Oregon, L'Hospitalet de Llobregat, Spain, Taoyuan, Taiwan

sites_addedEssential ThrombocythemiaMay 8Novartis PharmaceuticalsNCT07357727
Expanded from 3 to 4 sites

Expanded from 3 to 4 sites

eligibility_changeDuchenne Muscular DystrophyMay 7Sarepta Therapeutics, Inc.NCT06270719
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 7Ionis Pharmaceuticals, Inc.NCT07531745
Added 1 new site: Memphis, Tennessee

Added 1 new site: Memphis, Tennessee

Enrollment UpdateCystic FibrosisMay 7Centre Hospitalier Universitaire de la RéunionNCT06371261
Target enrollment decreased from 35 to 17

Target enrollment decreased from 35 to 17

sites_addedCystic FibrosisMay 7Verona Pharma, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USANCT06559150
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

Enrollment UpdateCystic FibrosisMay 7Verona Pharma, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USANCT06559150
Target enrollment increased from 180 to 284

Target enrollment increased from 180 to 284

eligibility_changeCystic FibrosisMay 7Verona Pharma, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USANCT06559150
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCushing DiseaseMay 7National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT06635629
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 7SanofiNCT06290128
Added 1 new site: Las Vegas, Nevada

Added 1 new site: Las Vegas, Nevada

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 7SanofiNCT06290141
Added 3 new sites: Bradenton, Florida, Las Vegas, Nevada, Milan, Italy

Added 3 new sites: Bradenton, Florida, Las Vegas, Nevada, Milan, Italy

sites_addedCholangiocarcinomaMay 7Bold Therapeutics, Inc.NCT04421820
Added 2 new sites: Bonn, Germany, Ulm, Germany

Added 2 new sites: Bonn, Germany, Ulm, Germany

sites_addedCholangiocarcinomaMay 7Kestrel Therapeutics, Inc.NCT07458347
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedCholangiocarcinomaMay 7Myeloid TherapeuticsNCT07334119
Added 1 new site: Melbourne, Australia

Added 1 new site: Melbourne, Australia

sites_removedCholangiocarcinomaMay 7University of NebraskaNCT02012699
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedCentral Diabetes InsipidusMay 7University Hospital, Basel, SwitzerlandNCT06036004
Added 1 new site: Berlin, Germany

Added 1 new site: Berlin, Germany

sites_addedCentral Diabetes InsipidusMay 7Children's Oncology GroupNCT06368817
Added 2 new sites: Indianapolis, Indiana, Winnipeg, Canada

Added 2 new sites: Indianapolis, Indiana, Winnipeg, Canada

sites_addedAmyotrophic Lateral SclerosisMay 6Reunion Neuroscience IncNCT07002034
Added 2 new sites: Kansas City, Kansas, Omaha, Nebraska

Added 2 new sites: Kansas City, Kansas, Omaha, Nebraska

Enrollment UpdateAlpha-1 Antitrypsin DeficiencyMay 6BioMarin PharmaceuticalNCT06738017
Target enrollment decreased from 12 to 6

Target enrollment decreased from 12 to 6

eligibility_changeBecker Muscular DystrophyMay 6Sarepta Therapeutics, Inc.NCT06270719
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 6AstraZenecaNCT06465810
Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 6BayerNCT07298044
Added 1 new site: Braunau am Inn, Austria

Added 1 new site: Braunau am Inn, Austria

eligibility_changeAmyotrophic Lateral SclerosisMay 6National Institute of Neurological Disorders and Stroke (NINDS)NCT07204977
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisMay 6Merit E. Cudkowicz, MDNCT04297683
Added 1 new site: Rochester, New York

Added 1 new site: Rochester, New York

eligibility_changeAmyotrophic Lateral SclerosisMay 6Centre hospitalier de l'Université de Montréal (CHUM)NCT06051123
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAlagille SyndromeMay 6Albireo, an Ipsen CompanyNCT05035030
Added 4 new sites: New York, New York, South Brisbane, Australia, Petah Tikva, Israel and 1 more

Added 4 new sites: New York, New York, South Brisbane, Australia, Petah Tikva, Israel and 1 more

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 5AstraZenecaNCT06465810
Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

sites_addedWaldenström MacroglobulinemiaMay 5Christian BuskeNCT05099471
Added 3 new sites: Mutlangen, Germany, Paderborn, Germany, Rostock, Germany

Added 3 new sites: Mutlangen, Germany, Paderborn, Germany, Rostock, Germany

sites_addedStargardt DiseaseMay 5Alkeus Pharmaceuticals, Inc.NCT07419334
Added 2 new sites: Phoenix, Arizona, Gainesville, Florida

Added 2 new sites: Phoenix, Arizona, Gainesville, Florida

Enrollment UpdateHereditary Spastic Paraplegia Type 4May 5National Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
Target enrollment decreased from 9300 to 2323

Target enrollment decreased from 9300 to 2323

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 5BayerNCT07298044
Added 1 new site: Braunau am Inn, Austria

Added 1 new site: Braunau am Inn, Austria

sites_addedWaldenström MacroglobulinemiaMay 5ModeX Therapeutics, An OPKO Health CompanyNCT07249905
Added 1 new site: Richmond, Australia

Added 1 new site: Richmond, Australia

sites_addedSpinal Muscular AtrophyMay 5Scholar Rock, Inc.NCT07047144
Added 2 new sites: Atlanta, Georgia, Leeds, United Kingdom

Added 2 new sites: Atlanta, Georgia, Leeds, United Kingdom

Enrollment UpdatePulmonary Arterial HypertensionMay 4The Hospital for Sick ChildrenNCT05636397
Target enrollment decreased from 60 to 36

Target enrollment decreased from 60 to 36

Enrollment UpdatePulmonary Arterial HypertensionMay 4Tempus AINCT06911632
Target enrollment decreased from 1000 to 900

Target enrollment decreased from 1000 to 900

eligibility_changePulmonary Arterial HypertensionMay 4Tempus AINCT06911632
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 4Merck Sharp & Dohme LLCNCT07218029
Added 8 new sites: Pittsburgh, Pennsylvania, Providence, Rhode Island, Charleston, South Carolina and 5 more

Added 8 new sites: Pittsburgh, Pennsylvania, Providence, Rhode Island, Charleston, South Carolina and 5 more

eligibility_changePulmonary Arterial HypertensionMay 4The Hospital for Sick ChildrenNCT05636397
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 4Insmed IncorporatedNCT07179380
Added 35 new sites: New Lambton Heights, Australia, Innsbruck, Austria, Leuven, Belgium and 32 more

Added 35 new sites: New Lambton Heights, Australia, Innsbruck, Austria, Leuven, Belgium and 32 more

sites_addedPrimary Biliary CholangitisMay 4Gilead SciencesNCT03301506
Added 1 new site: Saint Petersburg, Russia

Added 1 new site: Saint Petersburg, Russia

Enrollment UpdatePrimary Biliary CholangitisMay 4Gilead SciencesNCT03301506
Target enrollment decreased from 500 to 340

Target enrollment decreased from 500 to 340

sites_addedPhenylketonuriaMay 4Otsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

eligibility_changePhenylketonuriaMay 4Otsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 4Novartis PharmaceuticalsNCT06439082
Added 1 new site: Masaka, Uganda

Added 1 new site: Masaka, Uganda

sites_addedShort Bowel SyndromeMay 4Hanmi Pharmaceutical Company LimitedNCT04775706
Added 1 new site: Salford, United Kingdom

Added 1 new site: Salford, United Kingdom

sites_addedPulmonary Arterial HypertensionMay 4Regeneron PharmaceuticalsNCT07318597
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedPulmonary Arterial HypertensionMay 4Novartis PharmaceuticalsNCT06649110
Added 54 new sites: Scottsdale, Arizona, Orange, California, Orlando, Florida and 51 more

Added 54 new sites: Scottsdale, Arizona, Orange, California, Orlando, Florida and 51 more

sites_addedPulmonary Arterial HypertensionMay 4PfizerNCT07073820
Added 1 new site: Auchenflower, Australia

Added 1 new site: Auchenflower, Australia

sites_addedProgressive Familial Intrahepatic CholestasisMay 4IpsenNCT07185919
Added 1 new site: Gyeongsang, South Korea

Added 1 new site: Gyeongsang, South Korea

sites_addedPrimary Biliary CholangitisMay 4IpsenNCT06016842
Added 3 new sites: Guadalajara, Mexico, Monterrey, Mexico, Chiang Mai, Thailand

Added 3 new sites: Guadalajara, Mexico, Monterrey, Mexico, Chiang Mai, Thailand

sites_addedPrimary Biliary CholangitisMay 4Gilead SciencesNCT06051617
Added 6 new sites: Bobigny, France, Beersheba, Israel, Tel Aviv, Israel and 3 more

Added 6 new sites: Bobigny, France, Beersheba, Israel, Tel Aviv, Israel and 3 more

sites_addedPompe DiseaseMay 4Genzyme, a Sanofi CompanyNCT00231400
Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

sites_addedPolycythemia VeraMay 4Novartis PharmaceuticalsNCT07357727
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedPolycythemia VeraMay 4Disc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

sites_addedPolycythemia VeraMay 4National Cancer Institute (NCI)NCT06661915
Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

sites_addedPheochromocytomaMay 4Perspective TherapeuticsNCT05636618
Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

eligibility_changePheochromocytomaMay 4Perspective TherapeuticsNCT05636618
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdatePhenylketonuriaMay 4Otsuka Pharmaceutical Development & Commercialization, Inc.NCT06628128
Target enrollment increased from 45 to 240

Target enrollment increased from 45 to 240

eligibility_changePhenylketonuriaMay 4Otsuka Pharmaceutical Development & Commercialization, Inc.NCT06628128
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedParoxysmal Nocturnal HemoglobinuriaMay 4Hoffmann-La RocheNCT04434092
Reduced from 63 to 61 sites

Reduced from 63 to 61 sites

sites_addedParagangliomaMay 4Perspective TherapeuticsNCT05636618
Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

eligibility_changeParagangliomaMay 4Perspective TherapeuticsNCT05636618
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1May 3Centessa Pharmaceuticals (UK) LimitedNCT06752668
Added 8 new sites: El Paso, Texas, Léon, France, Bologna, Italy and 5 more

Added 8 new sites: El Paso, Texas, Léon, France, Bologna, Italy and 5 more

Enrollment UpdateNarcolepsy Type 1May 3Centessa Pharmaceuticals (UK) LimitedNCT06752668
Target enrollment increased from 96 to 248

Target enrollment increased from 96 to 248

sites_addedMyotonic DystrophyMay 3SanofiNCT06844214
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

eligibility_changeMyotonic DystrophyMay 3SanofiNCT06844214
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyotonic DystrophyMay 3PepGen IncNCT06667453
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisMay 3Novartis PharmaceuticalsNCT07357727
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedMyelofibrosisMay 3Disc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

sites_addedMyelofibrosisMay 3National Cancer Institute (NCI)NCT06661915
Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

sites_addedMyelofibrosisMay 3iOncturaNCT06887803
Added 4 new sites: Madrid, Spain, Boston, United Kingdom, Belfast, United Kingdom and 1 more

Added 4 new sites: Madrid, Spain, Boston, United Kingdom, Belfast, United Kingdom and 1 more

sites_addedMyelofibrosisMay 3Sumitomo Pharma America, Inc.NCT04176198
Added 1 new site: Aomori, Japan

Added 1 new site: Aomori, Japan

sites_removedMyelodysplastic SyndromesMay 3Hoffmann-La RocheNCT04434092
Reduced from 63 to 61 sites

Reduced from 63 to 61 sites

sites_addedMyelodysplastic SyndromesMay 3Disc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

eligibility_changeMultiple System AtrophyMay 3PepGen IncNCT06667453
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple System AtrophyMay 3Ionis Pharmaceuticals, Inc.NCT04165486
Added 1 new site: Göttingen, Germany

Added 1 new site: Göttingen, Germany

sites_addedMultiple SclerosisMay 3Hoffmann-La RocheNCT05269004
Added 3 new sites: Innsbruck, Austria, Calgary, Canada, Stockholm, Sweden

Added 3 new sites: Innsbruck, Austria, Calgary, Canada, Stockholm, Sweden

sites_addedMultiple SclerosisMay 3Hoffmann-La RocheNCT06780150
Added 6 new sites: Amberg, Germany, Celle, Germany, Frankfurt, Germany and 3 more

Added 6 new sites: Amberg, Germany, Celle, Germany, Frankfurt, Germany and 3 more

sites_removedMultiple SclerosisMay 3Hoffmann-La RocheNCT04544436
Reduced from 122 to 120 sites

Reduced from 122 to 120 sites

sites_addedMultiple SclerosisMay 3Novartis PharmaceuticalsNCT06846281
Added 4 new sites: Orlando, Florida, Las Vegas, Nevada, Overpelt, Belgium and 1 more

Added 4 new sites: Orlando, Florida, Las Vegas, Nevada, Overpelt, Belgium and 1 more

Enrollment UpdateMucopolysaccharidosis Type IMay 3REGENXBIO Inc.NCT06103487
Target enrollment increased from 7 to 21

Target enrollment increased from 7 to 21

sites_addedMixed Connective Tissue DiseaseMay 3Memorial Sloan Kettering Cancer CenterNCT05985161
Added 1 new site: Fort Worth, Texas

Added 1 new site: Fort Worth, Texas

Enrollment UpdateMesotheliomaMay 3National Cancer Institute (NCI)NCT00242723
Target enrollment increased from 1310 to 1559

Target enrollment increased from 1310 to 1559

eligibility_changeMesotheliomaMay 3National Cancer Institute (NCI)NCT00242723
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaMay 3Perspective TherapeuticsNCT06710756
Added 4 new sites: Miami, Florida, Lexington, Kentucky, Columbus, Ohio and 1 more

Added 4 new sites: Miami, Florida, Lexington, Kentucky, Columbus, Ohio and 1 more

sites_addedMerkel Cell CarcinomaMay 3University of ArizonaNCT06814496
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

eligibility_changeMerkel Cell CarcinomaMay 3Memorial Sloan Kettering Cancer CenterNCT06021626
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaMay 3Merck Sharp & Dohme LLCNCT07302347
Added 1 new site: Sapporo, Japan

Added 1 new site: Sapporo, Japan

sites_addedMembranous NephropathyMay 2Climb Bio, Inc.NCT07096843
Added 12 new sites: Temuco, Chile, Valdivia, Chile, Santiago, Chile and 9 more

Added 12 new sites: Temuco, Chile, Valdivia, Chile, Santiago, Chile and 9 more

sites_addedMedullary Thyroid CancerMay 2University of ArizonaNCT06814496
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedLight Chain AmyloidosisMay 2Regeneron PharmaceuticalsNCT06292780
Added 1 new site: Athens, Greece

Added 1 new site: Athens, Greece

sites_addedLambert-Eaton Myasthenic SyndromeMay 2argenxNCT06078553
Added 1 new site: Kodaira, Japan

Added 1 new site: Kodaira, Japan

sites_addedIgA NephropathyMay 2TakedaNCT06963827
Added 6 new sites: Surprise, Arizona, Riverview, Florida, East Providence, Rhode Island and 3 more

Added 6 new sites: Surprise, Arizona, Riverview, Florida, East Providence, Rhode Island and 3 more

sites_addedIgA NephropathyMay 2BiogenNCT06935357
Added 2 new sites: Pátrai, Greece, Taoyuan, Taiwan

Added 2 new sites: Pátrai, Greece, Taoyuan, Taiwan

eligibility_changeIgA NephropathyMay 2BiogenNCT06935357
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyMay 2Novartis PharmaceuticalsNCT06797518
Added 2 new sites: Córdoba, Argentina, Riyadh, Saudi Arabia

Added 2 new sites: Córdoba, Argentina, Riyadh, Saudi Arabia

sites_addedIdiopathic Pulmonary FibrosisMay 2Avalyn Pharma Inc.NCT06951217
Added 11 new sites: Birmingham, Alabama, Los Angeles, California, Baltimore, Maryland and 8 more

Added 11 new sites: Birmingham, Alabama, Los Angeles, California, Baltimore, Maryland and 8 more

sites_addedHypoparathyroidismMay 2Ascendis Pharma Bone Diseases A/SNCT07081997
Added 1 new site: Spokane Valley, Washington

Added 1 new site: Spokane Valley, Washington

sites_addedHuntington DiseaseMay 2University College, LondonNCT02855476
Added 1 new site: Lisbon, Portugal

Added 1 new site: Lisbon, Portugal

eligibility_changeHuntington DiseaseMay 2University of IowaNCT05707663
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseMay 2Novartis PharmaceuticalsNCT07326709
Added 6 new sites: Albany, New York, Houston, Texas, Seattle, Washington and 3 more

Added 6 new sites: Albany, New York, Houston, Texas, Seattle, Washington and 3 more

Enrollment UpdateHomozygous Familial HypercholesterolemiaMay 2Novartis PharmaceuticalsNCT05682378
Target enrollment increased from 131 to 195

Target enrollment increased from 131 to 195

eligibility_changeHomozygous Familial HypercholesterolemiaMay 2Novartis PharmaceuticalsNCT05682378
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaMay 2National Cancer Institute (NCI)NCT02003222
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 2Hoffmann-La RocheNCT06806033
Added 1 new site: Panorama City, California

Added 1 new site: Panorama City, California

Enrollment UpdateHodgkin LymphomaMay 2AstraZenecaNCT04594642
Target enrollment decreased from 317 to 227

Target enrollment decreased from 317 to 227

sites_addedHereditary Hemorrhagic TelangiectasiaMay 2Alnylam PharmaceuticalsNCT06659640
Added 8 new sites: Birmingham, Alabama, Gainesville, Florida, Indianapolis, Indiana and 5 more

Added 8 new sites: Birmingham, Alabama, Gainesville, Florida, Indianapolis, Indiana and 5 more

sites_addedGastrointestinal Stromal TumorMay 1AstraZenecaNCT07124000
Added 7 new sites: Bullhead City, Arizona, Boulder, Colorado, St. Petersburg, Florida and 4 more

Added 7 new sites: Bullhead City, Arizona, Boulder, Colorado, St. Petersburg, Florida and 4 more

sites_addedGastrointestinal Stromal TumorMay 1Deciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Frankfurt, Germany

Added 1 new site: Frankfurt, Germany

sites_addedHereditary AngioedemaMay 1Ionis Pharmaceuticals, Inc.NCT07298447
Added 1 new site: Warsaw, Poland

Added 1 new site: Warsaw, Poland

Enrollment UpdateGranulomatosis with PolyangiitisMay 1Imperial College LondonNCT06230354
Target enrollment decreased from 66 to 42

Target enrollment decreased from 66 to 42

eligibility_changeGranulomatosis with PolyangiitisMay 1Imperial College LondonNCT06230354
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGlycogen Storage Disease Type IIMay 1Genzyme, a Sanofi CompanyNCT00231400
Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

sites_addedGeographic AtrophyMay 1Kriya Therapeutics, Inc.NCT06765980
Added 1 new site: Tel Aviv, Israel

Added 1 new site: Tel Aviv, Israel

sites_addedFocal Segmental GlomerulosclerosisMay 1Boehringer IngelheimNCT07220083
Added 7 new sites: Denver, Colorado, Washington D.C., District of Columbia, Kalamazoo, Michigan and 4 more

Added 7 new sites: Denver, Colorado, Washington D.C., District of Columbia, Kalamazoo, Michigan and 4 more

sites_addedFabry DiseaseMay 1Genzyme, a Sanofi CompanyNCT00196742
Added 9 new sites: Kansas City, Kansas, Grand Rapids, Michigan, Minneapolis, Minnesota and 6 more

Added 9 new sites: Kansas City, Kansas, Grand Rapids, Michigan, Minneapolis, Minnesota and 6 more

sites_addedFabry DiseaseMay 1Amicus TherapeuticsNCT06906367
Added 1 new site: New York, New York

Added 1 new site: New York, New York