66 completed, 26 terminated, 17 active not recruiting, 11 unknown. 139 Phase 2, 45 Phase 1, 10 Phase 3.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 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 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 ENTR-601-45, ENTR-601-45 - matching placebo - Duchenne Muscular Dystrophy (DMD) | Phase 1 / Phase 2 | Recruiting | Entrada Therapeutics, Inc. | 24 | 2025-08-30 | 2026-08-13 |
| An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101 SAT-3247 - Duchenne Muscular Dystrophy (DMD) | Phase 2 | Enrolling by invitation | Satellos Bioscience, Inc. | 30 | 2025-08-20 | 2026-10-13 |
| A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 ENTR-601-44, ENTR-601-44 - matching placebo - Duchenne Muscular Dystrophy (DMD) | Phase 1 / Phase 2 | Recruiting | Entrada Therapeutics, Inc. | 24 | 2025-06-30 | 2026-08-13 |
| 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 |
| 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 |
| A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment IV infusion, Placebo - Duchenne Muscular Dystrophy (DMD) | Phase 2 | Withdrawn | PepGen Inc | 2024-12-17 | 2025-06-24 | |
| NS-050/NCNP-03 in Boys With DMD (Meteor50) NS-050/NCNP-03, Placebo - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Active not recruiting | NS Pharma, Inc. | 20 | 2024-09-18 | 2026-06-16 |
| Study of UC-MSC in DMD Patients Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) - Duchenne Muscular Dystrophy | Phase 2 | Active not recruiting | MED Institute Inc. | 35 | 2024-08-05 | 2026-08-14 |
| 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 |
| A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) SGT-003 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Recruiting | Solid Biosciences Inc. | 60 | 2024-05-06 | 2026-07-08 |
| Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3 - Duchenne Muscular Dystrophy | Phase 2 | Active not recruiting | Edgewise Therapeutics, Inc. | 43 | 2024-03-22 | 2025-11-06 |
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Muscular Dystrophies (23) · Neuromuscular Diseases (7) · Cardiomyopathies (4) · Muscular Diseases (3) · Muscular Dystrophies, Limb-Girdle (3) · Motor Activity (2) · Nervous System Diseases (2) · Tabes Dorsalis (2) · Aneurysm (1) · Cardiomyopathy, Dilated (1) · Fatigue (1) · Fractures, Bone (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (139) · Genetic Diseases, Inborn (138) · Genetic Diseases, X-Linked (138) · Muscular Disorders, Atrophic (138) · Musculoskeletal Diseases (138) · Nervous System Diseases (137) · Muscular Diseases (136) · Neuromuscular Diseases (132) · Muscular Dystrophies (116) · Cardiovascular Diseases (5) · Heart Diseases (4) · Pathological Conditions, Signs and Symptoms (4) · Bacterial Infections (2) · Bacterial Infections and Mycoses (2) · Behavior (2)
eteplirsen (9) · ataluren (8) · VBP15 compound (6) · Prednisone (4) · Catechin (3) · Sodium Chloride (3) · givinostat (3) · givinostat hydrochloride (3) · Glutamine (2) · Injections (2) · Pentoxifylline (2) · Prednisolone (2) · Sildenafil Citrate (2) · Tadalafil (2) · coenzyme Q10 (2)
Sarepta Therapeutics, Inc. (12) · PTC Therapeutics (8) · BioMarin Pharmaceutical (7) · Cooperative International Neuromuscular Research Group (6) · NS Pharma, Inc. (5) · ReveraGen BioPharma, Inc. (5) · Capricor Inc. (4) · Edgewise Therapeutics, Inc. (4) · Italfarmaco (4) · University of Florida (4) · Craig McDonald, MD (3) · Entrada Therapeutics, Inc. (3) · Hoffmann-La Roche (3) · Kevin Flanigan (3) · Pfizer (3)
United States (89) · United Kingdom (37) · Italy (34) · Canada (28) · Belgium (26) · Spain (20) · Australia (18) · Netherlands (18) · France (16) · Israel (12) · Japan (12) · Sweden (12) · Germany (10) · Turkey (Türkiye) (9) · Denmark (4)
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