Clinical Trials Directory

Trials / Conditions / Genetic Diseases, X-Linked

Genetic Diseases, X-Linked

13 registered clinical trials studyying Genetic Diseases, X-Linked — 1 currently recruiting.

StatusTrialSponsorPhase
RecruitingRett Syndrome Registry
NCT05432349
International Rett Syndrome Foundation—
Active Not RecruitingA Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
NCT05126758
Capricor Inc.Phase 3
CompletedA Trial of Metformin in Individuals With Fragile X Syndrome (Met)
NCT03862950
University of AlbertaPhase 2
CompletedDose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals
NCT03734588
Spark Therapeutics, Inc.Phase 1 / Phase 2
CompletedLead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy
NCT03876301
Spark Therapeutics, Inc.—
CompletedA Trial of Metformin in Individuals With Fragile X Syndrome
NCT03479476
University of California, DavisPhase 2 / Phase 3
CompletedA Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
NCT03406780
Capricor Inc.Phase 2
TerminatedKidney Information Network for Disease Research and Education
NCT03321604
Massachusetts General Hospital—
CompletedLong-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT03179631
PTC TherapeuticsPhase 3
TerminatedAn Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT02090959
PTC TherapeuticsPhase 3
CompletedPhase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01826487
PTC TherapeuticsPhase 3
CompletedGenetic Studies of X-linked Lymphoproliferative Disease
NCT00359411
National Institute of Allergy and Infectious Diseases (NIAID)—
AvailableManaged Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
NCT07250737
Avidity Biosciences, Inc.—