28 completed, 15 terminated, 5 active not recruiting, 5 recruiting. 59 Phase 3, 10 Phase 2.
Follow Muscular Dystrophy, Duchenne: new trials by email Literature Review of Muscular Dystrophy, Duchenne trials Research Report on Muscular Dystrophy, Duchenne
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 |
|---|---|---|---|---|---|---|
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 70 | 2026-09-01 | 2026-10-13 |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) Zeleciment Rostudirsen (DYNE-251), Placebo - Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne | Phase 3 | Recruiting | Dyne Therapeutics | 90 | 2026-06-01 | 2026-05-27 |
| A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) SGT-003, Placebo - Duchenne Muscular Dystrophy | Phase 3 | Recruiting | Solid Biosciences Inc. | 80 | 2025-10-22 | 2026-09-28 |
| Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy Givinostat, Placebo - Duchenne Muscular Dystrophy | Phase 3 | Recruiting | Italfarmaco | 138 | 2024-02-19 | 2026-07-24 |
| 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 Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. fordadistrogene movaparvovec - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Pfizer | 7 | 2023-03-13 | 2025-10-21 |
| AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) RGX-202 - Duchenne Muscular Dystrophy | Phase 2 / Phase 3 | Active not recruiting | REGENXBIO Inc. | 65 | 2023-01-04 | 2026-07-21 |
| A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy Deramiocel (CAP-1002), Placebo - Muscular Dystrophies, Muscular Dystrophy, Duchenne | Phase 3 | Active not recruiting | Capricor Inc. | 106 | 2022-06-22 | 2026-08-28 |
| Tadalafil as Adjuvant Therapy for DMD Tadalafil - Duchenne Muscular Dystrophy | Phase 2 / Phase 3 | Completed | University of Florida | 12 | 2021-12-14 | 2026-10-27 |
| 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 |
| The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD Metoprolol Succinate, Placebo - Muscular Dystrophy, Duchenne | Phase 3 | Unknown | Medical University of Gdansk | 150 | 2021-08-18 | 2021-10-04 |
| Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) Viltolarsen - Duchenne Muscular Dystrophy | Phase 3 | Unknown | NS Pharma, Inc. | 74 | 2021-04-13 | 2024-02-14 |
| Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD Pamrevlumab, Placebo, Corticosteroids - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Kyntra Bio | 73 | 2021-03-03 | 2024-08-26 |
| Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy PF-06939926, Placebo - Duchenne Muscular Dystrophy | Phase 3 | Active not recruiting | Pfizer | 114 | 2020-11-05 | 2026-07-22 |
| A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD） TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort], Placebo [Ambulatory Cohort] only - Duchenne Muscular Dystrophy | Phase 3 | Completed | Taiho Pharmaceutical Co., Ltd. | 104 | 2020-11-01 | 2026-08-17 |
| Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) Pamrevlumab, Placebo, Corticosteroids - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Kyntra Bio | 98 | 2020-08-10 | 2024-03-12 |
| A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) Eteplirsen - Muscular Dystrophy, Duchenne | Phase 3 | Active not recruiting | Sarepta Therapeutics, Inc. | 160 | 2020-07-13 | 2026-02-27 |
| Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) Viltolarsen, Placebo - Duchenne Muscular Dystrophy | Phase 3 | Completed | NS Pharma, Inc. | 77 | 2020-04-14 | 2024-12-11 |
| Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy WVE-210201 (suvodirsen), Placebo - Duchenne Muscular Dystrophy | Phase 2 / Phase 3 | Terminated | Wave Life Sciences USA, Inc. | 6 | 2019-09-04 | 2021-05-20 |
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Muscular Dystrophies (20) · Neuromuscular Diseases (11) · Genetic Diseases, Inborn (8) · Musculoskeletal Diseases (7) · Genetic Diseases, X-Linked (6) · Muscular Diseases (6) · Muscular Disorders, Atrophic (6) · Nervous System Diseases (6) · Cardiomyopathies (2) · Myotonic Dystrophy (2) · Osteoporosis (2) · Amyotrophic Lateral Sclerosis (1) · Cardiomyopathy, Dilated (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (58) · Genetic Diseases, X-Linked (53) · Muscular Diseases (53) · Muscular Disorders, Atrophic (53) · Nervous System Diseases (53) · Musculoskeletal Diseases (52) · Genetic Diseases, Inborn (51) · Neuromuscular Diseases (48) · Muscular Dystrophies (39) · Heredodegenerative Disorders, Nervous System (3) · Metabolic Diseases (3) · Myotonic Disorders (3) · Neurodegenerative Diseases (3) · Nutritional and Metabolic Diseases (3) · Bone Diseases (2)
ataluren (5) · Prednisone (4) · givinostat (3) · idebenone (3) · Adrenal Cortex Hormones (2) · Creatine (2) · Glutamine (2) · PRO051 (2) · Tadalafil (2) · Zoledronic Acid (2) · casimersen (2) · coenzyme Q10 (2) · deflazacort (2) · edasalonexent (2) · eteplirsen (2)
Sarepta Therapeutics, Inc. (7) · PTC Therapeutics (6) · Cooperative International Neuromuscular Research Group (4) · GlaxoSmithKline (3) · Italfarmaco (3) · Santhera Pharmaceuticals (3) · Assistance Publique - Hôpitaux de Paris (2) · Catabasis Pharmaceuticals (2) · Kyntra Bio (2) · NS Pharma, Inc. (2) · National Center for Research Resources (NCRR) (2) · Novartis Pharmaceuticals (2) · Pfizer (2) · University Hospital, Basel, Switzerland (2) · Avidity Biosciences, Inc. (1)
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Craig McDonald, MD (2) · Dirk Fischer, MD (2) · Vinay Penematsa, MD (2) · Ana Camacho Salas, MD (1) · Ass. Prof. Jan Verschuuren, MD, PhD (1) · Barry Byrne, MD (1) · Bryan W. Burnette, MD (1)
PMID 15984021, PMID 17389552, PMID 17450125, PMID 25907158, PMID 27488051, PMID 28189481, PMID 28728956, PMID 28972192, PMID 29203355, PMID 30135256, PMID 31549577, PMID 31664444, PMID 31752977, PMID 32851872, PMID 34105177, PMID 34120912, PMID 34228102, PMID 34383312, PMID 34693725, PMID 34791888, PMID 34857536, PMID 35381069, PMID 37739572, PMID 38190001, PMID 38508835, PMID 39385046, PMID 39589719, PMID 39879732, PMID 40098309, PMID 40217158, PMID 40354061, PMID 40830818, PMID 41182527, PMID 41518520, PMID 41671094, PMID 41691937, PMID 41722591, PMID 41879641, PMID 42139655, PMID 42202243, PMID 42249273, PMID 42443978, PMID 42526472
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