Sarepta Therapeutics, Inc.: all 52 studies

Page 1 of 1. Open to participants first (8), 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 First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's DiseaseRecruitingNCT07536061Phase 1
• 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
• Study of SRP-1003 in Participants With Type 1 Myotonic DystrophyEnrolling by invitationNCT06138743Phase 1/2
• Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1Enrolling by invitationNCT06131983Phase 1/2
• 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
• A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)CompletedNCT01976091Phase 1/2Results 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 Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult ParticipantsWithdrawnNCT06952686Phase 3
• Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United StatesTerminatedNCT06747273Phase 1
• 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 Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4Active, not recruitingNCT06246513Phase 3
• 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 Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)TerminatedNCT05906251Phase 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
• A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)Active, not recruitingNCT05876780Phase 1
• A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical PracticeActive, not recruitingNCT04475926
• 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
• Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)TerminatedNCT03652259Phase 1/2
• 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
• rAAVrh74.MHCK7.DYSF.DV for Treatment of DysferlinopathiesCompletedNCT02710500Phase 1
• Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)CompletedNCT02500381Phase 3
• A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7537 in Healthy Adult VolunteersWithdrawnNCT01593072Phase 1
• A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7288 in Healthy Adult VolunteersCompletedNCT01566877Phase 1
• Safety Study of Single Administration Intravenous Treatment for InfluenzaTerminatedNCT01375985Phase 1
• Safety Study of Single Administration Post-exposure Prophylaxis Treatment for Marburg VirusCompletedNCT01353040Phase 1
• Safety Study of Single Administration Post-Exposure Prophylaxis Treatment for Ebola VirusCompletedNCT01353027Phase 1
• Safety and Efficacy Study of AVI-5126 When Used on Vein Grafts Before Use in Heart by-Pass Graft Surgery (CABG)TerminatedNCT00451256Phase 2
• Pharmacokinetic Study in Cerebral Spinal Fluid After a Single Dose of AVI-4020CompletedNCT00387283Phase 1
• Pharmacokinetic Study of a Single Dose of AVI-4065 in Cerebral Spinal FluidCompletedNCT00381433Phase 1
• Pharmacokinetic Study of a Single Dose of AVI-4126 (RESTEN-NG®) in Cerebral Spinal FluidCompletedNCT00343148Phase 1
• Safety and Efficacy of RESTEN-MP When Used in Conjunction With a Bare Metal Stent in Coronary ArteriesCompletedNCT00248066Phase 2
• A Phase 1b Study Evaluating RESTEN-MP in Subjects With Focal de Novo StenosisTerminatedNCT00244647Phase 1
• Study of AVI-4065 in Healthy Volunteers and Chronic Active HCV PatientsTerminatedNCT00229749Phase 1/2
• An Exploratory Study of AVI-4020 in Patients With Possible Acute Neuroinvasive West Nile Virus (WNV) DiseaseTerminatedNCT00091845Phase 1

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