234 completed, 59 recruiting, 53 terminated, 48 unknown. 139 Phase 2, 93 Phase 1, 85 Not applicable, 59 Phase 3, 10 Phase 4.
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 |
|---|---|---|---|---|---|---|
| Urotherapy Education for Families of Children With Duchenne Muscular Dystrophy Urotherapy Education - Duchenne Muscular Dystrophy (DMD), Lower Urinary Tract Symptoms (LUTS) | Not applicable | Not yet recruiting | Lokman Hekim University | 50 | 2026-11-01 | 2026-09-15 |
| 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 |
| Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function Becker Muscular Dystrophy | Not yet recruiting | Institut de Myologie, France | 75 | 2026-09-15 | 2026-07-31 | |
| 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 |
| Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy Semaglutide (Rybelsus®), Placebo - Duchenne Muscular Dystrophy (DMD) | Phase 1 / Phase 2 | Not yet recruiting | Vanderbilt University Medical Center | 30 | 2026-09-01 | 2026-06-11 |
| 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 |
| Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy Multicomponent Intervention - Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA) | Not applicable | Active not recruiting | Aga Khan University | 34 | 2026-08-15 | 2026-10-09 |
| Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE) ENTR-601-44, ENTR-601-45 - Duchenne Muscular Dystrophy (DMD) | Phase 2 | Not yet recruiting | Entrada Therapeutics, Inc. | 80 | 2026-08-01 | 2026-07-02 |
| Digital Monitoring of Upper Limb Function in Non-Ambulant DMD Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength - Duchenne Muscular Dystrophy (DMD) | Not applicable | Not yet recruiting | Centre Hospitalier Universitaire de Liege | 50 | 2026-07-30 | 2026-06-23 |
| 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 |
| Registry for Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy | Recruiting | Dr. Andreas Ziegler | 1500 | 2026-07-13 | 2026-10-08 | |
| Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy SGLT-2 inhibitor, SGLT2 inhibitor - Duchenne Muscular Dystrophy (DMD) | Phase 1 | Recruiting | Vanderbilt University Medical Center | 10 | 2026-07-01 | 2026-08-24 |
| A Remote Study Using Technology to Assess Outcomes in DMD Duchenne Muscular Dystrophy (DMD) | Recruiting | University of Oxford | 60 | 2026-06-24 | 2026-06-10 | |
| The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy (DMD) | Recruiting | University of Rochester | 105 | 2026-06-03 | 2026-08-19 | |
| 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 |
| DMD and Gamified Physiotherapy Gamified Mobile Physiotherapy Exercise Application, Standard Home Exercise Instructions - Duchenne Muscular Dystrophy (DMD) | Not applicable | Not yet recruiting | Başak Çağla Arslan | 46 | 2026-05-08 | 2026-01-27 |
| Usability and User Experience of a Virtual Reality Rehabilitation Game Platform in Individuals With Duchenne Muscular Dystrophy Virtual Reality Gameplay Session - Duchenne Muscular Dystrophy (DMD) | Enrolling by invitation | Istanbul University | 6 | 2026-04-28 | 2026-05-04 | |
| PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD) PBGENE-DMD (IV) - Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD | Phase 1 / Phase 2 | Recruiting | Precision BioSciences, Inc. | 18 | 2026-04-24 | 2026-10-21 |
| A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children No interventions are applied; participants are followed for natural history observation only - Muscular Dystrophy, Becker, Muscular Dystrophy | Not yet recruiting | West China Second University Hospital | 500 | 2026-04-20 | 2026-04-06 |
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Muscular Dystrophies (89) · Neuromuscular Diseases (37) · Muscular Diseases (17) · Muscular Atrophy, Spinal (16) · Cardiomyopathies (14) · Genetic Diseases, Inborn (12) · Nervous System Diseases (12) · Motor Activity (11) · Muscular Dystrophies, Limb-Girdle (11) · Musculoskeletal Diseases (11) · Genetic Diseases, X-Linked (10) · Myotonic Dystrophy (10) · Muscular Disorders, Atrophic (9) · Muscular Dystrophy, Facioscapulohumeral (8)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (478) · Muscular Disorders, Atrophic (470) · Genetic Diseases, X-Linked (469) · Musculoskeletal Diseases (468) · Genetic Diseases, Inborn (467) · Nervous System Diseases (467) · Muscular Diseases (462) · Neuromuscular Diseases (442) · Muscular Dystrophies (390) · Neurodegenerative Diseases (30) · Central Nervous System Diseases (27) · Pathological Conditions, Signs and Symptoms (24) · Cardiovascular Diseases (23) · Spinal Cord Diseases (23) · Nutritional and Metabolic Diseases (21)
ataluren (13) · eteplirsen (12) · Prednisone (9) · VBP15 compound (9) · Tadalafil (7) · deflazacort (6) · givinostat (6) · givinostat hydrochloride (6) · idebenone (6) · casimersen (5) · golodirsen (5) · viltolarsen (5) · Catechin (4) · Metformin (4) · Prednisolone (4)
Sarepta Therapeutics, Inc. (27) · Hacettepe University (20) · PTC Therapeutics (18) · Cooperative International Neuromuscular Research Group (15) · BioMarin Pharmaceutical (10) · NS Pharma, Inc. (9) · Santhera Pharmaceuticals (9) · University of Florida (9) · Lokman Hekim University (8) · Pfizer (8) · Assistance Publique - Hôpitaux de Paris (7) · Cedars-Sinai Medical Center (7) · Italfarmaco (7) · Edgewise Therapeutics, Inc. (6) · GlaxoSmithKline (6)
United States (231) · United Kingdom (84) · Italy (82) · Turkey (Türkiye) (67) · France (66) · Canada (64) · Belgium (56) · Spain (54) · Australia (40) · Germany (40) · Netherlands (39) · Israel (29) · Sweden (27) · Japan (26) · South Korea (23)
Craig McDonald, MD (5) · Paula R Clemens, MD (5) · Ronald G Victor, MD (5) · Diana M Escolar, MD (4) · Dirk Fischer, MD (4) · Francesco Bibbiani, MD (4) · Kevin Flanigan, MD (4) · Mark Awadalla (4) · Carlos BM Monteiro, Ph.D. (3) · Diana Escolar, MD (3)
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