26 completed, 15 terminated, 7 active not recruiting, 5 enrolling by invitation. 31 Phase 1, 22 Phase 2, 9 Phase 3, 2 Phase 4.
By phase and status
Phase Trials Status Phase 1 31 Completed 15 , Terminated 10 , Enrolling by invitation 2 , Recruiting 2 , Active not recruiting 1 , Withdrawn 1 Phase 2 22 Completed 13 , Terminated 6 , Enrolling by invitation 2 , Active not recruiting 1 Phase 3 9 Active not recruiting 3 , Completed 3 , Enrolling by invitation 1 , Terminated 1 , Withdrawn 1 Phase 4 2 Recruiting 1 , Terminated 1
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix .
Trial Phase Status Sponsor Enrollment Start Updated
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting ELEVIDYS, Sirolimus, Glucocorticoids - Duchenne Muscular Dystrophy Phase 4 Recruiting Sarepta Therapeutics, Inc. 20 2026-10-30 2026-10-06 A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease SRP-1005, Placebo - Huntington's Disease Phase 1 Recruiting Sarepta Therapeutics, Inc. 32 2026-05-12 2026-10-19 A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants SRP-9005, Corticosteroid - Muscular Dystrophies, Limb-Girdle Phase 3 Withdrawn Sarepta Therapeutics, Inc. 2025-06-30 2025-08-06 Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb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 States SRP-9004 - Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3 Phase 1 Terminated Sarepta Therapeutics, Inc. 4 2025-01-09 2025-09-04 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 AAVrh74 delandistrogene moxeparvovec, Plasmapheresis - Duchenne Muscular Dystrophy Phase 1 Terminated Sarepta Therapeutics, Inc. 3 2024-09-18 2025-09-04 Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1 SRP-1001 for Injection, Placebo - Muscular Dystrophy, Facioscapulohumeral Phase 1 / Phase 2 Enrolling by invitation Sarepta Therapeutics, Inc. 60 2024-06-19 2026-10-05 Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy SRP-1003 IV Infusion, Placebo IV Infusion, SRP-1003 SC Injection - Myotonic Dystrophy 1 Phase 1 / Phase 2 Enrolling by invitation Sarepta Therapeutics, Inc. 78 2024-03-04 2026-10-05 An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy Delandistrogene Moxeparvovec, Standard of Care - Duchenne Muscular Dystrophy Enrolling by invitation Sarepta Therapeutics, Inc. 500 2024-02-07 2026-05-04 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) delandistrogene moxeparvovec, imlifidase - Duchenne Muscular Dystrophy Phase 1 Terminated Sarepta Therapeutics, Inc. 5 2024-01-29 2025-11-20 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/R4 SRP-9003, Glucocorticoid - Limb-girdle Muscular Dystrophy Phase 3 Active not recruiting Sarepta Therapeutics, Inc. 17 2024-01-15 2026-03-02 A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) delandistrogene moxeparvovec - Duchenne Muscular Dystrophy Phase 2 Active not recruiting Hoffmann-La Roche 13 2023-11-29 2026-09-09 A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study delandistrogene moxeparvovec - Duchenne Muscular Dystrophy Phase 3 Enrolling by invitation Sarepta Therapeutics, Inc. 400 2023-09-27 2026-02-06 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) delandistrogene moxeparvovec, placebo - Duchenne Muscular Dystrophy Phase 3 Active not recruiting Sarepta Therapeutics, Inc. 148 2023-05-31 2026-05-22 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) SRP-6004 - Limb Girdle Muscular Dystrophy Phase 1 Terminated Sarepta Therapeutics, Inc. 2 2023-05-22 2025-09-04 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) SRP-9003 - Limb Girdle Muscular Dystrophy Phase 1 Active not recruiting Sarepta Therapeutics, Inc. 6 2022-12-19 2026-02-19 Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies Classical conditioning task - DMD, BMD Unknown University College, London 100 2022-09-13 2024-09-03 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) delandistrogene moxeparvovec, placebo - Duchenne Muscular Dystrophy Phase 3 Completed Sarepta Therapeutics, Inc. 126 2021-10-27 2025-07-08 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 Practice Limb-girdle Muscular Dystrophy Active not recruiting Sarepta Therapeutics, Inc. 205 2021-04-22 2025-11-06 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 Cohort delandistrogene moxeparvovec - Duchenne Muscular Dystrophy Phase 1 Recruiting Sarepta Therapeutics, Inc. 83 2020-11-23 2026-09-11 An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53] - Duchenne Muscular Dystrophy Phase 4 Terminated Rare Disease Research, LLC 2 2020-10-31 2026-04-03
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Conditions studied Muscular Dystrophy, Duchenne (33) · Muscular Dystrophies, Limb-Girdle (9) · Coronary Artery Disease (2) · Hemorrhagic Fever, Ebola (2) · Hepatitis C (2) · Limb-girdle muscular dystrophy, type 2B (2) · Marburg Virus Disease (2) · Muscular Dystrophies (2) · Sarcoglycanopathies (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1)
Interventions eteplirsen (12) · casimersen (5) · golodirsen (5) · Glucocorticoids (2) · Injections (2) · Prednisone (2) · Saline Solution (2) · AVI-4126 (1) · AVI-5126 (1) · AVI-7288 (1) · Adrenal Cortex Hormones (1) · Anti-Bacterial Agents (1) · Mac-1-like protein, Streptococcus (1) · Plasmapheresis (1) · Sirolimus (1)
Countries United States (43) · United Kingdom (18) · Italy (15) · Germany (13) · Spain (13) · Belgium (12) · Canada (9) · France (8) · Australia (5) · Taiwan (5) · New Zealand (4) · Sweden (4) · Czechia (3) · Hong Kong (3) · Israel (3)
Top investigators Alison Heald, MD (4) · Paula M Shaw, MD (2) · Apinya B Vutikullird, MD (1) · Austen Eddy, MSM (1) · Christine Hale, MD (1) · Christine McSherry, R.N. (1) · Curt Scharfe, MD, PhD (1) · Francesco Muntoni, FRCPCH (1) · Francesco Muntoni, Professor (1) · Jerry R Mendell, MD (1) · Kate Bushby, MRCP (1) · Kevin Flanigan, MD (1) · Mark Holodniy, M.D. (1)
Results publications PMID 37539981 , PMID 26573217 , PMID 38753764 , PMID 19713152 , PMID 21784508 , PMID 23907995 , PMID 26200980 , PMID 31315600 , PMID 32539076 , PMID 34105177 , PMID 34788571 , PMID 35142618 , PMID 37207382 , PMID 38177855 , PMID 39385046 , PMID 39589719 , PMID 39747998 , PMID 40098309 , PMID 40354061 , PMID 41182527 , PMID 41518520 , PMID 41961051
NIH funding 2019 (3), 2023 (3), 2015 (1), 2017 (1), 2018 (1), U01AR060911 (1)