50 active trials 24 recruiting 36 sponsors 4 changes in 30 days
| Early Phase 1 | 5 | |
| Phase 1 | 4 | |
| Phase 1/2 | 4 | |
| Phase 2 | 7 | |
| Phase 2/3 | 1 | |
| Phase 3 | 3 |
| NCT ID | Title | Sponsor | Status | Updated |
|---|---|---|---|---|
| Phase 3 | ||||
| NCT07608432 | Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered… | Dyne Therapeutics | Recruiting | 2026-05-27 |
| NCT05881408 | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene… | Sarepta Therapeutics, Inc. | Active, Not Recruiting | 2026-05-22 |
| NCT03992430 | A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With… | Sarepta Therapeutics, Inc. | Active, Not Recruiting | 2026-02-27 |
| Phase 2/3 | ||||
| NCT05693142 | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy… | REGENXBIO Inc. | Active, Not Recruiting | 2026-07-21 |
| Phase 2 | ||||
| NCT05996003 | NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) | NS Pharma, Inc. | Recruiting | 2026-03-06 |
| NCT06450639 | A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy… | Hoffmann-La Roche | Active, Not Recruiting | 2026-08-13 |
| NCT06128564 | A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene… | Hoffmann-La Roche | Active, Not Recruiting | 2026-08-10 |
| NCT07573631 | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in… | BioMarin Pharmaceutical | Enrolling by Invitation | 2026-07-24 |
| NCT07682129 | Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to… | Entrada Therapeutics, Inc. | Not Yet Recruiting | 2026-07-02 |
| NCT06867107 | An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne… | Satellos Bioscience, Inc. | Enrolling by Invitation | 2026-04-23 |
| NCT05135663 | Extension Study of NS-089/NCNP-02 in DMD | Nippon Shinyaku Co., Ltd. | Active, Not Recruiting | 2025-07-20 |
| Phase 1/2 | ||||
| NCT07038824 | A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to… | Entrada Therapeutics, Inc. | Recruiting | 2026-08-13 |
| NCT07037862 | A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to… | Entrada Therapeutics, Inc. | Recruiting | 2026-08-13 |
| NCT05524883 | Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in… | Dyne Therapeutics | Active, Not Recruiting | 2026-08-11 |
| NCT07642635 | Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With… | Vanderbilt University Medical Center | Not Yet Recruiting | 2026-06-11 |
| Phase 1 | ||||
| NCT07172971 | Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy | Vanderbilt University Medical Center | Recruiting | 2026-08-24 |
| NCT06817382 | A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT)… | Insmed Gene Therapy LLC | Recruiting | 2026-07-28 |
| NCT04626674 | A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From… | Sarepta Therapeutics, Inc. | Recruiting | 2026-06-24 |
| NCT06114056 | A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single… | West China Hospital | Active, Not Recruiting | 2026-03-17 |
| Early Phase 1 | ||||
| NCT07188012 | Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients | Shanghai Siponuoyin Biotechnology Co Ltd | Recruiting | 2026-05-07 |
| NCT06900049 | Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne… | Shanghai Jiao Tong University School of Medicine | Recruiting | 2025-04-02 |
| NCT06641895 | Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne… | Shanghai Jiao Tong University School of Medicine | Recruiting | 2025-03-25 |
| NCT07282652 | A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With… | Peking Union Medical College Hospital | Active, Not Recruiting | 2026-01-16 |
| NCT06392724 | A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular… | Peking Union Medical College Hospital | Active, Not Recruiting | 2025-07-29 |
| Not applicable | ||||
| NCT07039799 | The Effect of Virtual Reality Applications on Upper Extremity Functions in Patients With… | Istanbul University | Recruiting | 2026-05-19 |
Entrada Therapeutics, Inc. (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) · Hoffmann-La Roche (2) · Istanbul University (2) · Lokman Hekim University (2) · Shanghai Jiao Tong University School of Medicine (2)