25 recruiting, 15 completed, 8 not yet recruiting, 6 enrolling by invitation. 18 Not applicable, 15 Phase 2, 10 Phase 1, 5 Early Phase 1, 3 Phase 3.
Follow Duchenne Muscular Dystrophy (DMD): new trials by email Literature Review of Duchenne Muscular Dystrophy (DMD) trials Research Report on Duchenne Muscular Dystrophy (DMD)
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 |
| 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 |
| 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 |
| 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 | |
| An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy BMN 351 - Duchenne Muscular Dystrophy (DMD) | Phase 2 | Enrolling by invitation | BioMarin Pharmaceutical | 18 | 2026-04-20 | 2026-07-24 |
| Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients? Transcutaneous auricular vagus nerve stimulation - Duchenne Muscular Dystrophy (DMD) | Not applicable | Enrolling by invitation | taVNS AB | 20 | 2026-04-13 | 2026-04-24 |
| Invasive Home Ventilation in Denmark Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis) | Recruiting | Copenhagen University Hospital, Rigshospitalet | 450 | 2026-04-13 | 2026-04-20 | |
| A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy Rituxan treatment - Duchenne Muscular Dystrophy (DMD) | Not applicable | Not yet recruiting | Peking Union Medical College Hospital | 5 | 2026-03-23 | 2026-03-16 |
| Urinary Titin Biomarker in DMD Descending stair walk - Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD) | Not applicable | Recruiting | Children's Hospital of Philadelphia | 50 | 2026-03-04 | 2026-04-08 |
| Factors Influencing Physical Activity Levels in Children With Duchenne Muscular Dystrophy: An ICF-CY-Based Study Duchenne Muscular Dystrophy (DMD) | Not yet recruiting | Hacettepe University | 30 | 2026-02-02 | 2026-01-29 | |
| DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy | Recruiting | Aristotle University Of Thessaloniki | 65 | 2026-01-26 | 2026-04-07 | |
| The Effect of Dual-tasking Program on Cognitive and Physical Functions and Independence in Activities of Daily Living in Children With Duchenne Muscular Dystrophy: A Single-blind Randomized Controlled Trial Conventional physiotherapy program, Conventional physiotherapy program + Dual task program - Duchenne Muscular Dystrophy (DMD) | Not applicable | Enrolling by invitation | Lokman Hekim University | 16 | 2026-01-14 | 2026-01-27 |
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Muscular Dystrophy, Duchenne (67) · Muscular Dystrophies (9) · Neuromuscular Diseases (8) · Muscular Atrophy, Spinal (5) · Cardiomyopathies (4) · Motor Activity (3) · Lower Urinary Tract Symptoms (2) · Amyotrophic Lateral Sclerosis (1) · Cerebral Palsy (1) · Chronic Pain (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Heart Failure (1) · Myotonic Dystrophy (1)
Muscular Diseases (67) · Muscular Disorders, Atrophic (67) · Musculoskeletal Diseases (67) · Nervous System Diseases (67) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (66) · Genetic Diseases, Inborn (66) · Genetic Diseases, X-Linked (66) · Neuromuscular Diseases (59) · Muscular Dystrophies (58) · Neurodegenerative Diseases (6) · Cardiovascular Diseases (5) · Central Nervous System Diseases (5) · Motor Neuron Disease (5) · Pathological Conditions, Signs and Symptoms (5) · Spinal Cord Diseases (5)
eteplirsen (3) · Prednisone (2) · idebenone (2) · Cognitive Behavioral Therapy (1) · Drug Evaluation (1) · Exercise (1) · Fitness Trackers (1) · Glucocorticoids (1) · Ibuprofen (1) · Infusions, Intravenous (1) · Isosorbide Dinitrate (1) · Lisinopril (1) · Losartan (1) · Observation (1) · Sodium-Glucose Transporter 2 Inhibitors (1)
Entrada Therapeutics, Inc. (3) · Lokman Hekim University (3) · Peking Union Medical College Hospital (3) · Sarepta Therapeutics, Inc. (3) · Vanderbilt University Medical Center (3) · Centre Hospitalier Universitaire de Liege (2) · Dyne Therapeutics (2) · Grünenthal GmbH (2) · Istanbul University (2) · Istituto Giannina Gaslini (2) · Santhera Pharmaceuticals (2) · Shanghai Jiao Tong University School of Medicine (2) · Aga Khan University (1) · Ankara University (1) · Ann & Robert H Lurie Children's Hospital of Chicago (1)
United States (21) · Italy (11) · Turkey (Türkiye) (10) · Belgium (9) · United Kingdom (7) · China (6) · Spain (6) · Netherlands (4) · France (3) · Australia (2) · Austria (2) · Denmark (2) · Egypt (2) · Germany (2) · Ireland (2)
Andreas Giannopoulos, Professor of Pediatrics and Pediatric Cardiology, MD, PhD (1) · Andreas Ziegler, Dr. (1) · Ann Martin, MS, CGC (1) · Benjamin Kozyak, MD (1) · Eliza Wasilewska, MD,PhD (1) · Emilio Clementi (1) · Enrico Bertini, MD (1) · Eric Camino, PhD (1) · Gunnar Buyse, MD PhD (1) · Jiwen Wang (1) · Larry W. Markham, MD (1)
PMID 11751186, PMID 15302625, PMID 16055882, PMID 17711917, PMID 21435876, PMID 21921156, PMID 22224768, PMID 22463839, PMID 22496194, PMID 22665702, PMID 23447789, PMID 24459612, PMID 25196721, PMID 26573217, PMID 27566866, PMID 28397169, PMID 29395989, PMID 29395990, PMID 34779542, PMID 38018736, PMID 39063545, PMID 41071520
2025 (3), 2023 (2), R01HD112908 (1), R01HL167969 (1), R61HL180327 (1), U01NS134672 (1)