Sarepta Therapeutics, Inc.: Muscular Dystrophy, Duchenne trials

The registry lists 27 Muscular Dystrophy, Duchenne studies with Sarepta Therapeutics, Inc. as lead sponsor. 5 are open to participants now and 15 have results posted.

Phase 1 10Phase 2 12Phase 3 7Phase 4 1No phase 2(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing SettingRecruitingNCT07542314Phase 4
• A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical PracticeEnrolling by invitationNCT06606340
• An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular DystrophyEnrolling by invitationNCT06270719
• A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical StudyEnrolling by invitationNCT05967351Phase 3
• A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory CohortRecruitingNCT04626674Phase 1
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)CompletedNCT05096221Phase 3Results posted
• A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)TerminatedNCT03985878Phase 2Results posted
• A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)CompletedNCT03769116Phase 1/2Results posted
• An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)TerminatedNCT03675126Phase 1/2Results posted
• An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophyTerminatedNCT03532542Phase 3Results posted
• A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)CompletedNCT03375164Phase 1/2Results posted
• Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 SkippingCompletedNCT03218995Phase 2Results posted
• Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) PatientsCompletedNCT02530905Phase 1Results posted
• Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyCompletedNCT02420379Phase 2Results posted
• Phase I/II Study of SRP-4053 in DMD PatientsCompletedNCT02310906Phase 1/2Results posted
• Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyCompletedNCT02286947Phase 2Results posted
• Study of Eteplirsen in DMD PatientsCompletedNCT02255552Phase 3Results posted
• Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyCompletedNCT01540409Phase 2Results posted
• Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsCompletedNCT01396239Phase 2Results posted
• Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsCompletedNCT00844597Phase 1/2Results posted
• A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74TerminatedNCT06597656Phase 1
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)TerminatedNCT06241950Phase 1
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)Active, not recruitingNCT05881408Phase 3
• Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentTerminatedNCT04004065Phase 2
• A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)Active, not recruitingNCT03992430Phase 3
• A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)CompletedNCT03375255Phase 1
• Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)CompletedNCT02500381Phase 3