55 completed, 11 unknown, 9 terminated, 8 recruiting. 93 Phase 1, 45 Phase 2.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants GRT6019 - Duchenne Muscular Dystrophy (DMD) | Phase 1 | Completed | Grünenthal GmbH | 24 | 2026-01-06 | 2026-08-12 |
| A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants GRT6019 - Duchenne Muscular Dystrophy (DMD) | Phase 1 | Completed | Grünenthal GmbH | 18 | 2025-11-21 | 2026-02-13 |
| 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 |
| A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) INS1201 - Duchenne Muscular Dystrophy | Phase 1 | Active not recruiting | Insmed Gene Therapy LLC | 12 | 2025-07-22 | 2026-10-28 |
| 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 |
| Trial of Cell Based Therapy for DMD MyoPAXon, Tacrolimus - Duchenne Muscular Dystrophy | Phase 1 | Recruiting | Masonic Cancer Center, University of Minnesota | 8 | 2025-03-20 | 2026-03-04 |
| 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 |
| A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 delandistrogene moxeparvovec, Plasmapheresis - Duchenne Muscular Dystrophy | Phase 1 | Terminated | Sarepta Therapeutics, Inc. | 3 | 2024-09-18 | 2025-09-04 |
| 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 |
| Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy EN001 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Unknown | ENCell | 88 | 2024-03-01 | 2024-03-25 |
| A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD) JWK007 Single intravenous infusion administration - Duchenne Muscular Dystrophy | Phase 1 | Active not recruiting | West China Hospital | 3 | 2024-01-31 | 2026-09-10 |
| A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) delandistrogene moxeparvovec, imlifidase - Duchenne Muscular Dystrophy | Phase 1 | Terminated | Sarepta Therapeutics, Inc. | 5 | 2024-01-29 | 2026-11-06 |
| A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy BMN 351 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 18 | 2024-01-03 | 2026-06-25 |
| Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy Phase 1, SQY51, Phase 2a, SQY51 (cohort 1), Phase 2a, SQY51 (cohort 2) - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Unknown | Sqy Therapeutics | 12 | 2023-04-26 | 2024-04-01 |
| Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Duchenne Muscular Dystrophy, Exon 44 | Phase 1 / Phase 2 | Completed | Avidity Biosciences, Inc. | 70 | 2022-11-09 | 2025-07-14 |
| Treatment of a Single Patient With CRD-TMH-001 CRD-TMH-001 - Duchenne Muscular Dystrophy | Phase 1 | Completed | Cure Rare Disease, Inc | 1 | 2022-08-31 | 2026-10-20 |
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Muscular Dystrophies (17) · Muscular Diseases (4) · Neuromuscular Diseases (3) · Cardiomyopathies (2) · Muscular Dystrophies, Limb-Girdle (2) · Musculoskeletal Diseases (2) · Nervous System Diseases (2) · Distal Myopathies (1) · Genetic Diseases, X-Linked (1) · Glycogen Storage Disease Type V (1) · Heart Failure (1) · Muscular Dystrophy, Facioscapulohumeral (1) · Myositis, Inclusion Body (1) · Myotonic Dystrophy (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (93) · Genetic Diseases, Inborn (93) · Muscular Disorders, Atrophic (93) · Genetic Diseases, X-Linked (92) · Musculoskeletal Diseases (91) · Nervous System Diseases (91) · Neuromuscular Diseases (90) · Muscular Diseases (89) · Muscular Dystrophies (76) · Cardiovascular Diseases (2) · Heart Diseases (2) · Nutritional and Metabolic Diseases (2) · Pathological Conditions, Signs and Symptoms (2) · Body Weight (1) · Carbohydrate Metabolism, Inborn Errors (1)
Catechin (3) · Sodium Chloride (3) · Arginine (2) · Injections (2) · Pentoxifylline (2) · SMT C1100 (2) · deflazacort (2) · eteplirsen (2) · givinostat (2) · givinostat hydrochloride (2) · sodium nitrate (2) · Clinical Trials, Phase I as Topic (1) · Dietary Supplements (1) · Drug Evaluation (1) · Gentamicins (1)
Sarepta Therapeutics, Inc. (10) · BioMarin Pharmaceutical (4) · Cedars-Sinai Medical Center (3) · Kevin Flanigan (3) · Nationwide Children's Hospital (3) · Chaitanya Hospital, Pune (2) · Cooperative International Neuromuscular Research Group (2) · Craig McDonald, MD (2) · ENCell (2) · Edgewise Therapeutics, Inc. (2) · Entrada Therapeutics, Inc. (2) · Grünenthal GmbH (2) · Italfarmaco (2) · Jerry R. Mendell (2) · National Center of Neurology and Psychiatry, Japan (2)
United States (55) · Italy (15) · United Kingdom (15) · Canada (10) · France (8) · Belgium (7) · Netherlands (6) · Spain (6) · Japan (5) · South Korea (5) · Turkey (Türkiye) (4) · India (3) · Australia (2) · China (2) · Jordan (2)
Kevin Flanigan, MD (3) · Diana Escolar, MD (2) · Hirofumi Komaki, MD, PhD (2) · Jerry R Mendell, MD (2) · Megan Waldrop, MD (2) · Ronald G Victor, MD (2) · A. Ferlini, PhD (1) · Austen Eddy, MSM (1) · Brenda Wong, MD (1)
PMID 37539981, PMID 38753764, PMID 40152935, PMID 19713152, PMID 20517938, PMID 20925545, PMID 21179598, PMID 21784508, PMID 22068425, PMID 25322757, PMID 26799743, PMID 27055247, PMID 27566866, PMID 32539076, PMID 34304968, PMID 34788571, PMID 37326950, PMID 38229112, PMID 39747998, PMID 39793573, PMID 40452637, PMID 40579547, PMID 40583273, PMID 40583275, PMID 40753271, PMID 42874619
2023 (2), 2015 (1), R01NS043186 (1), R61HL180327 (1), U54AR056953 (1)