50
Trials as lead sponsor
Most active programmes
The drugs appearing most often across Sarepta Therapeutics's recently active trials. Comparator arms are excluded.
- delandistrogene moxeparvovec8 trials
- Eteplirsen5 trials
- SRP-90033 trials
- SRP-40452 trials
- SRP-40532 trials
- Golodirsen2 trials
Top therapeutic areas
Conditions studied most often across Sarepta Therapeutics's recently active trials.
- Duchenne Muscular Dystrophy19 trials
- Muscular Dystrophy, Duchenne5 trials
- Limb Girdle Muscular Dystrophy3 trials
- Duchenne Muscular Dystrophy (DMD)3 trials
- Limb-girdle Muscular Dystrophy2 trials
- Marburg Hemorrhagic Fever2 trials
Sarepta Therapeutics trials
The 50 most significant of Sarepta Therapeutics's recently active trials, soonest estimated readout first.
Select any row for the full record; the change timeline unlocks with tracking.
| Trial | Phase | Stability | Status | Enrollment | Est. readout |
|---|---|---|---|---|---|
| MIS51ONA Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)NCT03992430Eteplirsen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 160 est. | 31 Oct 2026 |
| ENHANCEStudy to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing SettingNCT07542314ELEVIDYS | Phase 4 | Locked · track to reveal | OngoingNot yet recruiting | 20 est. | 31 Mar 2027 |
| ENVISIONA 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)NCT05881408delandistrogene moxeparvovec | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 148 est. | 31 May 2027 |
| A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's DiseaseNCT07536061SRP-1005 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 32 est. | 30 Nov 2027 |
| ENDEAVORA 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 CohortNCT04626674delandistrogene moxeparvovec | Phase 1 | Locked · track to reveal | OngoingRecruiting | 83 est. | 31 Dec 2027 |
| 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)NCT05876780SRP-9003 | Phase 1 | Locked · track to reveal | OngoingActive, not recruiting | 6 est. | 28 Aug 2028 |
| ENDUREAn Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular DystrophyNCT06270719Delandistrogene Moxeparvovec | — | Not yet scored | OngoingEnrolling by invitation | 500 est. | 31 Dec 2029 |
| 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 PracticeNCT04475926 | — | Not yet scored | OngoingActive, not recruiting | 205 est. | 5 Sept 2030 |
| COMPASSA Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult ParticipantsNCT06952686SRP-9005 | Phase 3 | Locked · track to reveal | StoppedWithdrawn | 0 est. | 30 Nov 2030 |
| EXPEDITIONA Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical StudyNCT05967351delandistrogene moxeparvovec | Phase 3 | Locked · track to reveal | OngoingEnrolling by invitation | 400 est. | 31 Oct 2033 |
| EVOLVEA Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical PracticeNCT06606340Eteplirsen | — | Not yet scored | OngoingEnrolling by invitation | 300 est. | 31 Dec 2033 |
| 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)NCT06241950delandistrogene moxeparvovec | Phase 1 | Locked · track to reveal | StoppedTerminated | 5 est. | 10 Oct 2025 |
| HORIZONA 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 AAVrh74NCT06597656delandistrogene moxeparvovec | Phase 1 | Locked · track to reveal | StoppedTerminated | 3 est. | 5 Aug 2025 |
| DISCOVERYStudy 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 StatesNCT06747273SRP-9004 | Phase 1 | Locked · track to reveal | StoppedTerminated | 4 est. | 18 Jun 2025 |
| 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)NCT05906251SRP-6004 | Phase 1 | Locked · track to reveal | StoppedTerminated | 2 est. | 13 Jun 2025 |
| EMERGENEA Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4NCT06246513SRP-9003 | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 17 est. | 4 Mar 2025 |
| Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)NCT03652259SRP-9003 | Phase 1 | Locked · track to reveal | StoppedTerminated | 6 est. | 14 Jan 2025 |
| ESSENCEStudy of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)NCT02500381SRP-4045 | Phase 3 | Locked · track to reveal | Completed | 228 est. | 12 Nov 2024 |
| MOMENTUMTwo-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 TreatmentNCT04004065Vesleteplirsen | Phase 2 | Locked · track to reveal | StoppedTerminated | 62 est. | 30 Oct 2023 |
| EMBARKA Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)NCT05096221delandistrogene moxeparvovec | Phase 3 | Locked · track to reveal | Completed | 126 est. | 4 Oct 2023 |
| An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophyNCT03532542Casimersen | Phase 3 | Locked · track to reveal | StoppedTerminated | 171 est. | 26 Jul 2023 |
| A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)NCT03375164delandistrogene moxeparvovec | Phase 1 | Locked · track to reveal | Completed | 4 est. | 25 Apr 2023 |
| A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)NCT03985878Eteplirsen | Phase 2 | Locked · track to reveal | StoppedTerminated | 15 est. | 31 Aug 2022 |
| An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)NCT03675126SRP-5051 | Phase 1 | Locked · track to reveal | StoppedTerminated | 15 est. | 25 Aug 2021 |
| Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 SkippingNCT03218995Eteplirsen | Phase 2 | Locked · track to reveal | Completed | 15 est. | 10 Mar 2021 |
| A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)NCT03769116delandistrogene moxeparvovec | Phase 1 | Locked · track to reveal | Completed | 41 est. | 8 Dec 2020 |
| A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)NCT03375255SRP-5051 | Phase 1 | Locked · track to reveal | Completed | 15 est. | 19 Aug 2019 |
| rAAVrh74.MHCK7.DYSF.DV for Treatment of DysferlinopathiesNCT02710500rAAVrh74.MHCK7.DYSF.DV | Phase 1 | Locked · track to reveal | Completed | 2 est. | Jul 2019 |
| PROMOVIStudy of Eteplirsen in DMD PatientsNCT02255552eteplirsen | Phase 3 | Locked · track to reveal | Completed | 109 est. | 14 Jun 2019 |
| Phase I/II Study of SRP-4053 in DMD PatientsNCT02310906SRP-4053 | Phase 1 | Locked · track to reveal | Completed | 39 est. | 25 Mar 2019 |
| A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)NCT01976091SRP-9004 | Phase 1 | Locked · track to reveal | Completed | 6 est. | 14 Mar 2019 |
| Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyNCT02420379eteplirsen | Phase 2 | Locked · track to reveal | Completed | 33 est. | 17 Dec 2018 |
| Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) PatientsNCT02530905SRP-4045 | Phase 1 | Locked · track to reveal | Completed | 12 est. | 3 Oct 2018 |
| Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyNCT02286947Eteplirsen | Phase 2 | Locked · track to reveal | Completed | 24 est. | 21 Apr 2017 |
| Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyNCT01540409AVI-4658 (Eteplirsen) | Phase 2 | Locked · track to reveal | Completed | 12 est. | 15 Apr 2016 |
| A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7288 in Healthy Adult VolunteersNCT01566877AVI-7288 | Phase 1 | Locked · track to reveal | Completed | 40 est. | Jan 2014 |
| A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7537 in Healthy Adult VolunteersNCT01593072AVI-7537 | Phase 1 | Locked · track to reveal | StoppedWithdrawn | 0 est. | Jan 2013 |
| Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsNCT01396239AVI-4658 (Eteplirsen) | Phase 2 | Locked · track to reveal | Completed | 12 est. | Feb 2012 |
| Safety Study of Single Administration Post-exposure Prophylaxis Treatment for Marburg VirusNCT01353040AVI-6003 | Phase 1 | Locked · track to reveal | Completed | 30 est. | Dec 2011 |
| Safety Study of Single Administration Post-Exposure Prophylaxis Treatment for Ebola VirusNCT01353027AVI-6002 | Phase 1 | Locked · track to reveal | Completed | 30 est. | Nov 2011 |
| Safety Study of Single Administration Intravenous Treatment for InfluenzaNCT01375985AVI-7100 | Phase 1 | Locked · track to reveal | StoppedTerminated | 8 est. | Aug 2011 |
| Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsNCT00844597AVI-4658 for Injection | Phase 1 | Locked · track to reveal | Completed | 19 est. | Jun 2010 |
| Safety and Efficacy Study of AVI-5126 When Used on Vein Grafts Before Use in Heart by-Pass Graft Surgery (CABG)NCT00451256AVI-5126 | Phase 2 | Locked · track to reveal | StoppedTerminated | 600 est. | Nov 2007 |
| Pharmacokinetic Study in Cerebral Spinal Fluid After a Single Dose of AVI-4020NCT00387283AVI-4020 Injection | Phase 1 | Locked · track to reveal | Completed | 14 est. | Dec 2006 |
| Pharmacokinetic Study of a Single Dose of AVI-4065 in Cerebral Spinal FluidNCT00381433AVI-4065 Injection | Phase 1 | Locked · track to reveal | Completed | 12 est. | Oct 2006 |
| Study of AVI-4065 in Healthy Volunteers and Chronic Active HCV PatientsNCT00229749AVI-4065 Injection | Phase 1 | Locked · track to reveal | StoppedTerminated | 80 est. | Oct 2006 |
| Pharmacokinetic Study of a Single Dose of AVI-4126 (RESTEN-NG®) in Cerebral Spinal FluidNCT00343148AVI-4126 Injection (RESTEN-NG®) | Phase 1 | Locked · track to reveal | Completed | 6 est. | Jun 2006 |
| Safety and Efficacy of RESTEN-MP When Used in Conjunction With a Bare Metal Stent in Coronary ArteriesNCT00248066RESTEN-MP | Phase 2 | Locked · track to reveal | Completed | 50 est. | — |
| An Exploratory Study of AVI-4020 in Patients With Possible Acute Neuroinvasive West Nile Virus (WNV) DiseaseNCT00091845AVI-4020 Injection | Phase 1 | Locked · track to reveal | StoppedTerminated | 50 est. | — |
| A Phase 1b Study Evaluating RESTEN-MP in Subjects With Focal de Novo StenosisNCT00244647RESTEN-MP | Phase 1 | Locked · track to reveal | StoppedTerminated | 40 est. | — |