147 completed, 39 terminated, 37 unknown, 27 active not recruiting. 95 Phase 2, 59 Phase 1, 47 Not applicable, 36 Phase 3, 9 Phase 4.
Follow Duchenne Muscular Dystrophy: new trials by email Literature Review of Duchenne Muscular Dystrophy trials Research Report on Duchenne Muscular Dystrophy
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 |
| Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy KER-065 - Duchenne Muscular Dystrophy | Phase 2 | Recruiting | Keros Therapeutics, Inc. | 36 | 2026-09-25 | 2026-09-30 |
| Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy Alternate-day prednisolone, Intermittent prednisolone - Duchenne Muscular Dystrophy | Not applicable | Not yet recruiting | Muhammad Aamir Latif | 40 | 2026-09-15 | 2026-08-26 |
| Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients VM100 Nutritional Supplement - Duchenne Muscular Dystrophy, Duchenne Disease | Phase 2 | Recruiting | University of Florida | 20 | 2026-07-27 | 2026-10-08 |
| Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy WVE-N531 - Duchenne Muscular Dystrophy | Phase 2 | Enrolling by invitation | Wave Life Sciences USA, Inc. | 175 | 2025-12-28 | 2026-03-06 |
| Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients SAT-3247, Placebo - Duchenne Muscular Dystrophy, Duchenne | Phase 2 | Active not recruiting | Satellos Bioscience, Inc. | 51 | 2025-12-08 | 2026-10-02 |
| 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 |
| A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy GNR-097, Placebo followed by GNR-097 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Recruiting | AO GENERIUM | 32 | 2025-09-30 | 2026-06-29 |
| A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) INS1201 - Duchenne Muscular Dystrophy | Phase 1 | Active not recruiting | Insmed Gene Therapy LLC | 12 | 2025-07-22 | 2026-09-16 |
| Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy Gamified occupational therapy programme, Classic occupational therapy programme - Duchenne Muscular Dystrophy, Gamification | Not applicable | Recruiting | Başak Çağla Arslan | 20 | 2025-05-01 | 2026-01-27 |
| A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) Satralizumab - Duchenne Muscular Dystrophy | Phase 2 | Active not recruiting | Hoffmann-La Roche | 30 | 2025-04-04 | 2026-09-04 |
| Trial of Cell Based Therapy for DMD MyoPAXon, Tacrolimus - Duchenne Muscular Dystrophy | Phase 1 | Recruiting | Masonic Cancer Center, University of Minnesota | 8 | 2025-03-20 | 2026-03-04 |
| Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Duchenne Muscular Dystrophy Evaluations - Executive Dysfunction, Occupational Problems | Enrolling by invitation | Lokman Hekim University | 38 | 2025-03-01 | 2026-02-23 | |
| Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies. Digital respiratory intervention program - Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Not applicable | Enrolling by invitation | University of Malaga | 12 | 2025-02-05 | 2025-04-01 |
| Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old Givinostat Hydrochloride, Givinostat Hydrochloride - Cohort 2 - Duchenne Muscular Dystrophy | Phase 2 | Recruiting | Italfarmaco | 18 | 2025-01-02 | 2025-07-10 |
| Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy Duchenne Muscular Dystrophy | Not yet recruiting | Assiut University | 42 | 2024-12-30 | 2024-12-13 | |
| The U.K. NorthStar Clinical Network Duchenne Muscular Dystrophy | Not yet recruiting | University College, London | 300 | 2024-12-01 | 2024-12-02 | |
| A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy vamorolone 40 mg/mL oral suspension - Duchenne Muscular Dystrophy | Phase 4 | Active not recruiting | Santhera Pharmaceuticals | 80 | 2024-11-10 | 2026-04-23 |
| Development of a Registry to Assess Natural History in Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy | Not yet recruiting | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 200 | 2024-11-01 | 2024-08-30 | |
| Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) Cardiac MRI, Blood assays - Duchenne Muscular Dystrophy | Not applicable | Withdrawn | Assistance Publique - Hôpitaux de Paris | 2024-11-01 | 2025-11-20 |
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Muscular Dystrophy, Duchenne (299) · Muscular Dystrophies (41) · Neuromuscular Diseases (11) · Cardiomyopathies (10) · Muscular Atrophy, Spinal (10) · Motor Activity (7) · Muscular Diseases (6) · Muscular Dystrophies, Limb-Girdle (5) · Myotonic Dystrophy (4) · Amyotrophic Lateral Sclerosis (3) · Anemia, Sickle Cell (3) · Cystic Fibrosis (3) · Gait Disorders, Neurologic (3) · Glycogen Storage Disease Type II (3) · Heart Failure (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (299) · Muscular Disorders, Atrophic (298) · Genetic Diseases, Inborn (297) · Genetic Diseases, X-Linked (297) · Nervous System Diseases (297) · Musculoskeletal Diseases (296) · Muscular Diseases (293) · Neuromuscular Diseases (288) · Muscular Dystrophies (258) · Central Nervous System Diseases (18) · Cardiovascular Diseases (16) · Neurodegenerative Diseases (16) · Spinal Cord Diseases (16) · Pathological Conditions, Signs and Symptoms (14) · Heart Diseases (13)
ataluren (10) · VBP15 compound (8) · eteplirsen (7) · Prednisone (6) · deflazacort (6) · casimersen (5) · golodirsen (5) · Tadalafil (4) · viltolarsen (4) · Adrenal Cortex Hormones (3) · Sildenafil Citrate (3) · Sodium Chloride (3) · TAS-205 (3) · Wearable Electronic Devices (3) · givinostat (3)
Sarepta Therapeutics, Inc. (19) · Hacettepe University (18) · PTC Therapeutics (14) · BioMarin Pharmaceutical (8) · Cooperative International Neuromuscular Research Group (8) · NS Pharma, Inc. (8) · University of Florida (7) · Pfizer (6) · Santhera Pharmaceuticals (6) · Assistance Publique - Hôpitaux de Paris (5) · Lokman Hekim University (5) · Italfarmaco (4) · Nationwide Children's Hospital (4) · ReveraGen BioPharma, Inc. (4) · Solid Biosciences Inc. (4)
United States (153) · United Kingdom (59) · Italy (49) · Canada (47) · Turkey (Türkiye) (45) · France (40) · Belgium (36) · Spain (33) · Australia (25) · Germany (25) · Netherlands (25) · Japan (20) · Sweden (20) · Israel (18) · South Korea (12)
Francesco Bibbiani, MD (4) · Paula R Clemens, MD (4) · Carlos BM Monteiro, Ph.D. (3) · Diana M Escolar, MD (3) · Kevin Flanigan, MD (3) · Mark Awadalla (3) · Brenda Wong, MD (2) · Carmen Castrillo, MD (2)
PMID 32956407, PMID 42613652, PMID 32434278, PMID 32522498, PMID 37539981, PMID 38521535, PMID 41774261, PMID 42139656, PMID 42202243, PMID 17136445, PMID 17385269, PMID 17389552, PMID 17450125, PMID 19713152, PMID 20517938, PMID 20558672, PMID 20587736, PMID 20925545, PMID 21179598, PMID 21784508, PMID 22068425, PMID 22453902, PMID 23907995, PMID 23913485, PMID 24349052, PMID 25042693, PMID 25554404, PMID 25604253, PMID 25635234, PMID 26573217, PMID 26799743, PMID 28219442, PMID 29125504, PMID 29174484, PMID 29907980, PMID 30443431, PMID 30706490, PMID 30761066, PMID 31023296, PMID 31046703, PMID 31315600, PMID 31549577, PMID 31573675, PMID 31752977, PMID 31792454, PMID 31852847, PMID 31876124, PMID 31957953, PMID 32417793, PMID 32453377
2023 (6), 2019 (5), 2015 (4), 2016 (3), 2025 (2), R44NS095423 (2), U34AR068616 (2), 2009 (1), 2014 (1), 2017 (1), R01AR056973 (1), R01NS043186 (1), R21AR064949 (1), R21AR079755 (1), R44NS073188 (1)