90 completed, 35 terminated, 23 active not recruiting, 18 recruiting. 192 Phase 2, 67 Phase 1, 16 Phase 3.
Follow Muscular Dystrophies: new trials by email Literature Review of Muscular Dystrophies trials Research Report on Muscular Dystrophies
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 Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) forazapadin, Placebo Comparator - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | Not yet recruiting | Satellos Bioscience, Inc. | 51 | 2026-12-15 | 2026-10-13 |
| A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy Tegacorat - Duchenne Muscular Dystrophy (DMD) | Phase 2 | Not yet recruiting | Grünenthal GmbH | 36 | 2026-11-09 | 2026-11-03 |
| 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 | 2026-11-04 |
| 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 Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD SFL-0821 for injection, Placebo - Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 1 / Phase 2 | Recruiting | Soufflé Therapeutics, Inc. | 90 | 2026-09-01 | 2026-10-30 |
| 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 |
| Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD Apitegromab, Placebo - Facioscapulohumeral Muscular Dystrophy, FSHD | Phase 2 | Recruiting | Scholar Rock, Inc. | 60 | 2026-07-30 | 2026-09-02 |
| 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 |
| An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) PGN-EDODM1 - Myotonic Dystrophy 1 | Phase 2 | Recruiting | PepGen Inc | 48 | 2025-12-23 | 2026-03-30 |
| 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 to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 SAR446268 - Myotonic Dystrophy | Phase 1 / Phase 2 | Recruiting | Sanofi | 32 | 2025-07-23 | 2026-09-21 |
| 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 First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy EPI-321 - Facioscapulohumeral Muscular Dystrophy | Phase 1 / Phase 2 | Active not recruiting | Epicrispr Biotechnologies, Inc. | 12 | 2025-05-08 | 2026-08-19 |
| A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I VX-670 - Myotonic Dystrophy Type 1 (DM1) | Phase 2 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 44 | 2025-04-28 | 2026-10-14 |
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Muscular Dystrophy, Duchenne (140) · Myotonic Dystrophy (20) · Muscular Dystrophy, Facioscapulohumeral (18) · Neuromuscular Diseases (15) · Muscular Dystrophies, Limb-Girdle (9) · Musculoskeletal Diseases (8) · Muscular Diseases (7) · Nervous System Diseases (6) · Genetic Diseases, Inborn (5) · Muscular Disorders, Atrophic (5) · Muscular Dystrophy, Oculopharyngeal (5) · Myotonic Disorders (5) · Cardiomyopathies (4) · Heredodegenerative Disorders, Nervous System (4)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (192) · Genetic Diseases, Inborn (187) · Muscular Disorders, Atrophic (187) · Nervous System Diseases (186) · Muscular Diseases (185) · Musculoskeletal Diseases (184) · Neuromuscular Diseases (177) · Genetic Diseases, X-Linked (139) · Neurodegenerative Diseases (19) · Heredodegenerative Disorders, Nervous System (18) · Myotonic Disorders (15) · Cardiovascular Diseases (7) · Heart Diseases (6) · Pathological Conditions, Signs and Symptoms (6)
eteplirsen (9) · ataluren (8) · VBP15 compound (6) · Sodium Chloride (5) · Injections (4) · Prednisone (4) · 6-(5-((cyclopropylamino)carbonyl)-3-fluoro-2-methylphenyl)-N-(2,2-dimethylprpyl)-3-pyridinecarboxamide (3) · Catechin (3) · givinostat (3) · givinostat hydrochloride (3) · tideglusib (3) · Glutamine (2) · Insulin-Like Growth Factor I (2) · Pentoxifylline (2) · Prednisolone (2)
Sarepta Therapeutics, Inc. (16) · BioMarin Pharmaceutical (8) · PTC Therapeutics (8) · Avidity Biosciences, Inc. (6) · Cooperative International Neuromuscular Research Group (6) · Hoffmann-La Roche (5) · NS Pharma, Inc. (5) · ReveraGen BioPharma, Inc. (5) · aTyr Pharma, Inc. (5) · Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA (4) · Capricor Inc. (4) · Edgewise Therapeutics, Inc. (4) · Italfarmaco (4) · PepGen Inc (4) · University of Florida (4)
United States (124) · United Kingdom (50) · Canada (49) · Italy (44) · Spain (30) · Belgium (29) · Australia (28) · France (28) · Netherlands (26) · Germany (17) · Israel (14) · Japan (12) · New Zealand (12) · Sweden (12) · Turkey (Türkiye) (9)
Diana M Escolar, MD (4) · Francesco Bibbiani, MD (3) · Kevin Flanigan, MD (3) · Mark Awadalla (3) · Paula R Clemens, MD (3) · Bernard Brais, MD (2) · Carmen Castrillo, MD (2) · Craig McDonald, MD (2) · Diana Escolar, MD (2) · Hirofumi Komaki, MD, PhD (2)
PMID 32956407, PMID 42613652, PMID 26573217, PMID 32434278, PMID 32522498, PMID 37539981, PMID 38190001, PMID 38521535, PMID 38753764, PMID 41671094, PMID 12122118, PMID 12210323, PMID 1281472, PMID 15293280, PMID 15781190, PMID 1580445, PMID 15984021, PMID 16602102, PMID 16763895, PMID 16763896, PMID 17584774, PMID 1824792, PMID 18401027, PMID 18541798, PMID 18645163, PMID 18678823, PMID 18929906, PMID 19237700, PMID 19421321, PMID 19713152, PMID 20357282, PMID 20393176, PMID 20631546, PMID 20655781, PMID 20837825, PMID 21428760, PMID 21435876, PMID 21784508, PMID 2199596, PMID 22578232, PMID 2295969, PMID 23031366, PMID 23406536, PMID 23507172, PMID 23831596, PMID 23907995, PMID 24349052, PMID 24453079, PMID 25042693, PMID 25209738
2023 (8), 2015 (5), 2019 (5), 2016 (4), 2014 (3), 2017 (3), 2025 (2), R44NS095423 (2), U34AR068616 (2), 2009 (1), R21AR079755 (1), U01AR060911 (1), U54NS048843 (1)