Rare Disease Trial Updates: July 2026

In July 2026, Trial Friend tracked 873 rare disease clinical trial updates across 144 conditions, including 8 FDA-related actions and 217 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 ChangeGaucher DiseaseJul 31Yuhan CorporationNCT06517914
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 30Father Flanagan's Boys' HomeNCT06586437
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 30Karen Ballen, MDNCT05233618
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 30Yonsei UniversityNCT06601777
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 30Galderma R&DNCT07074977
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 30West China HospitalNCT07657520
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJul 30Seattle Institute for Biomedical and Clinical ResearchNCT04049331
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJul 30Vir Biotechnology, Inc.NCT07128550
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 30Taiho Oncology, Inc.NCT06506955
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCerebral AdrenoleukodystrophyJul 30Minoryx Therapeutics, S.L.NCT05819866
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Akcea TherapeuticsNCT04850105
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJul 29
FDA Licenses First-Ever Freeze-Dried Plasma Product in the U.S.

FDA today licensed Ezplaz Freeze Dried Plasma (FDP), making it the first freeze-dried plasma product licensed for use in the United States. Ezplaz is intended for transfusion in adult patients who need plasma and for whom other plasma products are not available.

Status ChangeAngelman SyndromeJul 29MavriX Bio, LLCNCT07181837
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeAmyotrophic Lateral SclerosisJul 29Zydus Therapeutics Inc.NCT07023835
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeSystemic MastocytosisJul 28MacroGenicsNCT05362773
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 28University of California, San FranciscoNCT07064941
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 28Institute of Hematology & Blood Diseases Hospital, ChinaNCT06931912
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTay-Sachs DiseaseJul 28Azafaros B.V.NCT07082543
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJul 28City of Hope Medical CenterNCT05384756
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePulmonary Arterial HypertensionJul 27Pulnovo Medical (Wuxi) Co., Ltd.NCT05824923
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJul 27Tenax Therapeutics, Inc.NCT05983250
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick Disease Type CJul 26Azafaros B.V.NCT07082543
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick DiseaseJul 26Azafaros B.V.NCT07082543
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisJul 26PharmaEssentiaNCT06468033
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJul 26Karen Ballen, MDNCT05233618
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJul 26Ossium Health, Inc.NCT05589896
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJul 26University of RochesterNCT03739502
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 26Zura Bio IncNCT06843239
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisJul 25European Myeloma Network B.V.NCT06649695
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLambert-Eaton Myasthenic SyndromeJul 25argenxNCT06436742
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgG4-Related DiseaseJul 25Stanford UniversityNCT07025330
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseJul 25University of VirginiaNCT07213648
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 25University College, LondonNCT05004064
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 25Academic and Community Cancer Research UnitedNCT04626791
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 25David Bond, MDNCT05821088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFacioscapulohumeral Muscular DystrophyJul 24Epicrispr Biotechnologies, Inc.NCT06907875
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFriedreich AtaxiaJul 24Institut National de la Santé Et de la Recherche Médicale, FranceNCT05874388
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal Segmental GlomerulosclerosisJul 24Otsuka Pharmaceutical Development & Commercialization, Inc.NCT06799520
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal EpilepsyJul 24Ohio State UniversityNCT05032105
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23UConn HealthNCT04376333
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJul 23Peking University Third HospitalNCT06014034
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23GlaxoSmithKlineNCT04954859
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Nevro CorpNCT05301816
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Dartmouth-Hitchcock Medical CenterNCT03966872
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Blueprint Medicines CorporationNCT06931405
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisJul 23Mayo ClinicNCT07156357
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEhlers-Danlos SyndromeJul 23DeltaHealth Hospital · ShanghaiNCT07672210
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeDuchenne Muscular DystrophyJul 23REGENXBIO Inc.NCT05693142
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Atea Pharmaceuticals, Inc.NCT07037277
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Nocion TherapeuticsNCT06504446
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Zimmer BiometNCT05787821
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 23Nanfang Hospital, Southern Medical UniversityNCT06653413
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeChronic Graft-versus-Host DiseaseJul 23Novartis PharmaceuticalsNCT05677451
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJul 22
FDA Announces First Participant Selected for TEMPO for Digital Health Devices Pilot

The U.S. Food and Drug Administration today announced the first manufacturer selected for the Technology-Enabled Meaningful Patient Outcomes (TEMPO) for Digital Health Devices Pilot.

FDA NewsJul 22
FDA Takes Further Steps to Remove Outdated Authorizations for Color Additives in Food

The U.S. Food and Drug Administration today announced two actions targeting petroleum-based color additives in food, continuing the agency’s broader efforts to Make America Healthy Again.

Status ChangeBecker Muscular DystrophyJul 22REGENXBIO Inc.NCT05693142
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisJul 22Merit E. Cudkowicz, MDNCT07410806
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisJul 22Merit E. Cudkowicz, MDNCT04297683
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 21Bioray LaboratoriesNCT05577312
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinocerebellar AtaxiaJul 21Institut National de la Santé Et de la Recherche Médicale, FranceNCT05874388
No longer enrolling new participants

No longer enrolling new participants

Status ChangeVascular Ehlers-Danlos SyndromeJul 21DeltaHealth Hospital · ShanghaiNCT07672210
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeSystemic Juvenile Idiopathic ArthritisJul 21Eli Lilly and CompanyNCT04088396
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJul 20University of RochesterNCT06477640
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycystic Kidney DiseaseJul 20GlaxoSmithKlineNCT06734234
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParoxysmal Nocturnal HemoglobinuriaJul 20Omeros CorporationNCT06298955
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyotonic DystrophyJul 19Wake Forest University Health SciencesNCT05854433
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMyelofibrosisJul 19Karen Ballen, MDNCT05233618
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJul 19Omeros CorporationNCT06298955
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 19Hoffmann-La RocheNCT07483450
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 19EDMUS FoundationNCT05622643
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 19Rush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaJul 19UNICANCERNCT05078047
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMarfan SyndromeJul 18DeltaHealth Hospital · ShanghaiNCT07672210
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeLight Chain AmyloidosisJul 18Nexcella Inc.NCT06097832
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLennox-Gastaut SyndromeJul 18Longboard PharmaceuticalsNCT06719141
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisJul 18Daewoong Pharmaceutical Co. LTD.NCT05389215
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisJul 18Boehringer IngelheimNCT06241560
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 18Weill Medical College of Cornell UniversityNCT05934838
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 18University of PennsylvaniaNCT05989204
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 18University of UtahNCT06300528
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Hemorrhagic TelangiectasiaJul 18Alnylam PharmaceuticalsNCT06659640
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaJul 17Merck Sharp & Dohme LLCNCT06456346
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalJul 17
FDA Approves First Oral PCSK9 Inhibitor to Lower LDL Cholesterol in Adults with High Cholesterol

The U.S. Food and Drug Administration approved Lipfendra (enlicitide), the first oral inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9), as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C), or “bad” cholesterol, in adults with hypercholesterolemi

FDA DesignationHomocystinuriaJul 17
FDA grants orphan drug designation to betaine for Homocystinuria

The FDA granted orphan drug designation to betaine, developed by Cycle Pharmaceuticals Ltd., on 2026-07-17 for the treatment of homocystinuria. 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 ChangeFragile X SyndromeJul 17Rush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16Albert Einstein College of MedicineNCT05039554
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16Mercator MedSystems, Inc.NCT04858776
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16Fred Hutchinson Cancer CenterNCT04431479
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16National Center for Complementary and Integrative Health (NCCIH)NCT03952624
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16Tiziana Life Sciences LTDNCT06292923
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16Fundación FLS de Lucha Contra el Sida, las Enfermedades Infecciosas y la Promoción de la Salud y la CienciaNCT06766825
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16University of Maryland, BaltimoreNCT02995252
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16VA Office of Research and DevelopmentNCT04953806
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 16University Hospital, GhentNCT05277766
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJul 15
FDA Issues Emergency Use Authorization for Drug for Short-Term Prevention of New World Screwworm in Horses

The FDA today issued an Emergency Use Authorization (EUA) for Ivermectin Liquid for Horses (ivermectin oral solution) for the short-term prevention of New World screwworm (NWS) infestations (myiasis) in horses when administered within 24 hours of birth or at the time of initial wound care.

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Jul 15AstraZenecaNCT07608354
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeJul 15Rush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisJul 15Sunnybrook Health Sciences CentreNCT07193953
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUsher SyndromeJul 14Laboratoires TheaNCT06627179
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTuberous SclerosisJul 14Rush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyJul 14Novartis PharmaceuticalsNCT05335876
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRett SyndromeJul 13Rush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJul 13OrphAI TherapeuticsNCT05798923
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePhelan-McDermid SyndromeJul 13Rush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeShort Bowel SyndromeJul 13Children's Hospital of PhiladelphiaNCT05432648
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeRett SyndromeJul 13Taysha Gene Therapies, Inc.NCT05606614
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaJul 13Laboratoires TheaNCT06627179
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJul 13Tectonic TherapeuticNCT06616974
No longer enrolling new participants

No longer enrolling new participants

Status ChangeProgressive Supranuclear PalsyJul 13Beth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraJul 13FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETSNCT06506084
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyasthenia GravisJul 12Leiden University Medical CenterNCT06659627
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyJul 12Beth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyJul 12West China HospitalNCT07657520
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMultiple SclerosisJul 12University of Colorado, DenverNCT06641271
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMultiple SclerosisJul 12University of MichiganNCT06933160
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisJul 11Columbia UniversityNCT05451771
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseJul 11Beth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 11Washington University School of MedicineNCT06040320
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 11GenmabNCT05283720
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 11Reem KarmaliNCT06723457
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHodgkin LymphomaJul 11The Lymphoma Academic Research OrganisationNCT04984837
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 11Fondazione Italiana Linfomi - ETSNCT06070961
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 11ImmunityBio, Inc.NCT06334991
No longer enrolling new participants

No longer enrolling new participants

FDA NewsGastrointestinal Stromal TumorJul 10
FDA Proposes Rule to Modernize Drug Manufacturing Registration

The U.S. Food and Drug Administration today issued a proposed rule that, if finalized, would create a streamlined registration pathway for distributed manufacturing establishments that operate as a single establishment using a “hub-and-spoke” model.

Status ChangeChronic Graft-versus-Host DiseaseJul 9Juno Therapeutics, a Subsidiary of CelgeneNCT03331198
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 9Mundipharma Research LimitedNCT06794554
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 9Novartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 9Tam Anh Research InstituteNCT06638320
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeCholangiocarcinomaJul 9Nataliya UbohaNCT06048133
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyJul 9REGENXBIO Inc.NCT05683379
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDilated CardiomyopathyJul 9Assistance Publique - Hôpitaux de ParisNCT05774509
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCorticobasal DegenerationJul 9Beth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 9Avalyn Pharma Inc.NCT06329401
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 9The Hong Kong Polytechnic UniversityNCT07112911
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeCholangiocarcinomaJul 9TransThera Sciences (Nanjing), Inc.NCT05948475
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCharcot-Marie-Tooth DiseaseJul 9Elpida Therapeutics SPCNCT06151600
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBecker Muscular DystrophyJul 8REGENXBIO Inc.NCT05683379
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlagille SyndromeJul 8Albireo, an Ipsen CompanyNCT05035030
No longer enrolling new participants

No longer enrolling new participants

Status ChangeX-Linked HypophosphatemiaJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWilson DiseaseJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeVon Willebrand DiseaseJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUsher SyndromeJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUrea Cycle DisordersJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTyrosinemia Type IJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTuberous SclerosisJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 7Novartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 7Regeneron PharmaceuticalsNCT06421636
No longer enrolling new participants

No longer enrolling new participants

Status ChangeStickler SyndromeJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinocerebellar AtaxiaJul 7National Eye Institute (NEI)NCT02741440
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSjögren's SyndromeJul 7National Center for Complementary and Integrative Health (NCCIH)NCT03952624
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJul 7RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJul 7Instituto Nacional de Investigacao em Saude, AngolaNCT06417411
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSevere Combined ImmunodeficiencyJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRett SyndromeJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Langerhans Cell HistiocytosisJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrimary HyperoxaluriaJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrader-Willi SyndromeJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePompe DiseaseJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraJul 6Novartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangePhenylketonuriaJul 6RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePhelan-McDermid SyndromeJul 6Peking University First HospitalNCT07014020
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick Disease Type CJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick DiseaseJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisJul 5Novartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 5Tiziana Life Sciences LTDNCT06292923
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple Endocrine Neoplasia Type 1Jul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMucopolysaccharidosis Type VIJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMucopolysaccharidosis Type IVAJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSanfilippo SyndromeJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMucopolysaccharidosis Type IJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMethylmalonic AcidemiaJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMetachromatic LeukodystrophyJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMenkes DiseaseJul 5RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMedullary Thyroid CancerJul 5Jonsson Comprehensive Cancer CenterNCT07118176
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMarfan SyndromeJul 4IRCCS Policlinico S. DonatoNCT05700175
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMaple Syrup Urine Disease Type IIJul 4RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMaple Syrup Urine DiseaseJul 4RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLeber Congenital AmaurosisJul 4RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeKrabbe DiseaseJul 4RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHunter SyndromeJul 4RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomocystinuriaJul 4RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 4Baylor College of MedicineNCT03690011
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 4University of WashingtonNCT05455697
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary AngioedemaJul 4Astria Therapeutics, Inc.NCT06842823
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia BJul 3RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia AJul 3Novo Nordisk A/SNCT05685238
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHemophilia AJul 3RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGlycogen Storage Disease Type IIJul 3RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGeographic AtrophyJul 3Astellas Pharma IncNCT06970665
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGastrointestinal Stromal TumorJul 3IDRX, Inc., a wholly owned subsidiary of GSK, LLCNCT05489237
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGalactosemiaJul 3RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFragile X SyndromeJul 3RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal EpilepsyJul 3Biohaven Therapeutics Ltd.NCT06309966
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFanconi AnemiaJul 3Weill Medical College of Cornell UniversityNCT04613440
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 2Mirum Pharmaceuticals, Inc.NCT07298330
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 2BeOne MedicinesNCT06697184
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJul 2University of Colorado, DenverNCT04040959
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 2University of WashingtonNCT05596760
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 2BiogenNCT05531565
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 2Jonsson Comprehensive Cancer CenterNCT07118176
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 2Arvinas Inc.NCT07023731
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisJul 2Phathom Pharmaceuticals, Inc.NCT06851559
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDravet SyndromeJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCongenital Adrenal HyperplasiaJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCommon Variable ImmunodeficiencyJul 2Avni JoshiNCT05593588
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Granulomatous DiseaseJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 2BeOne MedicinesNCT06970743
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 2SanofiNCT06914908
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeCerebrotendinous XanthomatosisJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCerebral AdrenoleukodystrophyJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCDKL5 Deficiency DisorderJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCanavan DiseaseJul 2RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalSickle Cell DiseaseJul 1
FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT).

Status ChangeBecker Muscular DystrophyJul 1RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBarth SyndromeJul 1RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeJul 1RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlport SyndromeJul 1RTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBecker Muscular DystrophyJul 1BioMarin PharmaceuticalNCT06280209
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedGraves’ DiseaseJul 31Biohaven Therapeutics Ltd.NCT06980649
Added 1 new site: Herston, Australia

Added 1 new site: Herston, Australia

sites_addedGeographic AtrophyJul 31ADARx Pharmaceuticals, Inc.NCT06990269
Added 6 new sites: Walnut Creek, California, Malvern, Australia, Sydney, Australia and 3 more

Added 6 new sites: Walnut Creek, California, Malvern, Australia, Sydney, Australia and 3 more

sites_addedFocal EpilepsyJul 31Rapport Therapeutics Inc.NCT07563881
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

sites_addedFanconi AnemiaJul 31Novartis PharmaceuticalsNCT06910813
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedEssential ThrombocythemiaJul 31Prelude Therapeutics IncorporatedNCT07469891
Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

sites_addedHemophilia BJul 31CSL BehringNCT06003387
Added 3 new sites: São Paulo, Brazil, São Paulo, Brazil, Sofia, Bulgaria

Added 3 new sites: São Paulo, Brazil, São Paulo, Brazil, Sofia, Bulgaria

eligibility_changeHemophilia BJul 31CSL BehringNCT06003387
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedGraves’ DiseaseJul 31Yarrow Bioscience, Inc.NCT07682896
Reduced from 30 to 28 sites

Reduced from 30 to 28 sites

sites_addedGraves’ DiseaseJul 31Biohaven Therapeutics Ltd.NCT07661056
Added 3 new sites: Huntington Beach, California, Greenville, North Carolina, Raleigh, North Carolina

Added 3 new sites: Huntington Beach, California, Greenville, North Carolina, Raleigh, North Carolina

sites_addedGraves’ DiseaseJul 31Immunovant Sciences GmbHNCT07286006
Added 12 new sites: South Brisbane, Australia, Sliven, Bulgaria, Smolyan, Bulgaria and 9 more

Added 12 new sites: South Brisbane, Australia, Sliven, Bulgaria, Smolyan, Bulgaria and 9 more

sites_addedGraves’ DiseaseJul 31Immunovant Sciences GmbHNCT07018323
Added 4 new sites: Santiago, Chile, Acquaviva delle Fonti, Italy, Livorno, Italy and 1 more

Added 4 new sites: Santiago, Chile, Acquaviva delle Fonti, Italy, Livorno, Italy and 1 more

sites_addedGeographic AtrophyJul 31Regeneron PharmaceuticalsNCT06541704
Added 6 new sites: Los Angeles, California, Albury, Australia, Hurstville, Australia and 3 more

Added 6 new sites: Los Angeles, California, Albury, Australia, Hurstville, Australia and 3 more

eligibility_changeFragile X SyndromeJul 31Holland Bloorview Kids Rehabilitation HospitalNCT06081348
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateEssential ThrombocythemiaJul 31Eilean Therapeutics AU Pty LtdNCT07527221
Target enrollment increased from 40 to 64

Target enrollment increased from 40 to 64

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 30Immunovant Sciences GmbHNCT07032662
Added 3 new sites: Mission Hills, California, Belo Horizonte, Brazil, Miraflores, Peru

Added 3 new sites: Mission Hills, California, Belo Horizonte, Brazil, Miraflores, Peru

eligibility_changeChronic Graft-versus-Host DiseaseJul 30VU University of AmsterdamNCT06979089
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 30Regenerative Ocular Immunobiologics LLCNCT06852768
Target enrollment increased from 1 to 15

Target enrollment increased from 1 to 15

sites_removedChronic Graft-versus-Host DiseaseJul 30Upstream Bio Inc.NCT06981078
Reduced from 323 to 242 sites

Reduced from 323 to 242 sites

eligibility_changeChronic Graft-versus-Host DiseaseJul 30University of Sao PauloNCT07429578
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 30Eilean Therapeutics AU Pty LtdNCT07527221
Target enrollment increased from 40 to 64

Target enrollment increased from 40 to 64

eligibility_changeChronic Graft-versus-Host DiseaseJul 30National Institutes of Health Clinical Center (CC)NCT06498596
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 30Shandong Suncadia Medicine Co., Ltd.NCT07419828
Target enrollment increased from 1978 to 5386

Target enrollment increased from 1978 to 5386

eligibility_changeChronic Graft-versus-Host DiseaseJul 30Shandong Suncadia Medicine Co., Ltd.NCT07419828
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJul 30Maria Joyera RodríguezNCT06334575
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 30M.A. Med Alliance S.A.NCT05946629
Added 27 new sites: New Haven, Connecticut, Bradenton, Florida, St. Petersburg, Florida and 24 more

Added 27 new sites: New Haven, Connecticut, Bradenton, Florida, St. Petersburg, Florida and 24 more

sites_addedChronic Graft-versus-Host DiseaseJul 30AquaPass Medical Ltd.NCT06360380
Added 6 new sites: La Jolla, California, San Francisco, California, Minneapolis, Minnesota and 3 more

Added 6 new sites: La Jolla, California, San Francisco, California, Minneapolis, Minnesota and 3 more

eligibility_changeChronic Graft-versus-Host DiseaseJul 30AquaPass Medical Ltd.NCT06360380
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 30BeOne MedicinesNCT07277231
Added 25 new sites: Palo Alto, California, Chicago, Illinois, São Paulo, Brazil and 22 more

Added 25 new sites: Palo Alto, California, Chicago, Illinois, São Paulo, Brazil and 22 more

eligibility_changeChronic Graft-versus-Host DiseaseJul 30National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT06092333
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaJul 30InnoPharmax Inc.NCT06622057
Added 1 new site: Tucson, Arizona

Added 1 new site: Tucson, Arizona

sites_addedDravet SyndromeJul 30Longboard PharmaceuticalsNCT06660394
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

eligibility_changeDermatomyositisJul 30Allogene TherapeuticsNCT07085104
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisJul 30Cartesian TherapeuticsNCT07089121
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

sites_addedCongenital Adrenal HyperplasiaJul 30Crinetics Pharmaceuticals Inc.NCT07159841
Added 6 new sites: New York, New York, Porto Alegre, Brazil, Ribeirão Preto, Brazil and 3 more

Added 6 new sites: New York, New York, Porto Alegre, Brazil, Ribeirão Preto, Brazil and 3 more

sites_addedCongenital Adrenal HyperplasiaJul 30Crinetics Pharmaceuticals Inc.NCT07144163
Added 8 new sites: Baltimore, Maryland, Fortaleza, Brazil, Porto Alegre, Brazil and 5 more

Added 8 new sites: Baltimore, Maryland, Fortaleza, Brazil, Porto Alegre, Brazil and 5 more

phase_changeChronic Graft-versus-Host DiseaseJul 30University of ChicagoNCT06094491
Moved from Phase 2 to N/A

Moved from Phase 2 to N/A

sites_addedChronic Graft-versus-Host DiseaseJul 30BioPoly LLCNCT06915363
Added 3 new sites: Indianapolis, Indiana, Rochester, Minnesota, Chesterfield, Missouri

Added 3 new sites: Indianapolis, Indiana, Rochester, Minnesota, Chesterfield, Missouri

eligibility_changeChronic Graft-versus-Host DiseaseJul 30National Cancer Institute (NCI)NCT06364423
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 30Hoffmann-La RocheNCT07282574
Added 12 new sites: Ulm, Germany, Düsseldorf, Germany, Hamburg, Germany and 9 more

Added 12 new sites: Ulm, Germany, Düsseldorf, Germany, Hamburg, Germany and 9 more

sites_addedChronic Graft-versus-Host DiseaseJul 30SanofiNCT05263206
Added 1 new site: Reims, France

Added 1 new site: Reims, France

sites_addedCharcot-Marie-Tooth DiseaseJul 30Novartis PharmaceuticalsNCT07140614
Added 1 new site: Montreal, Canada

Added 1 new site: Montreal, Canada

sites_removedCerebral AdrenoleukodystrophyJul 30Minoryx Therapeutics, S.L.NCT05819866
Reduced from 13 to 11 sites

Reduced from 13 to 11 sites

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Yale UniversityNCT07062848
Added 2 new sites: Chicago, Illinois, The Bronx, New York

Added 2 new sites: Chicago, Illinois, The Bronx, New York

sites_addedAngelman SyndromeJul 29Ionis Pharmaceuticals, Inc.NCT06914609
Added 2 new sites: Los Angeles, California, Heidelberg, Australia

Added 2 new sites: Los Angeles, California, Heidelberg, Australia

sites_addedAmyotrophic Lateral SclerosisJul 29PrileniaNCT07322003
Added 9 new sites: Edmonton, Canada, Hamilton, Canada, Lille, France and 6 more

Added 9 new sites: Edmonton, Canada, Hamilton, Canada, Lille, France and 6 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Novo Nordisk A/SNCT07207811
Added 3 new sites: Genk, Belgium, Jinan, China, Guangzhou, China

Added 3 new sites: Genk, Belgium, Jinan, China, Guangzhou, China

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Alnylam PharmaceuticalsNCT07223203
Added 12 new sites: Houston, Texas, Woolloongabba, Australia, Ribeirão Preto, Brazil and 9 more

Added 12 new sites: Houston, Texas, Woolloongabba, Australia, Ribeirão Preto, Brazil and 9 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Alnylam PharmaceuticalsNCT07052903
Added 7 new sites: Columbus, Ohio, Córdoba, Argentina, Binhai, China and 4 more

Added 7 new sites: Columbus, Ohio, Córdoba, Argentina, Binhai, China and 4 more

Enrollment UpdateATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Alnylam PharmaceuticalsNCT07052903
Target enrollment increased from 1250 to 1750

Target enrollment increased from 1250 to 1750

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29Alnylam PharmaceuticalsNCT07052903
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29AstraZenecaNCT06465810
Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

eligibility_changeAngelman SyndromeJul 29Ultragenyx Pharmaceutical IncNCT06415344
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 29PrileniaNCT07322003
Added 10 new sites: Phoenix, Arizona, Aurora, Colorado, Baltimore, Maryland and 7 more

Added 10 new sites: Phoenix, Arizona, Aurora, Colorado, Baltimore, Maryland and 7 more

sites_addedAmyotrophic Lateral SclerosisJul 29PhenoNet, Inc.NCT07142291
Added 1 new site: Hradec Králové, Czechia

Added 1 new site: Hradec Králové, Czechia

sites_addedAlpha-1 Antitrypsin DeficiencyJul 29AIRNA CorporationNCT07431112
Added 2 new sites: Cambridge, United Kingdom, Southampton, United Kingdom

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

sites_addedAlpha-1 Antitrypsin DeficiencyJul 29TakedaNCT06512454
Added 7 new sites: Charleston, South Carolina, Nashville, Tennessee, Vienna, Austria and 4 more

Added 7 new sites: Charleston, South Carolina, Nashville, Tennessee, Vienna, Austria and 4 more

eligibility_changeAlpha-1 Antitrypsin DeficiencyJul 29TakedaNCT06512454
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAdrenocortical CarcinomaJul 29National Cancer Institute (NCI)NCT06900595
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedAchondroplasiaJul 29Tyra Biosciences, IncNCT06842355
Added 3 new sites: Paris, France, Utrecht, Netherlands, Madrid, Spain

Added 3 new sites: Paris, France, Utrecht, Netherlands, Madrid, Spain

sites_addedAchondroplasiaJul 29BioMarin PharmaceuticalNCT07441876
Added 1 new site: Osaka, Japan

Added 1 new site: Osaka, Japan

eligibility_change22q11.2 Deletion SyndromeJul 29Holland Bloorview Kids Rehabilitation HospitalNCT06081348
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedWild-Type ATTR Cardiac AmyloidosisJul 28AstraZenecaNCT06465810
Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

sites_addedTurner SyndromeJul 28Ascendis Pharma A/SNCT07221851
Added 3 new sites: Paris, France, Florence, Italy, Târgu Mureş, Romania

Added 3 new sites: Paris, France, Florence, Italy, Târgu Mureş, Romania

sites_addedWild-Type ATTR Cardiac AmyloidosisJul 28Alnylam PharmaceuticalsNCT07052903
Added 7 new sites: Columbus, Ohio, Córdoba, Argentina, Binhai, China and 4 more

Added 7 new sites: Columbus, Ohio, Córdoba, Argentina, Binhai, China and 4 more

Enrollment UpdateWild-Type ATTR Cardiac AmyloidosisJul 28Alnylam PharmaceuticalsNCT07052903
Target enrollment increased from 1250 to 1750

Target enrollment increased from 1250 to 1750

eligibility_changeWild-Type ATTR Cardiac AmyloidosisJul 28Alnylam PharmaceuticalsNCT07052903
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeTuberous SclerosisJul 28Holland Bloorview Kids Rehabilitation HospitalNCT06081348
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeThalassemiaJul 28New York Medical CollegeNCT02675959
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeThalassemiaJul 28Institute of Hematology & Blood Diseases Hospital, ChinaNCT06931912
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedTay-Sachs DiseaseJul 28Azafaros B.V.NCT07082543
Reduced from 36 to 26 sites

Reduced from 36 to 26 sites

eligibility_changeSickle Cell DiseaseJul 28New York Medical CollegeNCT02675959
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateSickle Cell DiseaseJul 28City of Hope Medical CenterNCT05384756
Target enrollment increased from 2 to 20

Target enrollment increased from 2 to 20

sites_addedPulmonary Arterial HypertensionJul 27Regeneron PharmaceuticalsNCT07318597
Added 4 new sites: Halle, Germany, Mainz, Germany, Bialystok, Poland and 1 more

Added 4 new sites: Halle, Germany, Mainz, Germany, Bialystok, Poland and 1 more

sites_addedPolycystic Kidney DiseaseJul 27PYC TherapeuticsNCT06714006
Added 2 new sites: Randwick, Australia, Shatin, Hong Kong

Added 2 new sites: Randwick, Australia, Shatin, Hong Kong

sites_addedRET Fusion CancerJul 27Children's Hospital of PhiladelphiaNCT06458036
Added 1 new site: San Francisco, California

Added 1 new site: San Francisco, California

sites_removedPulmonary Arterial HypertensionJul 27ImmuneCare Biopharmaceuticals (Shanghai) Co., Ltd.NCT07593027
Reduced from 32 to 29 sites

Reduced from 32 to 29 sites

eligibility_changePulmonary Arterial HypertensionJul 27Gustavo A Heresi, MD, MSNCT06992440
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePulmonary Arterial HypertensionJul 27ImmuneCare Biopharmaceuticals (Shanghai) Co., Ltd.NCT07593027
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdatePulmonary Arterial HypertensionJul 27Merck Sharp & Dohme LLCNCT06814145
Target enrollment increased from 130 to 440

Target enrollment increased from 130 to 440

eligibility_changePulmonary Arterial HypertensionJul 27Merck Sharp & Dohme LLCNCT06814145
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdatePulmonary Arterial HypertensionJul 27Pharmosa Biopharm Inc.NCT06429930
Target enrollment increased from 32 to 40

Target enrollment increased from 32 to 40

eligibility_changePulmonary Arterial HypertensionJul 27Pharmosa Biopharm Inc.NCT06429930
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJul 27Novartis PharmaceuticalsNCT06649110
Expanded from 85 to 87 sites

Expanded from 85 to 87 sites

sites_removedPulmonary Arterial HypertensionJul 27Tenax Therapeutics, Inc.NCT05983250
Reduced from 44 to 42 sites

Reduced from 44 to 42 sites

sites_addedPolycythemia VeraJul 27Incyte CorporationNCT07441694
Added 5 new sites: New York, New York, Toronto, Canada, Halle, Germany and 2 more

Added 5 new sites: New York, New York, Toronto, Canada, Halle, Germany and 2 more

sites_addedPhenylketonuriaJul 27PTC TherapeuticsNCT06302348
Added 2 new sites: Lille, France, Paris, France

Added 2 new sites: Lille, France, Paris, France

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 27AstraZenecaNCT07457151
Added 1 new site: Busan, South Korea

Added 1 new site: Busan, South Korea

sites_removedNiemann-Pick Disease Type CJul 26Azafaros B.V.NCT07082543
Reduced from 36 to 26 sites

Reduced from 36 to 26 sites

sites_removedNiemann-Pick DiseaseJul 26Azafaros B.V.NCT07082543
Reduced from 36 to 26 sites

Reduced from 36 to 26 sites

eligibility_changeNarcolepsy Type 1Jul 26Xijing HospitalNCT07654803
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyotonic DystrophyJul 26Avidity Biosciences, Inc.NCT07008469
Added 9 new sites: Minneapolis, Minnesota, Cincinnati, Ohio, Aarhus, Denmark and 6 more

Added 9 new sites: Minneapolis, Minnesota, Cincinnati, Ohio, Aarhus, Denmark and 6 more

sites_addedMyotonic DystrophyJul 26Dyne TherapeuticsNCT07486934
Added 1 new site: Roma, Italy

Added 1 new site: Roma, Italy

sites_addedMyelofibrosisJul 26PharmaEssentiaNCT06468033
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedMyelofibrosisJul 26Incyte CorporationNCT07441694
Added 5 new sites: New York, New York, Toronto, Canada, Halle, Germany and 2 more

Added 5 new sites: New York, New York, Toronto, Canada, Halle, Germany and 2 more

sites_addedMyelofibrosisJul 26Disc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changeMyelofibrosisJul 26Disc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisJul 26Aspera Biomedicines, Inc.NCT07250646
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedMyelodysplastic SyndromesJul 26Disc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changeMyelodysplastic SyndromesJul 26Disc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJul 26TakedaNCT07422480
Added 2 new sites: Tampa, Florida, Manchester, United Kingdom

Added 2 new sites: Tampa, Florida, Manchester, United Kingdom

sites_addedMyelodysplastic SyndromesJul 26National Cancer Institute (NCI)NCT05554406
Added 1 new site: Charleston, South Carolina

Added 1 new site: Charleston, South Carolina

sites_addedMyelodysplastic SyndromesJul 26AstraZenecaNCT07457151
Added 1 new site: Busan, South Korea

Added 1 new site: Busan, South Korea

eligibility_changeMyelodysplastic SyndromesJul 26Memorial Sloan Kettering Cancer CenterNCT02677064
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJul 26Vertex Pharmaceuticals IncorporatedNCT07501702
Added 7 new sites: Chattanooga, Tennessee, Bydgoszcz, Poland, Poznan, Poland and 4 more

Added 7 new sites: Chattanooga, Tennessee, Bydgoszcz, Poland, Poznan, Poland and 4 more

sites_addedMyasthenia GravisJul 26Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 1 new site: Kocaeli, Turkey (Türkiye)

Added 1 new site: Kocaeli, Turkey (Türkiye)

sites_addedMultiple System AtrophyJul 26Teva Branded Pharmaceutical Products R&D LLCNCT06568237
Added 1 new site: Belgrade, Serbia

Added 1 new site: Belgrade, Serbia

sites_addedMultiple SclerosisJul 26TG Therapeutics, Inc.NCT06433752
Expanded from 90 to 91 sites

Expanded from 90 to 91 sites

sites_removedMultiple SclerosisJul 26Hoffmann-La RocheNCT03523858
Reduced from 124 to 120 sites

Reduced from 124 to 120 sites

phase_changeMultiple SclerosisJul 26MGH Institute of Health ProfessionsNCT06136728
Moved from Phase 4 to Phase 3

Moved from Phase 4 to Phase 3

sites_addedMultiple SclerosisJul 26Zura Bio IncNCT06843239
Added 9 new sites: Los Angeles, California, Debrecen, Hungary, Lodz, Poland and 6 more

Added 9 new sites: Los Angeles, California, Debrecen, Hungary, Lodz, Poland and 6 more

sites_addedMultiple SclerosisJul 26Hoffmann-La RocheNCT07282574
Added 12 new sites: Ulm, Germany, Düsseldorf, Germany, Hamburg, Germany and 9 more

Added 12 new sites: Ulm, Germany, Düsseldorf, Germany, Hamburg, Germany and 9 more

eligibility_changeMixed Connective Tissue DiseaseJul 26University of FloridaNCT07042243
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMixed Connective Tissue DiseaseJul 26Terry L. WahlsNCT07180537
Target enrollment increased from 200 to 400

Target enrollment increased from 200 to 400

sites_addedMesotheliomaJul 26IDEAYA BiosciencesNCT07277413
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

eligibility_changeMesotheliomaJul 26National Cancer Institute (NCI)NCT00242723
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaJul 26University of ArizonaNCT06814496
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyJul 26BiogenNCT06962800
Added 1 new site: Shanghai, China

Added 1 new site: Shanghai, China

sites_addedMembranous NephropathyJul 26Vertex Pharmaceuticals IncorporatedNCT07204275
Added 6 new sites: Shenyang, China, Prague, Czechia, Cork, Ireland and 3 more

Added 6 new sites: Shenyang, China, Prague, Czechia, Cork, Ireland and 3 more

eligibility_changeMedullary Thyroid CancerJul 26University of ArizonaNCT06814496
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMedullary Thyroid CancerJul 26University of PennsylvaniaNCT04877613
Target enrollment decreased from 18 to 9

Target enrollment decreased from 18 to 9

eligibility_changeLight Chain AmyloidosisJul 25Gracell Biotechnologies (Shanghai) Co., Ltd.NCT07250269
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLambert-Eaton Myasthenic SyndromeJul 25argenxNCT06078553
Added 1 new site: São Paulo, Brazil

Added 1 new site: São Paulo, Brazil

sites_addedIgA NephropathyJul 25Novartis PharmaceuticalsNCT06858319
Added 3 new sites: Houston, Texas, Catamarca, Argentina, Toronto, Canada

Added 3 new sites: Houston, Texas, Catamarca, Argentina, Toronto, Canada

sites_addedIgA NephropathyJul 25BiogenNCT06935357
Added 3 new sites: Shanghai, China, Prague, Czechia, Modena, Italy

Added 3 new sites: Shanghai, China, Prague, Czechia, Modena, Italy

sites_addedIdiopathic Pulmonary FibrosisJul 25Rein TherapeuticsNCT06968845
Added 9 new sites: Indianapolis, Indiana, New York, New York, Cincinnati, Ohio and 6 more

Added 9 new sites: Indianapolis, Indiana, New York, New York, Cincinnati, Ohio and 6 more

sites_addedIdiopathic Pulmonary FibrosisJul 25Contineum TherapeuticsNCT07284459
Added 3 new sites: Córdoba, Argentina, Camperdown, Australia, London, United Kingdom

Added 3 new sites: Córdoba, Argentina, Camperdown, Australia, London, United Kingdom

sites_addedHuntington DiseaseJul 25Skyhawk Therapeutics, Inc.NCT07537075
Added 6 new sites: New Lambton Heights, Australia, Herston, Australia, Nedlands, Australia and 3 more

Added 6 new sites: New Lambton Heights, Australia, Herston, Australia, Nedlands, Australia and 3 more

sites_addedHomozygous Familial HypercholesterolemiaJul 25CRISPR Therapeutics AGNCT07491172
Added 19 new sites: Hialeah, Florida, Jacksonville, Florida, Orlando, Florida and 16 more

Added 19 new sites: Hialeah, Florida, Jacksonville, Florida, Orlando, Florida and 16 more

sites_addedHomozygous Familial HypercholesterolemiaJul 25Novartis PharmaceuticalsNCT06597006
Added 2 new sites: Salt Lake City, Utah, Johannesburg, South Africa

Added 2 new sites: Salt Lake City, Utah, Johannesburg, South Africa

sites_addedHodgkin LymphomaJul 25AstraZenecaNCT06549595
Added 9 new sites: Lubbock, Texas, Jena, Germany, Gdansk, Poland and 6 more

Added 9 new sites: Lubbock, Texas, Jena, Germany, Gdansk, Poland and 6 more

sites_addedHodgkin LymphomaJul 25Merck Sharp & Dohme LLCNCT07519772
Added 4 new sites: Denver, Colorado, Candiolo, Italy, Badalona, Spain and 1 more

Added 4 new sites: Denver, Colorado, Candiolo, Italy, Badalona, Spain and 1 more

eligibility_changeHodgkin LymphomaJul 25Hope Biosciences LLCNCT05951777
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 25St. Jude Children's Research HospitalNCT06390319
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedHodgkin LymphomaJul 25Tempus AINCT07154823
Added 3 new sites: Iowa City, Iowa, St Louis, Missouri, Columbus, Ohio

Added 3 new sites: Iowa City, Iowa, St Louis, Missouri, Columbus, Ohio

sites_removedHodgkin LymphomaJul 25Academic and Community Cancer Research UnitedNCT04626791
Reduced from 7 to 3 sites

Reduced from 7 to 3 sites

sites_addedHodgkin LymphomaJul 25University College, LondonNCT04685616
Added 3 new sites: Basingstoke, United Kingdom, Cardiff, United Kingdom, Nottingham, United Kingdom

Added 3 new sites: Basingstoke, United Kingdom, Cardiff, United Kingdom, Nottingham, United Kingdom

sites_addedHodgkin LymphomaJul 25InSilico Medicine Hong Kong LimitedNCT06445517
Added 2 new sites: Nanchang, China, Tianjin, China

Added 2 new sites: Nanchang, China, Tianjin, China

sites_addedHodgkin LymphomaJul 25National Cancer Institute (NCI)NCT06124157
Added 3 new sites: New Orleans, Louisiana, Neptune City, New Jersey, Edmonton, Canada

Added 3 new sites: New Orleans, Louisiana, Neptune City, New Jersey, Edmonton, Canada

eligibility_changeHodgkin LymphomaJul 25Union Hospital, Tongji Medical College, Huazhong University of Science and TechnologyNCT07101705
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJul 25GC Cell CorporationNCT06699771
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJul 25Chinese PLA General HospitalNCT06471738
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 25HutchmedNCT07409428
Added 11 new sites: Chongqing, China, Guangzhou, China, Kunming, China and 8 more

Added 11 new sites: Chongqing, China, Guangzhou, China, Kunming, China and 8 more

sites_addedHodgkin LymphomaJul 25Treeline Biosciences, Inc.NCT07082803
Added 2 new sites: New York, New York, Philadelphia, Pennsylvania

Added 2 new sites: New York, New York, Philadelphia, Pennsylvania

sites_addedHodgkin LymphomaJul 25Merck Sharp & Dohme LLCNCT07570173
Added 2 new sites: Copenhagen, Denmark, Tel Aviv, Israel

Added 2 new sites: Copenhagen, Denmark, Tel Aviv, Israel

eligibility_changeHodgkin LymphomaJul 25Mayo ClinicNCT03017820
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 25National Cancer Institute (NCI)NCT04840602
Added 1 new site: Irvine, California

Added 1 new site: Irvine, California

eligibility_changeHodgkin LymphomaJul 25Yale UniversityNCT06898905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 25AstraZenecaNCT06137144
Added 3 new sites: Birtinya, Australia, Dijon, France, Bunkyō City, Japan

Added 3 new sites: Birtinya, Australia, Dijon, France, Bunkyō City, Japan

sites_addedHodgkin LymphomaJul 25Canadian Cancer Trials GroupNCT07341191
Added 1 new site: Edmonton, Canada

Added 1 new site: Edmonton, Canada

sites_addedHodgkin LymphomaJul 25City of Hope Medical CenterNCT05861050
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_removedHereditary AngioedemaJul 25Pharvaris Netherlands B.V.NCT05396105
Reduced from 63 to 60 sites

Reduced from 63 to 60 sites

sites_addedHereditary AngioedemaJul 25OctapharmaNCT06361537
Added 2 new sites: Bangalore, India, Patna, India

Added 2 new sites: Bangalore, India, Patna, India

sites_addedGiant Cell ArteritisJul 24Novartis PharmaceuticalsNCT06331312
Added 5 new sites: The Woodlands, Texas, Cholet, France, Le Mans, France and 2 more

Added 5 new sites: The Woodlands, Texas, Cholet, France, Le Mans, France and 2 more

sites_addedFocal EpilepsyJul 24Rapport Therapeutics Inc.NCT07563881
Added 1 new site: Hackensack, New Jersey

Added 1 new site: Hackensack, New Jersey

sites_addedHemophilia BJul 24CSL BehringNCT07080905
Added 12 new sites: Orange, California, Gainesville, Florida, Atlanta, Georgia and 9 more

Added 12 new sites: Orange, California, Gainesville, Florida, Atlanta, Georgia and 9 more

eligibility_changeHemophilia BJul 24CSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseJul 24AmgenNCT07438405
Added 2 new sites: Orland Park, Illinois, Morgantown, West Virginia

Added 2 new sites: Orland Park, Illinois, Morgantown, West Virginia

sites_addedGraves’ DiseaseJul 24Biohaven Therapeutics Ltd.NCT07661056
Added 6 new sites: Champaign, Illinois, Las Vegas, Nevada, Asheville, North Carolina and 3 more

Added 6 new sites: Champaign, Illinois, Las Vegas, Nevada, Asheville, North Carolina and 3 more

eligibility_changeGlycogen Storage Disease Type IIJul 24AskBio IncNCT07282847
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyJul 24ONL TherapeuticsNCT06659445
Added 4 new sites: Glendale, California, Greenville, North Carolina, McAllen, Texas and 1 more

Added 4 new sites: Glendale, California, Greenville, North Carolina, McAllen, Texas and 1 more

sites_addedGeographic AtrophyJul 24i-Lumen Scientific AUS PTY LTDNCT06662162
Added 2 new sites: Reno, Nevada, Belfast, United Kingdom

Added 2 new sites: Reno, Nevada, Belfast, United Kingdom

sites_addedGastrointestinal Stromal TumorJul 24Deciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedGastrointestinal Stromal TumorJul 24AstraZenecaNCT07124000
Added 1 new site: Farmington, New Mexico

Added 1 new site: Farmington, New Mexico

sites_addedFocal Segmental GlomerulosclerosisJul 24Boehringer IngelheimNCT07220083
Added 5 new sites: Humble, Texas, San Miguel de Tucumán, Argentina, Pleven, Bulgaria and 2 more

Added 5 new sites: Humble, Texas, San Miguel de Tucumán, Argentina, Pleven, Bulgaria and 2 more

eligibility_changeFocal Segmental GlomerulosclerosisJul 24SanofiNCT06500702
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateFocal EpilepsyJul 24Ohio State UniversityNCT05032105
Target enrollment decreased from 10 to 2

Target enrollment decreased from 10 to 2

sites_addedFocal EpilepsyJul 24Rapport Therapeutics Inc.NCT07594119
Added 2 new sites: Raleigh, North Carolina, Houston, Texas

Added 2 new sites: Raleigh, North Carolina, Houston, Texas

sites_addedEssential ThrombocythemiaJul 24Incyte CorporationNCT07441694
Added 5 new sites: New York, New York, Toronto, Canada, Halle, Germany and 2 more

Added 5 new sites: New York, New York, Toronto, Canada, Halle, Germany and 2 more

sites_addedEssential ThrombocythemiaJul 24Disc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changeEssential ThrombocythemiaJul 24Disc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaJul 24PharmaEssentiaNCT04285086
Added 2 new sites: Scottsdale, Arizona, Incheon, South Korea

Added 2 new sites: Scottsdale, Arizona, Incheon, South Korea

sites_addedChronic Graft-versus-Host DiseaseJul 23SanofiNCT07424144
Added 19 new sites: Oklahoma City, Oklahoma, Québec, Canada, Québec, Canada and 16 more

Added 19 new sites: Oklahoma City, Oklahoma, Québec, Canada, Québec, Canada and 16 more

sites_addedChronic Graft-versus-Host DiseaseJul 23Omeza, LLCNCT07161830
Added 7 new sites: Evergreen Park, Illinois, Marksville, Louisiana, Slidell, Louisiana and 4 more

Added 7 new sites: Evergreen Park, Illinois, Marksville, Louisiana, Slidell, Louisiana and 4 more

phase_changeChronic Graft-versus-Host DiseaseJul 23University of Colorado, DenverNCT06810622
Moved from Phase 2 to N/A

Moved from Phase 2 to N/A

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 23Peking University Third HospitalNCT06014034
Target enrollment increased from 50 to 80

Target enrollment increased from 50 to 80

eligibility_changeChronic Graft-versus-Host DiseaseJul 23Peking University Third HospitalNCT06014034
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedChronic Graft-versus-Host DiseaseJul 23Bellus Health Inc. - a GSK companyNCT05600777
Reduced from 252 to 242 sites

Reduced from 252 to 242 sites

sites_addedChronic Graft-versus-Host DiseaseJul 23GlaxoSmithKlineNCT04954859
Added 99 new sites: San Jose, California, Colorado Springs, Colorado, La Plata, Argentina and 96 more

Added 99 new sites: San Jose, California, Colorado Springs, Colorado, La Plata, Argentina and 96 more

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 23GlaxoSmithKlineNCT04954859
Target enrollment decreased from 450 to 360

Target enrollment decreased from 450 to 360

eligibility_changeChronic Graft-versus-Host DiseaseJul 23Novartis PharmaceuticalsNCT04573478
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 23University of Southern CaliforniaNCT02935361
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedEhlers-Danlos SyndromeJul 23DeltaHealth Hospital · ShanghaiNCT07672210
Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

sites_addedDuchenne Muscular DystrophyJul 23Catalyst Pharmaceuticals, Inc.NCT06564974
Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

sites_addedDuchenne Muscular DystrophyJul 23ItalfarmacoNCT05933057
Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

sites_addedDuchenne Muscular DystrophyJul 23REGENXBIO Inc.NCT05693142
Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

eligibility_changeDuchenne Muscular DystrophyJul 23REGENXBIO Inc.NCT05693142
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeJul 23Longboard PharmaceuticalsNCT06908226
Added 52 new sites: Palo Alto, California, San Francisco, California, Aurora, Colorado and 49 more

Added 52 new sites: Palo Alto, California, San Francisco, California, Aurora, Colorado and 49 more

eligibility_changeDiamond-Blackfan AnemiaJul 23Apriligen, Inc.NCT07476183
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisJul 23Chugai PharmaceuticalNCT06371417
Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

eligibility_changeDermatomyositisJul 23Chugai PharmaceuticalNCT06371417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisJul 23Chugai PharmaceuticalNCT06723106
Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

sites_addedDermatomyositisJul 23argenxNCT05979441
Added 2 new sites: Rozzano, Italy, Yamaguchi, Japan

Added 2 new sites: Rozzano, Italy, Yamaguchi, Japan

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 23argenxNCT06920004
Added 7 new sites: Birmingham, Alabama, Bogotá, Colombia, Medellín, Colombia and 4 more

Added 7 new sites: Birmingham, Alabama, Bogotá, Colombia, Medellín, Colombia and 4 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 23argenxNCT07091630
Added 10 new sites: Córdoba, Argentina, Rosario, Argentina, São Paulo, Brazil and 7 more

Added 10 new sites: Córdoba, Argentina, Rosario, Argentina, São Paulo, Brazil and 7 more

sites_removedChronic Graft-versus-Host DiseaseJul 23Novartis PharmaceuticalsNCT05677451
Reduced from 66 to 50 sites

Reduced from 66 to 50 sites

sites_addedChronic Graft-versus-Host DiseaseJul 23Atea Pharmaceuticals, Inc.NCT07037277
Added 1 new site: Cairo, Egypt

Added 1 new site: Cairo, Egypt

eligibility_changeChronic Graft-versus-Host DiseaseJul 23The University of Texas Medical Branch, GalvestonNCT06347796
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 23ONWARD Medical, Inc.NCT07147296
Added 1 new site: Vancouver, Canada

Added 1 new site: Vancouver, Canada

eligibility_changeChronic Graft-versus-Host DiseaseJul 23AstraZenecaNCT06594068
Eligibility criteria have been updated

Eligibility criteria have been updated

phase_changeChronic Graft-versus-Host DiseaseJul 23Genentech, Inc.NCT06603246
Moved from Phase 1, Phase 2 to Phase 1

Moved from Phase 1, Phase 2 to Phase 1

sites_addedChronic Graft-versus-Host DiseaseJul 23Curis, Inc.NCT07271667
Added 4 new sites: Rochester, Minnesota, Pittsburgh, Pennsylvania, Milan, Italy and 1 more

Added 4 new sites: Rochester, Minnesota, Pittsburgh, Pennsylvania, Milan, Italy and 1 more

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 23Nanfang Hospital, Southern Medical UniversityNCT06653413
Target enrollment increased from 600 to 2500

Target enrollment increased from 600 to 2500

sites_addedChronic Graft-versus-Host DiseaseJul 23Incyte CorporationNCT06585774
Added 3 new sites: Tucson, Arizona, San Francisco, California, Ulm, Germany

Added 3 new sites: Tucson, Arizona, San Francisco, California, Ulm, Germany

sites_addedChronic Graft-versus-Host DiseaseJul 23Blueprint Medicines CorporationNCT07143669
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedChronic Graft-versus-Host DiseaseJul 23Merck Sharp & Dohme LLCNCT05947851
Added 1 new site: Buenos Aires, Argentina

Added 1 new site: Buenos Aires, Argentina

eligibility_changeChronic Graft-versus-Host DiseaseJul 23ADIR AssociationNCT06956742
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaJul 23BioNTech SENCT07186842
Added 6 new sites: Denver, Colorado, Clayton, Australia, Heidelberg, Australia and 3 more

Added 6 new sites: Denver, Colorado, Clayton, Australia, Heidelberg, Australia and 3 more

eligibility_changeCholangiocarcinomaJul 23BioNTech SENCT07186842
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedCholangiocarcinomaJul 23Eli Lilly and CompanyNCT06607185
Reduced from 47 to 45 sites

Reduced from 47 to 45 sites

sites_addedBullous PemphigoidJul 22Chugai PharmaceuticalNCT06371417
Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

eligibility_changeBullous PemphigoidJul 22Chugai PharmaceuticalNCT06371417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedBehçet DiseaseJul 22Chugai PharmaceuticalNCT06371417
Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

eligibility_changeBehçet DiseaseJul 22Chugai PharmaceuticalNCT06371417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 22Regeneron PharmaceuticalsNCT06351592
Added 3 new sites: Ulm, Germany, Zabrze, Poland, Warsaw, Poland

Added 3 new sites: Ulm, Germany, Zabrze, Poland, Warsaw, Poland

sites_addedBullous PemphigoidJul 22Chugai PharmaceuticalNCT06723106
Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

sites_addedBehçet DiseaseJul 22Chugai PharmaceuticalNCT06723106
Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

sites_addedBecker Muscular DystrophyJul 22Catalyst Pharmaceuticals, Inc.NCT06564974
Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

sites_addedBecker Muscular DystrophyJul 22Solid Biosciences Inc.NCT07160634
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

sites_addedBecker Muscular DystrophyJul 22ItalfarmacoNCT05933057
Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

sites_addedBecker Muscular DystrophyJul 22REGENXBIO Inc.NCT05693142
Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

eligibility_changeBecker Muscular DystrophyJul 22REGENXBIO Inc.NCT05693142
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 22AstraZenecaNCT06355934
Added 3 new sites: Lecce, Italy, Messina, Italy, Milan, Italy

Added 3 new sites: Lecce, Italy, Messina, Italy, Milan, Italy

eligibility_changeAplastic AnemiaJul 22Apriligen, Inc.NCT07476183
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateAmyotrophic Lateral SclerosisJul 22Merit E. Cudkowicz, MDNCT07410806
Target enrollment increased from 160 to 240

Target enrollment increased from 160 to 240

eligibility_changeAmyotrophic Lateral SclerosisJul 22Merit E. Cudkowicz, MDNCT07410806
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 22Eli Lilly and CompanyNCT07571200
Added 1 new site: London, Canada

Added 1 new site: London, Canada

sites_addedAmyotrophic Lateral SclerosisJul 22Eli Lilly and CompanyNCT07571174
Added 1 new site: London, Canada

Added 1 new site: London, Canada

sites_addedAlpha-1 Antitrypsin DeficiencyJul 22Tessera Therapeutics, Inc.NCT07227207
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedAdrenocortical CarcinomaJul 22National Cancer Institute (NCI)NCT06900595
Added 5 new sites: Dallas, Texas, Dallas, Texas, Dallas, Texas and 2 more

Added 5 new sites: Dallas, Texas, Dallas, Texas, Dallas, Texas and 2 more

Enrollment UpdateTurner SyndromeJul 21Dong-A ST Co., Ltd.NCT04798690
Target enrollment increased from 2500 to 5000

Target enrollment increased from 2500 to 5000

sites_addedThalassemiaJul 21Bioray LaboratoriesNCT05577312
Added 3 new sites: Guanzhou, China, Shenzhen, China, Nanning, China

Added 3 new sites: Guanzhou, China, Shenzhen, China, Nanning, China

sites_removedSystemic Juvenile Idiopathic ArthritisJul 21AbbVieNCT05609630
Reduced from 56 to 47 sites

Reduced from 56 to 47 sites

sites_addedVascular Ehlers-Danlos SyndromeJul 21DeltaHealth Hospital · ShanghaiNCT07672210
Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

sites_addedStargardt DiseaseJul 21Astellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Pittsburgh, Pennsylvania

Added 1 new site: Pittsburgh, Pennsylvania

eligibility_changeStargardt DiseaseJul 21Astellas Pharma Global Development, Inc.NCT07425574
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseJul 21Novartis PharmaceuticalsNCT06546670
Added 2 new sites: Philadelphia, Pennsylvania, Ankara, Turkey (Türkiye)

Added 2 new sites: Philadelphia, Pennsylvania, Ankara, Turkey (Türkiye)

sites_addedPulmonary Arterial HypertensionJul 20Tenax Therapeutics, Inc.NCT07436689
Added 2 new sites: Omaha, Nebraska, Madison, Wisconsin

Added 2 new sites: Omaha, Nebraska, Madison, Wisconsin

eligibility_changePompe DiseaseJul 20AskBio IncNCT07282847
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraJul 20Disc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changePolycythemia VeraJul 20Disc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Langerhans Cell HistiocytosisJul 20National Cancer Institute (NCI)NCT05828069
Added 1 new site: Baltimore, Maryland

Added 1 new site: Baltimore, Maryland

sites_addedPulmonary Arterial HypertensionJul 20Merck Sharp & Dohme LLCNCT07218029
Added 1 new site: São Paulo, Brazil

Added 1 new site: São Paulo, Brazil

sites_addedPulmonary Arterial HypertensionJul 20Regeneron PharmaceuticalsNCT07318597
Added 9 new sites: South Brisbane, Australia, Leuven, Belgium, Brussels, Belgium and 6 more

Added 9 new sites: South Brisbane, Australia, Leuven, Belgium, Brussels, Belgium and 6 more

sites_addedPrimary Biliary CholangitisJul 20Gilead SciencesNCT06051617
Added 2 new sites: South San Francisco, California, Petah Tikva, Israel

Added 2 new sites: South San Francisco, California, Petah Tikva, Israel

sites_addedPolycythemia VeraJul 20Novartis PharmaceuticalsNCT07357727
Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

sites_addedPolycystic Kidney DiseaseJul 20GlaxoSmithKlineNCT06734234
Added 8 new sites: Rochester, Minnesota, Ghent, Belgium, Toronto, Canada and 5 more

Added 8 new sites: Rochester, Minnesota, Ghent, Belgium, Toronto, Canada and 5 more

eligibility_changePolycystic Kidney DiseaseJul 20Otsuka Pharmaceutical Development & Commercialization, Inc.NCT04782258
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPhenylketonuriaJul 20Maze TherapeuticsNCT07694440
Added 1 new site: Indianapolis, Indiana

Added 1 new site: Indianapolis, Indiana

sites_addedNarcolepsy Type 1Jul 19Centessa Pharmaceuticals (UK) LimitedNCT07598708
Added 2 new sites: Miami, Florida, Miami Lakes, Florida

Added 2 new sites: Miami, Florida, Miami Lakes, Florida

sites_addedMyotonic DystrophyJul 19Dyne TherapeuticsNCT07486934
Added 2 new sites: Richmond, Virginia, Milan, Italy

Added 2 new sites: Richmond, Virginia, Milan, Italy

sites_addedMyelofibrosisJul 19Novartis PharmaceuticalsNCT07357727
Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

sites_addedMyelodysplastic SyndromesJul 19Regeneron PharmaceuticalsNCT05744921
Added 1 new site: Chandigarh, India

Added 1 new site: Chandigarh, India

sites_addedMyelodysplastic SyndromesJul 19National Cancer Institute (NCI)NCT05564390
Added 1 new site: Reno, Nevada

Added 1 new site: Reno, Nevada

sites_addedMyelodysplastic SyndromesJul 19M.D. Anderson Cancer CenterNCT06566742
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

eligibility_changeMyelodysplastic SyndromesJul 19M.D. Anderson Cancer CenterNCT06566742
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJul 19AstraZenecaNCT07247279
Added 1 new site: Ufa, Russia

Added 1 new site: Ufa, Russia

sites_removedMyasthenia GravisJul 19UCB Biopharma SRLNCT06149559
Reduced from 13 to 9 sites

Reduced from 13 to 9 sites

sites_addedMultiple SclerosisJul 19Novartis PharmaceuticalsNCT05344469
Added 6 new sites: Aalen, Germany, Bogen, Germany, Dillingen Saar, Germany and 3 more

Added 6 new sites: Aalen, Germany, Bogen, Germany, Dillingen Saar, Germany and 3 more

sites_addedMultiple SclerosisJul 19Zenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Livingston, New Jersey, Lublin, Poland

Added 2 new sites: Livingston, New Jersey, Lublin, Poland

sites_addedMultiple SclerosisJul 19Novartis PharmaceuticalsNCT06675864
Added 3 new sites: Mainz, Germany, Barcelona, Spain, Madrid, Spain

Added 3 new sites: Mainz, Germany, Barcelona, Spain, Madrid, Spain

sites_addedMultiple SclerosisJul 19Zenas BioPharma (USA), LLCNCT07299019
Added 5 new sites: Boca Raton, Florida, Clearwater, Florida, Indianapolis, Indiana and 2 more

Added 5 new sites: Boca Raton, Florida, Clearwater, Florida, Indianapolis, Indiana and 2 more

sites_addedMultiple SclerosisJul 19PhenoNet, Inc.NCT07142291
Added 1 new site: Málaga, Spain

Added 1 new site: Málaga, Spain

sites_removedMultiple SclerosisJul 19Hoffmann-La RocheNCT05123703
Reduced from 102 to 71 sites

Reduced from 102 to 71 sites

eligibility_changeMultiple SclerosisJul 19Hoffmann-La RocheNCT05123703
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 19Hoffmann-La RocheNCT06780150
Added 2 new sites: Lübeck, Germany, Neuruppin, Germany

Added 2 new sites: Lübeck, Germany, Neuruppin, Germany

eligibility_changeMultiple SclerosisJul 19Hoffmann-La RocheNCT06780150
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 19Novartis PharmaceuticalsNCT07225504
Added 10 new sites: Aurora, Colorado, Wellesley, Massachusetts, Dallas, Texas and 7 more

Added 10 new sites: Aurora, Colorado, Wellesley, Massachusetts, Dallas, Texas and 7 more

eligibility_changeMultiple SclerosisJul 19Novartis PharmaceuticalsNCT07225504
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMesotheliomaJul 19University College, LondonNCT05628376
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMerkel Cell CarcinomaJul 19UNICANCERNCT05078047
Reduced from 40 to 33 sites

Reduced from 40 to 33 sites

sites_addedMerkel Cell CarcinomaJul 19Neonc Technologies, Inc.NCT06047379
Added 1 new site: Boynton Beach, Florida

Added 1 new site: Boynton Beach, Florida

sites_removedMembranous NephropathyJul 19Climb Bio, Inc.NCT07096843
Reduced from 45 to 43 sites

Reduced from 45 to 43 sites

sites_addedMarfan SyndromeJul 18DeltaHealth Hospital · ShanghaiNCT07672210
Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

sites_addedLight Chain AmyloidosisJul 18Rajshekhar Chakraborty, MDNCT07110844
Added 2 new sites: Boston, Massachusetts, Milwaukee, Wisconsin

Added 2 new sites: Boston, Massachusetts, Milwaukee, Wisconsin

sites_addedLennox-Gastaut SyndromeJul 18Longboard PharmaceuticalsNCT06719141
Added 2 new sites: Porto Alegre, Brazil, Mexico City, Mexico

Added 2 new sites: Porto Alegre, Brazil, Mexico City, Mexico

sites_addedLennox-Gastaut SyndromeJul 18Longboard PharmaceuticalsNCT06908226
Added 52 new sites: Palo Alto, California, San Francisco, California, Aurora, Colorado and 49 more

Added 52 new sites: Palo Alto, California, San Francisco, California, Aurora, Colorado and 49 more

sites_addedIgA NephropathyJul 18BiogenNCT06935357
Added 1 new site: Guiyang, China

Added 1 new site: Guiyang, China

sites_addedIgA NephropathyJul 18TakedaNCT06963827
Added 1 new site: Toronto, Canada

Added 1 new site: Toronto, Canada

sites_addedIdiopathic Pulmonary FibrosisJul 18Contineum TherapeuticsNCT07284459
Added 2 new sites: San Miguel de Tucumán, Argentina, London, United Kingdom

Added 2 new sites: San Miguel de Tucumán, Argentina, London, United Kingdom

sites_addedHuntington DiseaseJul 18Novartis PharmaceuticalsNCT07326709
Added 14 new sites: Tampa, Florida, Indianapolis, Indiana, Iowa City, Iowa and 11 more

Added 14 new sites: Tampa, Florida, Indianapolis, Indiana, Iowa City, Iowa and 11 more

sites_addedHuntington DiseaseJul 18Skyhawk Therapeutics, Inc.NCT07378644
Added 9 new sites: Buenos Aires, Argentina, Belo Horizonte, Brazil, Campina Grande do Sul, Brazil and 6 more

Added 9 new sites: Buenos Aires, Argentina, Belo Horizonte, Brazil, Campina Grande do Sul, Brazil and 6 more

sites_addedHodgkin LymphomaJul 18Memorial Sloan Kettering Cancer CenterNCT07223021
Added 2 new sites: Houston, Texas, Houston, Texas

Added 2 new sites: Houston, Texas, Houston, Texas

sites_addedHodgkin LymphomaJul 18International Extranodal Lymphoma Study Group (IELSG)NCT05700149
Added 15 new sites: Rochester, Minnesota, Columbus, Ohio, Buenos Aires, Argentina and 12 more

Added 15 new sites: Rochester, Minnesota, Columbus, Ohio, Buenos Aires, Argentina and 12 more

sites_addedHodgkin LymphomaJul 18The Lymphoma Academic Research OrganisationNCT06284122
Added 68 new sites: Graz, Austria, Krems, Austria, Leoben, Austria and 65 more

Added 68 new sites: Graz, Austria, Krems, Austria, Leoben, Austria and 65 more

eligibility_changeHodgkin LymphomaJul 18The Lymphoma Academic Research OrganisationNCT06284122
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 18Neonc Technologies, Inc.NCT05023018
Added 2 new sites: San Francisco, California, Boston, Massachusetts

Added 2 new sites: San Francisco, California, Boston, Massachusetts

eligibility_changeHodgkin LymphomaJul 18Alliance for Clinical Trials in OncologyNCT05976763
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 18Miltenyi Biomedicine GmbHNCT04792489
Added 7 new sites: Birmingham, Alabama, Denver, Colorado, St Louis, Missouri and 4 more

Added 7 new sites: Birmingham, Alabama, Denver, Colorado, St Louis, Missouri and 4 more

Enrollment UpdateHodgkin LymphomaJul 18Miltenyi Biomedicine GmbHNCT04792489
Target enrollment increased from 248 to 315

Target enrollment increased from 248 to 315

eligibility_changeHodgkin LymphomaJul 18Miltenyi Biomedicine GmbHNCT04792489
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 18Janssen Research & Development, LLCNCT06788509
Added 3 new sites: Randwick, Australia, Madrid, Spain, Salamanca, Spain

Added 3 new sites: Randwick, Australia, Madrid, Spain, Salamanca, Spain

sites_addedHodgkin LymphomaJul 18Merck Sharp & Dohme LLCNCT06890884
Added 6 new sites: Philadelphia, Pennsylvania, Dallas, Texas, Tübingen, Germany and 3 more

Added 6 new sites: Philadelphia, Pennsylvania, Dallas, Texas, Tübingen, Germany and 3 more

sites_addedHodgkin LymphomaJul 18National Cancer Institute (NCI)NCT03739814
Added 1 new site: Stony Brook, New York

Added 1 new site: Stony Brook, New York

sites_addedHodgkin LymphomaJul 18Bantam PharmaceuticalsNCT06792734
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedHodgkin LymphomaJul 18Children's Oncology GroupNCT03959085
Added 1 new site: Saint Hobart, Australia

Added 1 new site: Saint Hobart, Australia

Enrollment UpdateHodgkin LymphomaJul 18University of UtahNCT06300528
Target enrollment decreased from 27 to 1

Target enrollment decreased from 27 to 1

eligibility_changeHodgkin LymphomaJul 18University of PennsylvaniaNCT02650414
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 18Merck Sharp & Dohme LLCNCT06717347
Added 5 new sites: Mobile, Alabama, Portland, Oregon, Lubbock, Texas and 2 more

Added 5 new sites: Mobile, Alabama, Portland, Oregon, Lubbock, Texas and 2 more

sites_addedHodgkin LymphomaJul 18Medical College of WisconsinNCT07117136
Added 1 new site: Jacksonville, Florida

Added 1 new site: Jacksonville, Florida

sites_addedHodgkin LymphomaJul 18Regeneron PharmaceuticalsNCT06091865
Added 11 new sites: Quimper, France, Lyon, France, Marseille, France and 8 more

Added 11 new sites: Quimper, France, Lyon, France, Marseille, France and 8 more

sites_addedHodgkin LymphomaJul 18Novartis PharmaceuticalsNCT04104776
Added 69 new sites: Tampa, Florida, Atlanta, Georgia, Chicago, Illinois and 66 more

Added 69 new sites: Tampa, Florida, Atlanta, Georgia, Chicago, Illinois and 66 more

eligibility_changeHodgkin LymphomaJul 18Novartis PharmaceuticalsNCT04104776
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 18Hoffmann-La RocheNCT07200375
Added 1 new site: Shanghai, China

Added 1 new site: Shanghai, China

eligibility_changeHodgkin LymphomaJul 18The Lymphoma Academic Research OrganisationNCT06558604
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHereditary Hemorrhagic TelangiectasiaJul 18Alnylam PharmaceuticalsNCT06659640
Target enrollment decreased from 120 to 89

Target enrollment decreased from 120 to 89

sites_addedEssential ThrombocythemiaJul 17Novartis PharmaceuticalsNCT07357727
Added 1 new site: Hefei, China

Added 1 new site: Hefei, China

sites_addedGraves’ DiseaseJul 17Biohaven Therapeutics Ltd.NCT07661056
Added 3 new sites: Doral, Florida, El Paso, Texas, Midlothian, Virginia

Added 3 new sites: Doral, Florida, El Paso, Texas, Midlothian, Virginia

sites_addedGeographic AtrophyJul 17Complement TherapeuticsNCT07144137
Added 1 new site: Oak Forest, Illinois

Added 1 new site: Oak Forest, Illinois

sites_addedGeographic AtrophyJul 17AbbVieNCT07160179
Added 1 new site: Reno, Nevada

Added 1 new site: Reno, Nevada

sites_addedGeographic AtrophyJul 17ONL TherapeuticsNCT06659445
Added 26 new sites: Bakersfield, California, Beverly Hills, California, Huntington Beach, California and 23 more

Added 26 new sites: Bakersfield, California, Beverly Hills, California, Huntington Beach, California and 23 more

sites_addedGastrointestinal Stromal TumorJul 17Deciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Paris, France

Added 1 new site: Paris, France

sites_addedFocal Segmental GlomerulosclerosisJul 17Akebia TherapeuticsNCT07268638
Added 9 new sites: Boca Raton, Florida, Livonia, Michigan, New Bern, North Carolina and 6 more

Added 9 new sites: Boca Raton, Florida, Livonia, Michigan, New Bern, North Carolina and 6 more

eligibility_changeFocal Segmental GlomerulosclerosisJul 17Akebia TherapeuticsNCT07268638
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal Segmental GlomerulosclerosisJul 17Boehringer IngelheimNCT07220083
Added 11 new sites: Brunswick, Georgia, Greensboro, North Carolina, Plovdiv, Bulgaria and 8 more

Added 11 new sites: Brunswick, Georgia, Greensboro, North Carolina, Plovdiv, Bulgaria and 8 more

eligibility_changeFocal Segmental GlomerulosclerosisJul 17Boehringer IngelheimNCT07220083
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateFocal EpilepsyJul 17Assistance Publique - Hôpitaux de ParisNCT06022055
Target enrollment increased from 150 to 300

Target enrollment increased from 150 to 300

sites_addedEssential ThrombocythemiaJul 17Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Buenos Aires, Argentina, Johor Bahru, Malaysia, Kuala Selangor, Malaysia and 1 more

Added 4 new sites: Buenos Aires, Argentina, Johor Bahru, Malaysia, Kuala Selangor, Malaysia and 1 more

sites_addedDuchenne Muscular DystrophyJul 16Solid Biosciences Inc.NCT07160634
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

sites_addedChronic Graft-versus-Host DiseaseJul 16Regeneron PharmaceuticalsNCT07271186
Added 4 new sites: New Haven, Connecticut, DeSoto, Texas, Houston, Texas and 1 more

Added 4 new sites: New Haven, Connecticut, DeSoto, Texas, Houston, Texas and 1 more

sites_removedChronic Graft-versus-Host DiseaseJul 16Novartis PharmaceuticalsNCT06092879
Reduced from 59 to 52 sites

Reduced from 59 to 52 sites

sites_addedChronic Graft-versus-Host DiseaseJul 16CytokineticsNCT06736574
Added 8 new sites: Lanzhou, China, Guangzhou, China, Shenzhen, China and 5 more

Added 8 new sites: Lanzhou, China, Guangzhou, China, Shenzhen, China and 5 more

sites_addedChronic Graft-versus-Host DiseaseJul 16Merz Therapeutics GmbHNCT07018713
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 16University of MiamiNCT06578156
Target enrollment increased from 30 to 45

Target enrollment increased from 30 to 45

eligibility_changeChronic Graft-versus-Host DiseaseJul 16BiogenNCT06044337
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 16AstraZenecaNCT07520162
Added 11 new sites: Changchun, China, Changsha, China, Chongqing, China and 8 more

Added 11 new sites: Changchun, China, Changsha, China, Chongqing, China and 8 more

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 16Fred Hutchinson Cancer CenterNCT04431479
Target enrollment decreased from 300 to 211

Target enrollment decreased from 300 to 211

sites_addedChronic Graft-versus-Host DiseaseJul 16Novartis PharmaceuticalsNCT07465653
Added 1 new site: Montreal, Canada

Added 1 new site: Montreal, Canada

eligibility_changeChronic Graft-versus-Host DiseaseJul 16Novartis PharmaceuticalsNCT07465653
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCentral Diabetes InsipidusJul 16Children's Oncology GroupNCT06368817
Added 6 new sites: New Haven, Connecticut, Scarborough, Maine, Boston, Massachusetts and 3 more

Added 6 new sites: New Haven, Connecticut, Scarborough, Maine, Boston, Massachusetts and 3 more

sites_addedEosinophilic EsophagitisJul 16Dr. Falk Pharma GmbHNCT06596252
Added 1 new site: Plymouth, Michigan

Added 1 new site: Plymouth, Michigan

sites_addedCushing DiseaseJul 16University Hospital, AngersNCT05881005
Added 2 new sites: Lille, France, Vandœuvre-lès-Nancy, France

Added 2 new sites: Lille, France, Vandœuvre-lès-Nancy, France

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16Immunovant Sciences GmbHNCT07032662
Added 3 new sites: Chicago, Illinois, Rosario, Argentina, Lublin, Poland

Added 3 new sites: Chicago, Illinois, Rosario, Argentina, Lublin, Poland

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16Dianthus TherapeuticsNCT06858579
Added 11 new sites: New Haven, Connecticut, Niigata, Japan, Kawasaki, Japan and 8 more

Added 11 new sites: New Haven, Connecticut, Niigata, Japan, Kawasaki, Japan and 8 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16SanofiNCT06290141
Added 2 new sites: New York, New York, Rozzano, Italy

Added 2 new sites: New York, New York, Rozzano, Italy

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16SanofiNCT06859099
Added 2 new sites: Münster, Germany, Tlalnepantla, Mexico

Added 2 new sites: Münster, Germany, Tlalnepantla, Mexico

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 16National Center for Complementary and Integrative Health (NCCIH)NCT03952624
Target enrollment decreased from 692 to 37

Target enrollment decreased from 692 to 37

sites_addedChronic Graft-versus-Host DiseaseJul 16M.D. Anderson Cancer CenterNCT06566742
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

eligibility_changeChronic Graft-versus-Host DiseaseJul 16M.D. Anderson Cancer CenterNCT06566742
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 16Immunovant Sciences GmbHNCT07032662
Added 3 new sites: Chicago, Illinois, Rosario, Argentina, Lublin, Poland

Added 3 new sites: Chicago, Illinois, Rosario, Argentina, Lublin, Poland

eligibility_changeChronic Graft-versus-Host DiseaseJul 16The Second Affiliated Hospital of Chongqing Medical UniversityNCT07573943
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 16Medtronic VascularNCT06907147
Added 2 new sites: Chapel Hill, North Carolina, Erlangen, Germany

Added 2 new sites: Chapel Hill, North Carolina, Erlangen, Germany

sites_addedChronic Graft-versus-Host DiseaseJul 16Merck Sharp & Dohme LLCNCT06136559
Added 1 new site: Boca Raton, Florida

Added 1 new site: Boca Raton, Florida

sites_addedCholangiocarcinomaJul 16InnoPharmax Inc.NCT06622057
Added 1 new site: Miami Beach, Florida

Added 1 new site: Miami Beach, Florida

Enrollment UpdateCholangiocarcinomaJul 16GenfitNCT05874414
Target enrollment increased from 74 to 98

Target enrollment increased from 74 to 98

eligibility_changeCholangiocarcinomaJul 16GenfitNCT05874414
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedAutoimmune HepatitisJul 15Hannover Medical SchoolNCT05810480
Reduced from 8 to 6 sites

Reduced from 8 to 6 sites

Enrollment UpdateAutoimmune HepatitisJul 15Hannover Medical SchoolNCT05810480
Target enrollment increased from 200 to 400

Target enrollment increased from 200 to 400

eligibility_changeAutoimmune HepatitisJul 15Hannover Medical SchoolNCT05810480
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedC3 GlomerulopathyJul 15Novartis PharmaceuticalsNCT03955445
Added 3 new sites: Heidelberg, Germany, Bari, Italy, Roma, Italy

Added 3 new sites: Heidelberg, Germany, Bari, Italy, Roma, Italy

sites_addedBarth SyndromeJul 15LMU KlinikumNCT05554835
Added 15 new sites: Bologna, Italy, Bologna, Italy, Bologna, Italy and 12 more

Added 15 new sites: Bologna, Italy, Bologna, Italy, Bologna, Italy and 12 more

sites_removedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 15AstraZenecaNCT07608354
Reduced from 78 to 76 sites

Reduced from 78 to 76 sites

sites_addedAmyotrophic Lateral SclerosisJul 15PrileniaNCT07322003
Added 1 new site: Ulm, Germany

Added 1 new site: Ulm, Germany

sites_addedAmyotrophic Lateral SclerosisJul 15PhenoNet, Inc.NCT07142291
Added 1 new site: Málaga, Spain

Added 1 new site: Málaga, Spain

sites_addedAlpha-1 Antitrypsin DeficiencyJul 15Grifols Therapeutics LLCNCT07555483
Added 22 new sites: Birmingham, Alabama, Phoenix, Arizona, Scottsdale, Arizona and 19 more

Added 22 new sites: Birmingham, Alabama, Phoenix, Arizona, Scottsdale, Arizona and 19 more

sites_addedAchondroplasiaJul 15Tyra Biosciences, IncNCT06842355
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedWaldenström MacroglobulinemiaJul 14National Cancer Institute (NCI)NCT04840602
Added 1 new site: Irvine, California

Added 1 new site: Irvine, California

sites_addedSickle Cell DiseaseJul 14Novartis PharmaceuticalsNCT06546670
Added 1 new site: Adana, Turkey (Türkiye)

Added 1 new site: Adana, Turkey (Türkiye)

Enrollment UpdateX-Linked HypophosphatemiaJul 14Kyowa Kirin Pharmaceutical Development LtdNCT03193476
Target enrollment increased from 1343 to 1489

Target enrollment increased from 1343 to 1489

sites_addedVon Willebrand DiseaseJul 14Hoffmann-La RocheNCT06998524
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

sites_removedUsher SyndromeJul 14Laboratoires TheaNCT06627179
Reduced from 28 to 26 sites

Reduced from 28 to 26 sites

sites_addedTurner SyndromeJul 14Novo Nordisk A/SNCT05723835
Added 1 new site: Yangsan, South Korea

Added 1 new site: Yangsan, South Korea

sites_addedTurner SyndromeJul 14Ascendis Pharma A/SNCT07221851
Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

eligibility_changeThalassemiaJul 14National Heart, Lung, and Blood Institute (NHLBI)NCT07599176
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeThalassemiaJul 14CelgeneNCT04143724
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateSpinocerebellar AtaxiaJul 14BiogenNCT06628687
Target enrollment decreased from 20 to 10

Target enrollment decreased from 20 to 10

eligibility_changeSpinal Muscular AtrophyJul 14St. Jude Children's Research HospitalNCT06532474
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateSpinal Muscular AtrophyJul 14Novartis PharmaceuticalsNCT05335876
Target enrollment decreased from 175 to 20

Target enrollment decreased from 175 to 20

eligibility_changeSickle Cell DiseaseJul 14National Heart, Lung, and Blood Institute (NHLBI)NCT07599176
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedRetinitis PigmentosaJul 13LMU KlinikumNCT05554835
Added 15 new sites: Bologna, Italy, Bologna, Italy, Bologna, Italy and 12 more

Added 15 new sites: Bologna, Italy, Bologna, Italy, Bologna, Italy and 12 more

sites_addedPhelan-McDermid SyndromeJul 13Neuren Pharmaceuticals LimitedNCT07593391
Added 1 new site: Chevy Chase, Maryland

Added 1 new site: Chevy Chase, Maryland

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 13Regeneron PharmaceuticalsNCT05744921
Added 1 new site: Chandigarh, India

Added 1 new site: Chandigarh, India

sites_addedRett SyndromeJul 13Taysha Gene Therapies, Inc.NCT05606614
Added 3 new sites: Duarte, California, Oakland, California, Baltimore, Maryland

Added 3 new sites: Duarte, California, Oakland, California, Baltimore, Maryland

sites_removedRetinitis PigmentosaJul 13Laboratoires TheaNCT06627179
Reduced from 28 to 26 sites

Reduced from 28 to 26 sites

sites_removedPulmonary Arterial HypertensionJul 13Tectonic TherapeuticNCT06616974
Reduced from 86 to 72 sites

Reduced from 86 to 72 sites

sites_addedPulmonary Arterial HypertensionJul 13Merck Sharp & Dohme LLCNCT07218029
Added 1 new site: Adelaide, Australia

Added 1 new site: Adelaide, Australia

sites_addedPrimary Biliary CholangitisJul 13Cascade Pharmaceuticals, IncNCT07282353
Added 5 new sites: Hefei, China, Xiamen, China, Wuhan, China and 2 more

Added 5 new sites: Hefei, China, Xiamen, China, Wuhan, China and 2 more

eligibility_changePrimary Biliary CholangitisJul 13Cascade Pharmaceuticals, IncNCT07282353
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraJul 13Novartis PharmaceuticalsNCT07357727
Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

sites_addedPolycythemia VeraJul 13Ono Pharmaceutical Co., Ltd.NCT07429266
Added 1 new site: The Bronx, New York

Added 1 new site: The Bronx, New York

Enrollment UpdatePolycythemia VeraJul 13FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETSNCT06506084
Target enrollment increased from 319 to 429

Target enrollment increased from 319 to 429

sites_addedPhenylketonuriaJul 13Otsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Added 1 new site: Mainz, Germany

Added 1 new site: Mainz, Germany

sites_addedPhelan-McDermid SyndromeJul 13Neuren Pharmaceuticals LimitedNCT07281079
Added 3 new sites: Palo Alto, California, Houston, Texas, Toronto, Canada

Added 3 new sites: Palo Alto, California, Houston, Texas, Toronto, Canada

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 13Regeneron PharmaceuticalsNCT07154745
Added 3 new sites: Santo André, Brazil, Lévis, Canada, Barcelona, Spain

Added 3 new sites: Santo André, Brazil, Lévis, Canada, Barcelona, Spain

sites_addedNoonan SyndromeJul 12Novo Nordisk A/SNCT05723835
Added 1 new site: Yangsan, South Korea

Added 1 new site: Yangsan, South Korea

sites_addedNoonan SyndromeJul 12Ascendis Pharma A/SNCT07221851
Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

sites_removedNeurofibromatosis Type 1Jul 12SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, GermanyNCT03962543
Reduced from 50 to 43 sites

Reduced from 50 to 43 sites

sites_addedNarcolepsy Type 1Jul 12Alkermes, Inc.NCT07540897
Added 5 new sites: Miami, Florida, Winter Park, Florida, Lansing, Michigan and 2 more

Added 5 new sites: Miami, Florida, Winter Park, Florida, Lansing, Michigan and 2 more

sites_addedNarcolepsy Type 1Jul 12TakedaNCT07363720
Added 23 new sites: Redwood City, California, Brandon, Florida, Miami, Florida and 20 more

Added 23 new sites: Redwood City, California, Brandon, Florida, Miami, Florida and 20 more

sites_addedMyotonic DystrophyJul 12Dyne TherapeuticsNCT07486934
Added 3 new sites: La Jolla, California, Nantes, France, Toulouse, France

Added 3 new sites: La Jolla, California, Nantes, France, Toulouse, France

sites_addedMyelofibrosisJul 12Novartis PharmaceuticalsNCT07357727
Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

sites_removedMyelofibrosisJul 12FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETSNCT06533813
Reduced from 36 to 27 sites

Reduced from 36 to 27 sites

sites_addedMyelofibrosisJul 12Incyte CorporationNCT07008118
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedMyelodysplastic SyndromesJul 12University of Southern CaliforniaNCT02935361
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_removedMyelodysplastic SyndromesJul 12GenmabNCT07384715
Reduced from 9 to 6 sites

Reduced from 9 to 6 sites

sites_addedMyelodysplastic SyndromesJul 12Janssen Research & Development, LLCNCT06788509
Added 3 new sites: Randwick, Australia, Madrid, Spain, Salamanca, Spain

Added 3 new sites: Randwick, Australia, Madrid, Spain, Salamanca, Spain

eligibility_changeMyelodysplastic SyndromesJul 12Jennifer SaultzNCT03874052
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJul 12National Cancer Institute (NCI)NCT06577441
Added 3 new sites: Fairway, Kansas, Kansas City, Kansas, Westwood, Kansas

Added 3 new sites: Fairway, Kansas, Kansas City, Kansas, Westwood, Kansas

sites_addedMyelodysplastic SyndromesJul 12Regeneron PharmaceuticalsNCT07154745
Added 3 new sites: Santo André, Brazil, Lévis, Canada, Barcelona, Spain

Added 3 new sites: Santo André, Brazil, Lévis, Canada, Barcelona, Spain

sites_addedMyelodysplastic SyndromesJul 12Orca Biosystems, Inc.NCT07216443
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedMyasthenia GravisJul 12AmgenNCT06987539
Added 1 new site: Headington, United Kingdom

Added 1 new site: Headington, United Kingdom

sites_addedMyasthenia GravisJul 12Janssen Research & Development, LLCNCT07217587
Added 1 new site: New Orleans, Louisiana

Added 1 new site: New Orleans, Louisiana

sites_addedMyasthenia GravisJul 12Kyverna TherapeuticsNCT06193889
Added 5 new sites: Los Angeles, California, Portland, Oregon, Riyadh, Saudi Arabia and 2 more

Added 5 new sites: Los Angeles, California, Portland, Oregon, Riyadh, Saudi Arabia and 2 more

sites_addedMultiple System AtrophyJul 12Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: München, Germany, Münster, Germany

Added 2 new sites: München, Germany, Münster, Germany

sites_addedMultiple System AtrophyJul 12BiogenNCT07221669
Added 1 new site: Tokyo, Japan

Added 1 new site: Tokyo, Japan

eligibility_changeMultiple SclerosisJul 12University of Colorado, DenverNCT06641271
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 12Novartis PharmaceuticalsNCT06655896
Added 4 new sites: CABA, Argentina, Córdoba, Argentina, Curitiba, Brazil and 1 more

Added 4 new sites: CABA, Argentina, Córdoba, Argentina, Curitiba, Brazil and 1 more

sites_addedMultiple SclerosisJul 12Zenas BioPharma (USA), LLCNCT07067463
Added 6 new sites: Roeland Park, Kansas, Plovdiv, Bulgaria, Sofia, Bulgaria and 3 more

Added 6 new sites: Roeland Park, Kansas, Plovdiv, Bulgaria, Sofia, Bulgaria and 3 more

sites_addedMultiple SclerosisJul 12Johns Hopkins UniversityNCT07497399
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedMultiple SclerosisJul 12Hoffmann-La RocheNCT07282574
Added 18 new sites: New Lambton Heights, Australia, Box Hill, Australia, Strasbourg, France and 15 more

Added 18 new sites: New Lambton Heights, Australia, Box Hill, Australia, Strasbourg, France and 15 more

eligibility_changeMixed Connective Tissue DiseaseJul 12St. Jude Children's Research HospitalNCT06239272
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaJul 12AstraZenecaNCT06535607
Added 1 new site: Natal, Brazil

Added 1 new site: Natal, Brazil

sites_addedMesotheliomaJul 12Dutch Society of Physicians for Pulmonology and TuberculosisNCT06996249
Added 13 new sites: Alkmaar, Netherlands, Amsterdam, Netherlands, Breda, Netherlands and 10 more

Added 13 new sites: Alkmaar, Netherlands, Amsterdam, Netherlands, Breda, Netherlands and 10 more

sites_addedMesotheliomaJul 12Perspective TherapeuticsNCT06710756
Added 3 new sites: Kansas City, Kansas, Rochester, Minnesota, Madison, Wisconsin

Added 3 new sites: Kansas City, Kansas, Rochester, Minnesota, Madison, Wisconsin

sites_addedMesotheliomaJul 12IDEAYA BiosciencesNCT07277413
Added 1 new site: Washington D.C., District of Columbia

Added 1 new site: Washington D.C., District of Columbia

sites_addedMesotheliomaJul 12Nuvectis Pharma, Inc.NCT05873686
Added 3 new sites: La Jolla, California, New York, New York, Cleveland, Ohio

Added 3 new sites: La Jolla, California, New York, New York, Cleveland, Ohio

eligibility_changeMesotheliomaJul 12Nuvectis Pharma, Inc.NCT05873686
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMembranous NephropathyJul 12Nanjing University School of MedicineNCT06285279
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMedullary Thyroid CancerJul 12National Cancer Institute (NCI)NCT03866382
Added 1 new site: Westerville, Ohio

Added 1 new site: Westerville, Ohio

sites_addedLight Chain AmyloidosisJul 11Jin Lu, MDNCT06192979
Added 1 new site: Qingdao, China

Added 1 new site: Qingdao, China

eligibility_changeIgA Vasculitis with NephritisJul 11Nanjing University School of MedicineNCT06285279
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeIgG4-Related DiseaseJul 11Nanjing University School of MedicineNCT06285279
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyJul 11TakedaNCT06963827
Added 1 new site: Barakaldo, Spain

Added 1 new site: Barakaldo, Spain

sites_addedIgA NephropathyJul 11Jade Biosciences, Inc.NCT07541287
Added 2 new sites: Seoul, South Korea, Seoul, South Korea

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

sites_addedIgA NephropathyJul 11BiogenNCT06935357
Added 1 new site: Konya, Turkey (Türkiye)

Added 1 new site: Konya, Turkey (Türkiye)

sites_addedIdiopathic Pulmonary FibrosisJul 11Contineum TherapeuticsNCT07284459
Added 1 new site: San Miguel de Tucumán, Argentina

Added 1 new site: San Miguel de Tucumán, Argentina

sites_addedIdiopathic Pulmonary FibrosisJul 11Boehringer IngelheimNCT06238622
Added 5 new sites: The Woodlands, Texas, Brussels, Belgium, Belo Horizonte,Minas Gerais, Brazil and 2 more

Added 5 new sites: The Woodlands, Texas, Brussels, Belgium, Belo Horizonte,Minas Gerais, Brazil and 2 more

Enrollment UpdateHodgkin LymphomaJul 11OHSU Knight Cancer InstituteNCT06249191
Target enrollment increased from 30 to 40

Target enrollment increased from 30 to 40

eligibility_changeHodgkin LymphomaJul 11OHSU Knight Cancer InstituteNCT06249191
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 11Merck Sharp & Dohme LLCNCT06717347
Added 10 new sites: Long Beach, California, Owensboro, Kentucky, Bethesda, Maryland and 7 more

Added 10 new sites: Long Beach, California, Owensboro, Kentucky, Bethesda, Maryland and 7 more

sites_addedHodgkin LymphomaJul 11Merck Sharp & Dohme LLCNCT07519772
Added 17 new sites: Nashville, Tennessee, Ciudad Autonoma de Buenos Aires, Argentina, Rosario, Argentina and 14 more

Added 17 new sites: Nashville, Tennessee, Ciudad Autonoma de Buenos Aires, Argentina, Rosario, Argentina and 14 more

sites_addedHodgkin LymphomaJul 11Merck Sharp & Dohme LLCNCT05139017
Added 3 new sites: Isehara, Japan, Miyazaki, Japan, Osaka, Japan

Added 3 new sites: Isehara, Japan, Miyazaki, Japan, Osaka, Japan

eligibility_changeHodgkin LymphomaJul 11Alliance for Clinical Trials in OncologyNCT05303792
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 11National Cancer Institute (NCI)NCT04840602
Added 4 new sites: Maywood, Illinois, Coon Rapids, Minnesota, Minneapolis, Minnesota and 1 more

Added 4 new sites: Maywood, Illinois, Coon Rapids, Minnesota, Minneapolis, Minnesota and 1 more

sites_addedHodgkin LymphomaJul 11Merck Sharp & Dohme LLCNCT06890884
Added 9 new sites: Boston, Massachusetts, Westbury, New York, Sint-Niklaas, Belgium and 6 more

Added 9 new sites: Boston, Massachusetts, Westbury, New York, Sint-Niklaas, Belgium and 6 more

sites_addedHodgkin LymphomaJul 11Novartis PharmaceuticalsNCT07422610
Added 3 new sites: Saint-Herblain, France, Pozuelo de Alarcón, Spain, Barcelona, Spain

Added 3 new sites: Saint-Herblain, France, Pozuelo de Alarcón, Spain, Barcelona, Spain

sites_addedHodgkin LymphomaJul 11Tempus AINCT07154823
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

sites_addedHodgkin LymphomaJul 11Great Novel Therapeutics Biotech & Medicals CorporationNCT05833724
Added 1 new site: New Taipei City, Taiwan

Added 1 new site: New Taipei City, Taiwan

sites_addedHodgkin LymphomaJul 11Lungpacer Medical Inc.NCT06832306
Added 1 new site: Winston-Salem, North Carolina

Added 1 new site: Winston-Salem, North Carolina

sites_addedHodgkin LymphomaJul 11Dizal PharmaceuticalsNCT07234162
Added 1 new site: Khon Kaen, Thailand

Added 1 new site: Khon Kaen, Thailand

eligibility_changeHodgkin LymphomaJul 11Dizal PharmaceuticalsNCT07234162
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 11National Cancer Institute (NCI)NCT06649812
Added 2 new sites: Canton, Ohio, Madison, Wisconsin

Added 2 new sites: Canton, Ohio, Madison, Wisconsin

eligibility_changeHodgkin LymphomaJul 11National Cancer Institute (NCI)NCT06649812
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaJul 11GenmabNCT05283720
Target enrollment decreased from 496 to 264

Target enrollment decreased from 496 to 264

sites_addedHodgkin LymphomaJul 11PfizerNCT03460977
Added 2 new sites: Louisville, Kentucky, Irving, Texas

Added 2 new sites: Louisville, Kentucky, Irving, Texas

sites_addedHodgkin LymphomaJul 11Bristol-Myers SquibbNCT06090539
Added 8 new sites: Beijing, China, Guangzhou, China, Zhengzhou, China and 5 more

Added 8 new sites: Beijing, China, Guangzhou, China, Zhengzhou, China and 5 more

Enrollment UpdateHodgkin LymphomaJul 11Bristol-Myers SquibbNCT06090539
Target enrollment increased from 308 to 460

Target enrollment increased from 308 to 460

eligibility_changeHodgkin LymphomaJul 11Bristol-Myers SquibbNCT06090539
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 11National Cancer Institute (NCI)NCT05675410
Added 3 new sites: Madera, California, Carroll, Iowa, Houston, Texas

Added 3 new sites: Madera, California, Carroll, Iowa, Houston, Texas

sites_addedHodgkin LymphomaJul 11Merck Sharp & Dohme LLCNCT07570173
Added 6 new sites: Aarhus Nord, Denmark, Odense, Denmark, Bunkyo, Japan and 3 more

Added 6 new sites: Aarhus Nord, Denmark, Odense, Denmark, Bunkyo, Japan and 3 more

Enrollment UpdateHodgkin LymphomaJul 11ImmunityBio, Inc.NCT06334991
Target enrollment increased from 10 to 20

Target enrollment increased from 10 to 20

sites_addedHodgkin LymphomaJul 11Merck Sharp & Dohme LLCNCT07634471
Added 2 new sites: Tel Aviv, Israel, Istanbul, Turkey (Türkiye)

Added 2 new sites: Tel Aviv, Israel, Istanbul, Turkey (Türkiye)

sites_addedGraves’ DiseaseJul 10Biohaven Therapeutics Ltd.NCT07661056
Added 1 new site: Layton, Utah

Added 1 new site: Layton, Utah

sites_addedGraves’ DiseaseJul 10AmgenNCT06401044
Added 4 new sites: Newport Beach, California, Boston, Massachusetts, Houston, Texas and 1 more

Added 4 new sites: Newport Beach, California, Boston, Massachusetts, Houston, Texas and 1 more

sites_addedGraves’ DiseaseJul 10Immunovant Sciences GmbHNCT07018323
Added 5 new sites: Santiago, Chile, Nagoya, Japan, Kamogawa, Japan and 2 more

Added 5 new sites: Santiago, Chile, Nagoya, Japan, Kamogawa, Japan and 2 more

Enrollment UpdateFriedreich AtaxiaJul 10BiogenNCT06628687
Target enrollment decreased from 20 to 10

Target enrollment decreased from 20 to 10

sites_addedHemophilia BJul 10Regeneron PharmaceuticalsNCT06379789
Added 2 new sites: Herston, Australia, Adelaide, Australia

Added 2 new sites: Herston, Australia, Adelaide, Australia

sites_addedHemophilia AJul 10Hoffmann-La RocheNCT07416604
Added 4 new sites: Iowa City, Iowa, Madrid, Spain, Málaga, Spain and 1 more

Added 4 new sites: Iowa City, Iowa, Madrid, Spain, Málaga, Spain and 1 more

sites_addedHemophilia AJul 10Regeneron PharmaceuticalsNCT06379789
Added 2 new sites: Herston, Australia, Adelaide, Australia

Added 2 new sites: Herston, Australia, Adelaide, Australia

sites_addedGraves’ DiseaseJul 10Biohaven Therapeutics Ltd.NCT07661056
Added 6 new sites: San Diego, California, Hialeah, Florida, Morehead City, North Carolina and 3 more

Added 6 new sites: San Diego, California, Hialeah, Florida, Morehead City, North Carolina and 3 more

sites_addedGraves’ DiseaseJul 10SanofiNCT06984627
Added 2 new sites: Merano, Italy, Milan, Italy

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

sites_addedGeographic AtrophyJul 10Regeneron PharmaceuticalsNCT06541704
Added 1 new site: Lille, France

Added 1 new site: Lille, France

sites_addedGeographic AtrophyJul 10Novartis PharmaceuticalsNCT07441642
Added 10 new sites: Philadelphia, Pennsylvania, London, Canada, Ludwigshafen, Germany and 7 more

Added 10 new sites: Philadelphia, Pennsylvania, London, Canada, Ludwigshafen, Germany and 7 more

sites_addedGastrointestinal Stromal TumorJul 10Kumquat Biosciences Inc.NCT07406633
Added 8 new sites: Miami, Florida, Chicago, Illinois, Iowa City, Iowa and 5 more

Added 8 new sites: Miami, Florida, Chicago, Illinois, Iowa City, Iowa and 5 more

sites_addedFocal EpilepsyJul 10Xenon Pharmaceuticals Inc.NCT05718817
Added 2 new sites: Philadelphia, Pennsylvania, Nuevo León, Mexico

Added 2 new sites: Philadelphia, Pennsylvania, Nuevo León, Mexico

sites_addedEssential ThrombocythemiaJul 10Incyte CorporationNCT07008118
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedEssential ThrombocythemiaJul 10Novartis PharmaceuticalsNCT07357727
Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

sites_addedDravet SyndromeJul 9Encoded TherapeuticsNCT05419492
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedDilated CardiomyopathyJul 9Affinia TherapeuticsNCT07426419
Added 1 new site: Kansas City, Missouri

Added 1 new site: Kansas City, Missouri

eligibility_changeChronic Graft-versus-Host DiseaseJul 9Incyte Biosciences International SàrlNCT03934372
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedChronic Graft-versus-Host DiseaseJul 9Juno Therapeutics, a Subsidiary of CelgeneNCT03331198
Reduced from 86 to 84 sites

Reduced from 86 to 84 sites

sites_addedChronic Graft-versus-Host DiseaseJul 9Regeneron PharmaceuticalsNCT06585410
Added 6 new sites: Phoenix, Arizona, Oceanside, California, Delray Beach, Florida and 3 more

Added 6 new sites: Phoenix, Arizona, Oceanside, California, Delray Beach, Florida and 3 more

eligibility_changeChronic Graft-versus-Host DiseaseJul 9Regeneron PharmaceuticalsNCT06585410
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 9University of Alabama at BirminghamNCT05143840
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 9Loxo Oncology, Inc.NCT05254743
Target enrollment increased from 662 to 737

Target enrollment increased from 662 to 737

eligibility_changeChronic Graft-versus-Host DiseaseJul 9Western University, CanadaNCT06396416
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 9BeOne MedicinesNCT06846671
Added 7 new sites: Westbury, New York, Caba, Argentina, San Miguel de Tucumán, Argentina and 4 more

Added 7 new sites: Westbury, New York, Caba, Argentina, San Miguel de Tucumán, Argentina and 4 more

eligibility_changeChronic Graft-versus-Host DiseaseJul 9C. Babis AndreadisNCT04545762
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 9R1 TherapeuticsNCT06712654
Added 21 new sites: Huntsville, Alabama, Chula Vista, California, San Dimas, California and 18 more

Added 21 new sites: Huntsville, Alabama, Chula Vista, California, San Dimas, California and 18 more

sites_removedChronic Graft-versus-Host DiseaseJul 9Novartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

sites_addedChronic Graft-versus-Host DiseaseJul 9Tam Anh Research InstituteNCT06638320
Added 3 new sites: Hà Nội, Vietnam, Hà Nội, Vietnam, Ho Chi Minh City, Vietnam

Added 3 new sites: Hà Nội, Vietnam, Hà Nội, Vietnam, Ho Chi Minh City, Vietnam

sites_removedChronic Graft-versus-Host DiseaseJul 9Arthrosi TherapeuticsNCT06846515
Reduced from 89 to 83 sites

Reduced from 89 to 83 sites

sites_addedCatecholaminergic Polymorphic Ventricular TachycardiaJul 9Cardurion Pharmaceuticals, Inc.NCT06658899
Added 3 new sites: Salt Lake City, Utah, Bron, France, Paris, France

Added 3 new sites: Salt Lake City, Utah, Bron, France, Paris, France

sites_addedDilated CardiomyopathyJul 9Kardigan, Inc.NCT07210723
Added 14 new sites: Minneapolis, Minnesota, Durham, North Carolina, Cincinnati, Ohio and 11 more

Added 14 new sites: Minneapolis, Minnesota, Durham, North Carolina, Cincinnati, Ohio and 11 more

Enrollment UpdateCystic FibrosisJul 9University of Alabama at BirminghamNCT03256773
Target enrollment increased from 60 to 160

Target enrollment increased from 60 to 160

eligibility_changeCystic FibrosisJul 9Physio-AssistNCT06487273
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCongenital Adrenal HyperplasiaJul 9Crinetics Pharmaceuticals Inc.NCT07159841
Added 2 new sites: San Francisco, California, Tübingen, Germany

Added 2 new sites: San Francisco, California, Tübingen, Germany

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 9Grifols Therapeutics LLCNCT07540221
Added 1 new site: Port Charlotte, Florida

Added 1 new site: Port Charlotte, Florida

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 9Janssen Research & Development, LLCNCT05327114
Added 3 new sites: Miami, Florida, Halle, Germany, Catania, Italy

Added 3 new sites: Miami, Florida, Halle, Germany, Catania, Italy

eligibility_changeChronic Graft-versus-Host DiseaseJul 9Ellipses PharmaNCT04581512
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 9Eli Lilly and CompanyNCT06338995
Added 8 new sites: Huntsville, Alabama, Tucson, Arizona, La Mesa, California and 5 more

Added 8 new sites: Huntsville, Alabama, Tucson, Arizona, La Mesa, California and 5 more

eligibility_changeChronic Graft-versus-Host DiseaseJul 9M.D. Anderson Cancer CenterNCT07325253
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJul 9Adia Med of Winter Park LLCNCT07572890
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 9Avalyn Pharma Inc.NCT06329401
Added 2 new sites: Mount Kisco, New York, Beaverton, Oregon

Added 2 new sites: Mount Kisco, New York, Beaverton, Oregon

sites_addedChronic Graft-versus-Host DiseaseJul 9BiogenNCT05531565
Added 1 new site: Belo Horizonte, Brazil

Added 1 new site: Belo Horizonte, Brazil

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 9Reistone Biopharma Company LimitedNCT07349524
Target enrollment increased from 237 to 300

Target enrollment increased from 237 to 300

eligibility_changeChronic Graft-versus-Host DiseaseJul 9Umeå UniversityNCT06580093
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 9The Hong Kong Polytechnic UniversityNCT07112911
Added 2 new sites: Hong Kong, Hong Kong, Hong Kong, Hong Kong

Added 2 new sites: Hong Kong, Hong Kong, Hong Kong, Hong Kong

Enrollment UpdateCholangiocarcinomaJul 9InnoPharmax Inc.NCT06622057
Target enrollment increased from 195 to 240

Target enrollment increased from 195 to 240

sites_addedAmyotrophic Lateral SclerosisJul 8PrileniaNCT07322003
Added 3 new sites: Jacksonville, Florida, Berlin, Germany, Madrid, Spain

Added 3 new sites: Jacksonville, Florida, Berlin, Germany, Madrid, Spain

sites_addedAdrenocortical CarcinomaJul 8National Cancer Institute (NCI)NCT06900595
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedBecker Muscular DystrophyJul 8ITF Therapeutics LLCNCT07127978
Added 2 new sites: Durham, North Carolina, Portland, Oregon

Added 2 new sites: Durham, North Carolina, Portland, Oregon

sites_addedAmyotrophic Lateral SclerosisJul 8PrileniaNCT07322003
Added 2 new sites: Paris, France, Jena, Germany

Added 2 new sites: Paris, France, Jena, Germany

sites_removedAlagille SyndromeJul 8Albireo, an Ipsen CompanyNCT05035030
Reduced from 39 to 35 sites

Reduced from 39 to 35 sites

sites_addedAdrenocortical CarcinomaJul 8National Cancer Institute (NCI)NCT06900595
Added 1 new site: Madera, California

Added 1 new site: Madera, California

sites_addedAchondroplasiaJul 8BioMarin PharmaceuticalNCT07441876
Added 10 new sites: Wilmington, Delaware, Baltimore, Maryland, Cincinnati, Ohio and 7 more

Added 10 new sites: Wilmington, Delaware, Baltimore, Maryland, Cincinnati, Ohio and 7 more

sites_addedUsher SyndromeJul 7BlueRock TherapeuticsNCT06789445
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_removedThalassemiaJul 7Novartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

Enrollment UpdateThalassemiaJul 7Regeneron PharmaceuticalsNCT06421636
Target enrollment decreased from 95 to 19

Target enrollment decreased from 95 to 19

sites_addedStargardt DiseaseJul 7Alkeus Pharmaceuticals, Inc.NCT07419334
Added 1 new site: Fort Worth, Texas

Added 1 new site: Fort Worth, Texas

sites_addedSpinal Muscular AtrophyJul 7BiogenNCT07221669
Added 1 new site: Tokyo, Japan

Added 1 new site: Tokyo, Japan

Enrollment UpdateSjögren's SyndromeJul 7National Center for Complementary and Integrative Health (NCCIH)NCT03952624
Target enrollment decreased from 692 to 37

Target enrollment decreased from 692 to 37

sites_addedSickle Cell DiseaseJul 7Kamau TherapeuticsNCT04819841
Added 2 new sites: New York, New York, New York, New York

Added 2 new sites: New York, New York, New York, New York

Enrollment UpdateSickle Cell DiseaseJul 7Bristol-Myers SquibbNCT06481306
Target enrollment increased from 184 to 224

Target enrollment increased from 184 to 224

eligibility_changeSickle Cell DiseaseJul 7Bristol-Myers SquibbNCT06481306
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedShort Bowel SyndromeJul 6Boston Children's HospitalNCT03530852
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

eligibility_changeShort Bowel SyndromeJul 6Boston Children's HospitalNCT03530852
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedShort Bowel SyndromeJul 6Boston Children's HospitalNCT05635747
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedRetinitis PigmentosaJul 6BlueRock TherapeuticsNCT06789445
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

Enrollment UpdatePulmonary Arterial HypertensionJul 6University of Sao Paulo General HospitalNCT07013149
Target enrollment increased from 121 to 183

Target enrollment increased from 121 to 183

eligibility_changePulmonary Arterial HypertensionJul 6University of Sao Paulo General HospitalNCT07013149
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeNTRK Fusion CancerJul 6St. Jude Children's Research HospitalNCT06239272
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSarcoidosisJul 6Ottawa Heart Institute Research CorporationNCT03593759
Added 1 new site: Auckland, New Zealand

Added 1 new site: Auckland, New Zealand

sites_addedRett SyndromeJul 6International Rett Syndrome FoundationNCT05432349
Added 2 new sites: San Diego, California, Miami, Florida

Added 2 new sites: San Diego, California, Miami, Florida

eligibility_changePulmonary Arterial HypertensionJul 6Guangxi Medical UniversityNCT07658586
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJul 6Tempus AINCT06911632
Added 5 new sites: Reno, Nevada, Albuquerque, New Mexico, Philadelphia, Pennsylvania and 2 more

Added 5 new sites: Reno, Nevada, Albuquerque, New Mexico, Philadelphia, Pennsylvania and 2 more

sites_addedProgressive Familial Intrahepatic CholestasisJul 6IpsenNCT07588880
Added 2 new sites: Beijing, China, Hunan, China

Added 2 new sites: Beijing, China, Hunan, China

sites_addedPrimary Sclerosing CholangitisJul 6IpsenNCT07387549
Added 2 new sites: Colorado Springs, Colorado, Johnson City, Tennessee

Added 2 new sites: Colorado Springs, Colorado, Johnson City, Tennessee

sites_addedPrimary Biliary CholangitisJul 6IpsenNCT07387549
Added 2 new sites: Colorado Springs, Colorado, Johnson City, Tennessee

Added 2 new sites: Colorado Springs, Colorado, Johnson City, Tennessee

sites_addedPrimary Biliary CholangitisJul 6IpsenNCT06016842
Added 3 new sites: Rochester, Minnesota, Huntington, West Virginia, Coimbra, Portugal

Added 3 new sites: Rochester, Minnesota, Huntington, West Virginia, Coimbra, Portugal

sites_removedPolycythemia VeraJul 6Novartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

sites_addedPolycythemia VeraJul 6Ono Pharmaceutical Co., Ltd.NCT07429266
Added 4 new sites: Plantation, Florida, Tamarac, Florida, Bethesda, Maryland and 1 more

Added 4 new sites: Plantation, Florida, Tamarac, Florida, Bethesda, Maryland and 1 more

sites_addedPolycythemia VeraJul 6Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Guangzhou, China, Yantai, China

Added 2 new sites: Guangzhou, China, Yantai, China

phase_changePhelan-McDermid SyndromeJul 6Peking University First HospitalNCT07014020
Moved from N/A to Phase 1

Moved from N/A to Phase 1

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 6Swedish Orphan BiovitrumNCT05776472
Added 15 new sites: Garran, Australia, Anderlecht, Belgium, Turnhout, Belgium and 12 more

Added 15 new sites: Garran, Australia, Anderlecht, Belgium, Turnhout, Belgium and 12 more

sites_addedNarcolepsy Type 1Jul 5TakedaNCT07633301
Added 17 new sites: Redwood City, California, Miami, Florida, Cincinnati, Ohio and 14 more

Added 17 new sites: Redwood City, California, Miami, Florida, Cincinnati, Ohio and 14 more

sites_addedNarcolepsy Type 1Jul 5Centessa Pharmaceuticals (UK) LimitedNCT07598708
Added 3 new sites: Denver, North Carolina, Cincinnati, Ohio, Sugar Land, Texas

Added 3 new sites: Denver, North Carolina, Cincinnati, Ohio, Sugar Land, Texas

sites_addedNarcolepsy Type 1Jul 5Alkermes, Inc.NCT07455383
Added 5 new sites: Cullman, Alabama, Kansas City, Kansas, Middletown, New Jersey and 2 more

Added 5 new sites: Cullman, Alabama, Kansas City, Kansas, Middletown, New Jersey and 2 more

sites_addedMyotonic DystrophyJul 5Vertex Pharmaceuticals IncorporatedNCT06926621
Added 1 new site: München, Germany

Added 1 new site: München, Germany

sites_addedMyotonic DystrophyJul 5Dyne TherapeuticsNCT07486934
Added 8 new sites: Indianapolis, Indiana, Leuven, Belgium, Copenhagen, Denmark and 5 more

Added 8 new sites: Indianapolis, Indiana, Leuven, Belgium, Copenhagen, Denmark and 5 more

sites_removedMyelofibrosisJul 5Novartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

sites_addedMyelofibrosisJul 5Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Guangzhou, China, Yantai, China

Added 2 new sites: Guangzhou, China, Yantai, China

sites_addedMyelodysplastic SyndromesJul 5Orca Biosystems, Inc.NCT03802695
Added 1 new site: Hackensack, New Jersey

Added 1 new site: Hackensack, New Jersey

sites_removedMyelodysplastic SyndromesJul 5AstraZenecaNCT07457151
Reduced from 5 to 2 sites

Reduced from 5 to 2 sites

Enrollment UpdateMyelodysplastic SyndromesJul 5OHSU Knight Cancer InstituteNCT06357182
Target enrollment increased from 24 to 30

Target enrollment increased from 24 to 30

eligibility_changeMyelodysplastic SyndromesJul 5OHSU Knight Cancer InstituteNCT06357182
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJul 5Swedish Orphan BiovitrumNCT05776472
Added 15 new sites: Garran, Australia, Anderlecht, Belgium, Turnhout, Belgium and 12 more

Added 15 new sites: Garran, Australia, Anderlecht, Belgium, Turnhout, Belgium and 12 more

sites_addedMyasthenia GravisJul 5Kite, A Gilead CompanyNCT07304154
Added 2 new sites: New York, New York, Westmead, Australia

Added 2 new sites: New York, New York, Westmead, Australia

sites_addedMyasthenia GravisJul 5Vertex Pharmaceuticals IncorporatedNCT07501702
Added 6 new sites: Jacksonville, Florida, Miami, Florida, Box Hill, Australia and 3 more

Added 6 new sites: Jacksonville, Florida, Miami, Florida, Box Hill, Australia and 3 more

Enrollment UpdateMultiple System AtrophyJul 5Kessler FoundationNCT06841198
Target enrollment increased from 5 to 15

Target enrollment increased from 5 to 15

eligibility_changeMultiple System AtrophyJul 5Kessler FoundationNCT06841198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 5Zenas BioPharma (USA), LLCNCT07299019
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_addedMultiple SclerosisJul 5Kite, A Gilead CompanyNCT07304154
Added 3 new sites: Palo Alto, California, New York, New York, Westmead, Australia

Added 3 new sites: Palo Alto, California, New York, New York, Westmead, Australia

sites_addedMultiple SclerosisJul 5Regeneron PharmaceuticalsNCT06351592
Added 1 new site: Umeå, Sweden

Added 1 new site: Umeå, Sweden

Enrollment UpdateMultiple SclerosisJul 5Regeneron PharmaceuticalsNCT06351592
Target enrollment increased from 42 to 54

Target enrollment increased from 42 to 54

sites_addedMultiple SclerosisJul 5OculisNCT07623668
Added 1 new site: Livonia, Michigan

Added 1 new site: Livonia, Michigan

eligibility_changeMultiple SclerosisJul 5Centre Hospitalier Universitaire de NīmesNCT07292480
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 5Zenas BioPharma (USA), LLCNCT07067463
Added 1 new site: Poznan, Poland

Added 1 new site: Poznan, Poland

sites_addedMixed Connective Tissue DiseaseJul 5Milton S. Hershey Medical CenterNCT06465199
Added 1 new site: Kansas City, Kansas

Added 1 new site: Kansas City, Kansas

eligibility_changeMixed Connective Tissue DiseaseJul 5University of FloridaNCT06625190
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMesotheliomaJul 5Memorial Sloan Kettering Cancer CenterNCT07532902
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyJul 5AstraZenecaNCT07571746
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedMembranous NephropathyJul 5Vertex Pharmaceuticals IncorporatedNCT07204275
Added 4 new sites: Clayton, Australia, Concord, Australia, Bari, Italy and 1 more

Added 4 new sites: Clayton, Australia, Concord, Australia, Bari, Italy and 1 more

eligibility_changeLight Chain AmyloidosisJul 4Alexion Pharmaceuticals, Inc.NCT07081646
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLennox-Gastaut SyndromeJul 4EpygenixNCT05066217
Added 6 new sites: Orlando, Florida, New York, New York, Philadelphia, Pennsylvania and 3 more

Added 6 new sites: Orlando, Florida, New York, New York, Philadelphia, Pennsylvania and 3 more

sites_addedIgG4-Related DiseaseJul 4SanofiNCT07190196
Added 3 new sites: Kitakyushu, Japan, Kobe, Japan, Nagaoka, Japan

Added 3 new sites: Kitakyushu, Japan, Kobe, Japan, Nagaoka, Japan

eligibility_changeIdiopathic Pulmonary FibrosisJul 4Hospital de GranollersNCT07482917
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJul 4Contineum TherapeuticsNCT07284459
Added 6 new sites: San Miguel de Tucumán, Argentina, Buenos Aires, Argentina, Santa Fe, Argentina and 3 more

Added 6 new sites: San Miguel de Tucumán, Argentina, Buenos Aires, Argentina, Santa Fe, Argentina and 3 more

eligibility_changeIdiopathic Pulmonary FibrosisJul 4Contineum TherapeuticsNCT07284459
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJul 4Avalyn Pharma Inc.NCT06951217
Added 7 new sites: Kansas City, Kansas, Baltimore, Maryland, Salt Lake City, Utah and 4 more

Added 7 new sites: Kansas City, Kansas, Baltimore, Maryland, Salt Lake City, Utah and 4 more

sites_addedIdiopathic Pulmonary FibrosisJul 4Rein TherapeuticsNCT06968845
Added 2 new sites: Salt Lake City, Utah, Warsaw, Poland

Added 2 new sites: Salt Lake City, Utah, Warsaw, Poland

sites_addedHypersensitivity PneumonitisJul 4Avalyn Pharma Inc.NCT06329401
Added 2 new sites: Mount Kisco, New York, Beaverton, Oregon

Added 2 new sites: Mount Kisco, New York, Beaverton, Oregon

sites_addedHuntington DiseaseJul 4Hoffmann-La RocheNCT07246941
Added 2 new sites: Clayton, Australia, Cambridge, United Kingdom

Added 2 new sites: Clayton, Australia, Cambridge, United Kingdom

eligibility_changeHodgkin LymphomaJul 4Alliance for Clinical Trials in OncologyNCT03150693
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 4National Cancer Institute (NCI)NCT06317662
Added 4 new sites: Chicago, Illinois, Des Moines, Iowa, Las Vegas, Nevada and 1 more

Added 4 new sites: Chicago, Illinois, Des Moines, Iowa, Las Vegas, Nevada and 1 more

sites_removedHodgkin LymphomaJul 4Swedish Orphan BiovitrumNCT06716814
Reduced from 24 to 21 sites

Reduced from 24 to 21 sites

eligibility_changeHodgkin LymphomaJul 4Taylor BrooksNCT06447376
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaJul 4Baylor College of MedicineNCT03690011
Target enrollment increased from 27 to 45

Target enrollment increased from 27 to 45

sites_addedHodgkin LymphomaJul 4Baylor College of MedicineNCT04664179
Added 2 new sites: Houston, Texas, Houston, Texas

Added 2 new sites: Houston, Texas, Houston, Texas

sites_addedHodgkin LymphomaJul 4Allogene TherapeuticsNCT06500273
Added 9 new sites: Chicago, Illinois, Fort Wayne, Indiana, Pittsburgh, Pennsylvania and 6 more

Added 9 new sites: Chicago, Illinois, Fort Wayne, Indiana, Pittsburgh, Pennsylvania and 6 more

eligibility_changeHodgkin LymphomaJul 4AstraZenecaNCT06526793
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJul 4Children's Oncology GroupNCT03959085
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJul 4Alliance for Clinical Trials in OncologyNCT07215910
Added 7 new sites: Chicago, Illinois, Glenview, Illinois, Highland Park, Illinois and 4 more

Added 7 new sites: Chicago, Illinois, Glenview, Illinois, Highland Park, Illinois and 4 more

sites_removedHodgkin LymphomaJul 4Fondazione Italiana Linfomi - ETSNCT05058404
Reduced from 71 to 54 sites

Reduced from 71 to 54 sites

sites_addedHemophilia AJul 3Swedish Orphan BiovitrumNCT06940830
Added 2 new sites: Catania, Italy, Padua, Italy

Added 2 new sites: Catania, Italy, Padua, Italy

sites_addedHemophilia AJul 3Wayne State UniversityNCT05022459
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedHemophilia AJul 3Novo Nordisk A/SNCT05685238
Added 1 new site: Tianjin, China

Added 1 new site: Tianjin, China

sites_addedGraves’ DiseaseJul 3Merida BiosciencesNCT07305818
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

eligibility_changeGraves’ DiseaseJul 3Merida BiosciencesNCT07305818
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseJul 3AmgenNCT06401044
Added 3 new sites: Montreal, Canada, Québec, Canada, Frankfurt am Main, Germany

Added 3 new sites: Montreal, Canada, Québec, Canada, Frankfurt am Main, Germany

sites_addedGraves’ DiseaseJul 3Biohaven Therapeutics Ltd.NCT07661056
Added 4 new sites: Burbank, California, Walnut Creek, California, Lenexa, Kansas and 1 more

Added 4 new sites: Burbank, California, Walnut Creek, California, Lenexa, Kansas and 1 more

sites_addedGlycogen Storage Disease Type IIJul 3Denali Therapeutics Inc.NCT07354724
Added 2 new sites: Irvine, California, Durham, North Carolina

Added 2 new sites: Irvine, California, Durham, North Carolina

sites_addedGeographic AtrophyJul 3SanofiNCT07215234
Added 1 new site: Austin, Texas

Added 1 new site: Austin, Texas

sites_removedFocal EpilepsyJul 3Biohaven Therapeutics Ltd.NCT06309966
Reduced from 174 to 152 sites

Reduced from 174 to 152 sites

Enrollment UpdateFanconi AnemiaJul 3Weill Medical College of Cornell UniversityNCT04613440
Target enrollment decreased from 820 to 524

Target enrollment decreased from 820 to 524

sites_addedEssential ThrombocythemiaJul 3Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Guangzhou, China, Yantai, China

Added 2 new sites: Guangzhou, China, Yantai, China

sites_addedDuchenne Muscular DystrophyJul 2ITF Therapeutics LLCNCT07127978
Added 2 new sites: Durham, North Carolina, Portland, Oregon

Added 2 new sites: Durham, North Carolina, Portland, Oregon

sites_addedDilated CardiomyopathyJul 2Affinia TherapeuticsNCT07426419
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

sites_addedChronic Graft-versus-Host DiseaseJul 2Nurix Therapeutics, Inc.NCT07221500
Added 2 new sites: Detroit, Michigan, Pittsburgh, Pennsylvania

Added 2 new sites: Detroit, Michigan, Pittsburgh, Pennsylvania

sites_addedChronic Graft-versus-Host DiseaseJul 2Fisher and Paykel HealthcareNCT07222410
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

eligibility_changeChronic Graft-versus-Host DiseaseJul 2Cosima LocherNCT06350786
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJul 2Brigham and Women's HospitalNCT07599345
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 2Kedrion S.p.A.NCT06752356
Added 1 new site: Lebanon, New Hampshire

Added 1 new site: Lebanon, New Hampshire

eligibility_changeChronic Graft-versus-Host DiseaseJul 2Kedrion S.p.A.NCT06752356
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 2BeOne MedicinesNCT06697184
Target enrollment increased from 56 to 258

Target enrollment increased from 56 to 258

eligibility_changeChronic Graft-versus-Host DiseaseJul 2BeOne MedicinesNCT06697184
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCholangiocarcinomaJul 2University of California, DavisNCT06101277
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaJul 2University of MiamiNCT06260943
Target enrollment increased from 100 to 210

Target enrollment increased from 100 to 210

sites_addedCholangiocarcinomaJul 2Astellas Pharma Global Development, Inc.NCT07488676
Added 2 new sites: Birmingham, Alabama, Indianapolis, Indiana

Added 2 new sites: Birmingham, Alabama, Indianapolis, Indiana

sites_addedCholangiocarcinomaJul 2Alentis Therapeutics AGNCT07169734
Added 13 new sites: Chicago, Illinois, Dijon, France, Marseille, France and 10 more

Added 13 new sites: Chicago, Illinois, Dijon, France, Marseille, France and 10 more

sites_addedDravet SyndromeJul 2EpygenixNCT04462770
Added 2 new sites: New Haven, Connecticut, New Delhi, India

Added 2 new sites: New Haven, Connecticut, New Delhi, India

eligibility_changeDravet SyndromeJul 2EpygenixNCT04462770
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedDravet SyndromeJul 2Stoke Therapeutics, IncNCT06872125
Reduced from 61 to 58 sites

Reduced from 61 to 58 sites

eligibility_changeDilated CardiomyopathyJul 2Emory UniversityNCT06464588
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeDesmoplastic Small Round Cell TumorJul 2University of FloridaNCT06625190
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCongenital Adrenal HyperplasiaJul 2Crinetics Pharmaceuticals Inc.NCT06712823
Added 4 new sites: Philadelphia, Pennsylvania, Curitiba, Brazil, Porto Alegre, Brazil and 1 more

Added 4 new sites: Philadelphia, Pennsylvania, Curitiba, Brazil, Porto Alegre, Brazil and 1 more

Enrollment UpdateCongenital Adrenal HyperplasiaJul 2Crinetics Pharmaceuticals Inc.NCT06712823
Target enrollment increased from 150 to 200

Target enrollment increased from 150 to 200

eligibility_changeCongenital Adrenal HyperplasiaJul 2Crinetics Pharmaceuticals Inc.NCT06712823
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 2Kite, A Gilead CompanyNCT07304154
Added 2 new sites: New York, New York, Westmead, Australia

Added 2 new sites: New York, New York, Westmead, Australia

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 2Kedrion S.p.A.NCT06752356
Added 1 new site: Lebanon, New Hampshire

Added 1 new site: Lebanon, New Hampshire

eligibility_changeChronic Inflammatory Demyelinating PolyneuropathyJul 2Kedrion S.p.A.NCT06752356
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 2BayerNCT05457283
Added 23 new sites: Buenos Aires, Argentina, Buenos Aires, Argentina, San Luis, Argentina and 20 more

Added 23 new sites: Buenos Aires, Argentina, Buenos Aires, Argentina, San Luis, Argentina and 20 more

sites_addedChronic Graft-versus-Host DiseaseJul 2Kite, A Gilead CompanyNCT07304154
Added 3 new sites: Palo Alto, California, New York, New York, Westmead, Australia

Added 3 new sites: Palo Alto, California, New York, New York, Westmead, Australia

eligibility_changeChronic Graft-versus-Host DiseaseJul 2Umoja BiopharmaNCT06528301
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 2argenxNCT07194850
Added 2 new sites: Berlin, Germany, Roma, Italy

Added 2 new sites: Berlin, Germany, Roma, Italy

sites_addedChronic Graft-versus-Host DiseaseJul 2Merit Medical Systems, Inc.NCT06422871
Added 4 new sites: Washington D.C., District of Columbia, Pittsburgh, Pennsylvania, Houston, Texas and 1 more

Added 4 new sites: Washington D.C., District of Columbia, Pittsburgh, Pennsylvania, Houston, Texas and 1 more

sites_addedChronic Graft-versus-Host DiseaseJul 2Merck Sharp & Dohme LLCNCT06136559
Added 3 new sites: Owensboro, Kentucky, Hefei, China, Wuhan, China

Added 3 new sites: Owensboro, Kentucky, Hefei, China, Wuhan, China

sites_addedChronic Graft-versus-Host DiseaseJul 2Boehringer IngelheimNCT06531824
Added 32 new sites: Beverly Hills, California, Atlanta, Georgia, Louisville, Kentucky and 29 more

Added 32 new sites: Beverly Hills, California, Atlanta, Georgia, Louisville, Kentucky and 29 more

sites_removedChronic Graft-versus-Host DiseaseJul 2GlaxoSmithKlineNCT04398745
Reduced from 19 to 4 sites

Reduced from 19 to 4 sites

sites_addedChronic Graft-versus-Host DiseaseJul 2Janssen Research & Development, LLCNCT06788509
Added 2 new sites: Nedlands, Australia, Barcelona, Spain

Added 2 new sites: Nedlands, Australia, Barcelona, Spain

sites_addedChronic Graft-versus-Host DiseaseJul 2REVA Medical, Inc.NCT05406622
Added 13 new sites: El Centro, California, Fremont, California, Stanford, California and 10 more

Added 13 new sites: El Centro, California, Fremont, California, Stanford, California and 10 more

eligibility_changeChronic Graft-versus-Host DiseaseJul 2George Washington UniversityNCT06243068
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJul 2CytokineticsNCT06736574
Added 21 new sites: Beijing, China, Yuncheng, China, Tianjin, China and 18 more

Added 21 new sites: Beijing, China, Yuncheng, China, Tianjin, China and 18 more

sites_addedCholangiocarcinomaJul 2Kestrel Therapeutics, Inc.NCT07458347
Added 1 new site: Salt Lake City, Utah

Added 1 new site: Salt Lake City, Utah

eligibility_changeCholangiocarcinomaJul 2National Cancer Institute (NCI)NCT06050252
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedCholangiocarcinomaJul 2J-Pharma Co., Ltd.NCT07265674
Reduced from 23 to 18 sites

Reduced from 23 to 18 sites

sites_addedCholangiocarcinomaJul 2University Hospital, AngersNCT06990659
Added 5 new sites: Marseille, France, Marseille, France, Nantes, France and 2 more

Added 5 new sites: Marseille, France, Marseille, France, Nantes, France and 2 more

eligibility_changeCholangiocarcinomaJul 2University Hospital, AngersNCT06990659
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCholangiocarcinomaJul 2Guangxi Medical UniversityNCT07658586
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 1PrileniaNCT07322003
Added 2 new sites: Tampa, Florida, Milan, Italy

Added 2 new sites: Tampa, Florida, Milan, Italy

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 1BayerNCT07298044
Added 13 new sites: Feldkirch, Austria, Brussels, Belgium, Bruges, Belgium and 10 more

Added 13 new sites: Feldkirch, Austria, Brussels, Belgium, Bruges, Belgium and 10 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 1AstraZenecaNCT07338942
Added 40 new sites: Beijing, China, Beijing, China, Beijing, China and 37 more

Added 40 new sites: Beijing, China, Beijing, China, Beijing, China and 37 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 1Eidos Therapeutics, a BridgeBio companyNCT06563895
Added 3 new sites: CABA, Argentina, Aarhus, Denmark, A Coruña, Spain

Added 3 new sites: CABA, Argentina, Aarhus, Denmark, A Coruña, Spain

sites_addedAmyotrophic Lateral SclerosisJul 1Centre hospitalier de l'Université de Montréal (CHUM)NCT06051123
Added 2 new sites: Fredericton, Canada, Saskatoon, Canada

Added 2 new sites: Fredericton, Canada, Saskatoon, Canada

sites_addedAmyotrophic Lateral SclerosisJul 1PrileniaNCT07322003
Added 2 new sites: Milan, Italy, Umeå, Sweden

Added 2 new sites: Milan, Italy, Umeå, Sweden

sites_addedAmyotrophic Lateral SclerosisJul 1Washington University School of MedicineNCT07294144
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedAcromegalyJul 1Camurus ABNCT07583563
Added 1 new site: Würzburg, Germany

Added 1 new site: Würzburg, Germany

sites_addedAchondroplasiaJul 1BioMarin PharmaceuticalNCT06168201
Added 1 new site: Baltimore, Maryland

Added 1 new site: Baltimore, Maryland

sites_addedAchondroplasiaJul 1Tyra Biosciences, IncNCT06842355
Added 1 new site: Stockholm, Sweden

Added 1 new site: Stockholm, Sweden