7 recruiting, 3 active not recruiting, 2 unknown, 1 approved for marketing. 6 Phase 2, 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Active not recruiting 3, Recruiting 3 |
| Phase 3 | 2 | Recruiting 2 |
| Phase 1 | 1 | Recruiting 1 |
Follow DMD: new trials by email Literature Review of DMD trials Research Report on 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy. Single dose intravenous of BBM-D101 - DMD | Phase 1 / Phase 2 | Recruiting | Belief BioMed (Beijing) Co., Ltd | 9 | 2025-07-31 | 2025-12-04 |
| ML-Based Multi-Sensor Fall Risk Screening in DMD Performance of the Upper Limb, Brooke Scale, grip strength test - DMD | Recruiting | Seoul National University Hospital | 30 | 2024-07-18 | 2025-06-13 | |
| Vasodilator and Exercise Study for DMD (VASO-REx) Tadalafil, Placebo, Exercise Training - Duchenne Muscular Dystrophy, Duchenne Disease | Phase 2 | Recruiting | University of Florida | 50 | 2024-06-05 | 2026-05-15 |
| NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) NS-089/NCNP-02 - Duchenne Muscular Dystrophy, Exon 44 | Phase 2 | Active not recruiting | NS Pharma, Inc. | 20 | 2024-02-22 | 2026-09-15 |
| Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping AOC 1044 - DMD, Duchenne Muscular Dystrophy | Phase 2 | Active not recruiting | Avidity Biosciences, Inc. | 39 | 2024-01-22 | 2026-07-30 |
| 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 | |
| Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives Specimen sample - Age-Related Macular Degeneration, Allergies | Unknown | Sanguine Biosciences | 20000 | 2021-10-26 | 2024-05-07 | |
| Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children Xlsma, DMD | Withdrawn | Children's Hospital of Fudan University | 2021-09-30 | 2021-08-11 | ||
| Swiss Registry for Neuromuscular Disorders SMA, DMD | Recruiting | University of Bern | 2000 | 2018-06-20 | 2026-01-15 | |
| Constipation and Gut Transit in DMD Patients SmartPill ingestion - Constipation, DMD | Completed | Children's Hospital Medical Center, Cincinnati | 8 | 2013-05-01 | 2020-09-16 | |
| The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation viltolarsen - Muscular Dystrophy, Duchenne, DMD | Approved for marketing | NS Pharma, Inc. | 2020-08-18 |
Muscular Dystrophy, Duchenne (8) · Muscular Dystrophies (6) · Neuromuscular Diseases (4) · Genetic Diseases, Inborn (3) · Genetic Diseases, X-Linked (2) · Muscular Diseases (2) · Muscular Disorders, Atrophic (2) · Musculoskeletal Diseases (2) · Myotonic Dystrophy (2) · Nervous System Diseases (2) · Tabes Dorsalis (2) · Adrenoleukodystrophy (1) · Alopecia Areata (1) · Alzheimer Disease (1) · Amyloidosis (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Nervous System Diseases (8) · Genetic Diseases, Inborn (7) · Genetic Diseases, X-Linked (7) · Muscular Diseases (7) · Musculoskeletal Diseases (7) · Muscular Disorders, Atrophic (6) · Neuromuscular Diseases (5) · Central Nervous System Diseases (4) · Bacterial Infections (3) · Bacterial Infections and Mycoses (3) · Heredodegenerative Disorders, Nervous System (3) · Infections (3) · Neurodegenerative Diseases (3) · Pathological Conditions, Signs and Symptoms (3)
Exercise (1) · Tadalafil (1) · viltolarsen (1)
Avidity Biosciences, Inc. (2) · NS Pharma, Inc. (2) · University of Florida (2) · Belief BioMed (Beijing) Co., Ltd (1) · Children's Hospital Medical Center, Cincinnati (1) · Children's Hospital of Fudan University (1) · Dyne Therapeutics (1) · Sanguine Biosciences (1) · Satellos Bioscience, Inc. (1) · Seoul National University Hospital (1) · University College, London (1) · University of Bern (1)
United States (8) · Australia (3) · United Kingdom (3) · Belgium (2) · Canada (2) · China (2) · South Korea (2) · Spain (2) · France (1) · Germany (1) · Italy (1) · Japan (1) · Netherlands (1) · New Zealand (1) · Poland (1)
Ajay Kaul, MD (1) · Carmen Castrillo, MD (1) · Claudia E Kuehni, Prof. MD (1) · Francesco Muntoni, Professor (1) · Houman Hemmati, MD (1) · Tanja Taivassalo, MD (1) · Tanja Taivassalo, Ph.D. (1) · Yi Dai, MD (1)
2025 (2), 2018 (1), R21AR079755 (1)