3 active not recruiting, 3 recruiting, 2 completed, 1 terminated. 9 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Early Phase 1 | 9 | Active not recruiting 3, Recruiting 3, Completed 2, Terminated 1 |
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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy RAG-18 - Duchenne Muscular Dystrophy (DMD) | Early Phase 1 | Active not recruiting | Peking Union Medical College Hospital | 12 | 2025-12-12 | 2026-01-16 |
| Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients SPOT-03 - Duchenne Muscular Dystrophy (DMD) | Early Phase 1 | Recruiting | Shanghai Siponuoyin Biotechnology Co Ltd | 9 | 2025-08-06 | 2026-05-07 |
| An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE) HG302 - Duchenne Muscular Dystrophin (DMD) | Early Phase 1 | Active not recruiting | HuidaGene Therapeutics Co., Ltd. | 4 | 2024-11-06 | 2026-08-03 |
| Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy LE051 - Duchenne Muscular Dystrophy (DMD) | Early Phase 1 | Recruiting | Shanghai Jiao Tong University School of Medicine | 12 | 2024-10-24 | 2025-04-02 |
| First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort SAT-3247, matched placebo - Duchenne Muscular Dystrophy | Early Phase 1 | Completed | Satellos Bioscience, Inc. | 77 | 2024-08-21 | 2025-05-15 |
| Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy BBM-D101 - Duchenne Muscular Dystrophy (DMD) | Early Phase 1 | Recruiting | Shanghai Jiao Tong University School of Medicine | 6 | 2024-07-25 | 2025-03-25 |
| A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. GEN6050X intravenous injection - Duchenne Muscular Dystrophy (DMD) | Early Phase 1 | Active not recruiting | Peking Union Medical College Hospital | 3 | 2024-07-05 | 2025-07-29 |
| The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy Tadalafil 20 MG, beetroot juice extract - Muscular Dystrophy | Early Phase 1 | Terminated | Cedars-Sinai Medical Center | 6 | 2017-11-01 | 2020-01-14 |
| Tadalafil and Sildenafil for Duchenne Muscular Dystrophy Tadalafil, Sildenafil - Duchenne Muscular Dystrophy | Early Phase 1 | Completed | Cedars-Sinai Medical Center | 30 | 2011-05-01 | 2020-01-14 |
Muscular Dystrophies (3) · Motor Activity (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Genetic Diseases, X-Linked (9) · Muscular Diseases (9) · Muscular Disorders, Atrophic (9) · Musculoskeletal Diseases (9) · Nervous System Diseases (9) · Neuromuscular Diseases (9) · Muscular Dystrophies (6) · Behavior (1)
Tadalafil (2) · Sildenafil Citrate (1)
Cedars-Sinai Medical Center (2) · Peking Union Medical College Hospital (2) · Shanghai Jiao Tong University School of Medicine (2) · HuidaGene Therapeutics Co., Ltd. (1) · Satellos Bioscience, Inc. (1) · Shanghai Siponuoyin Biotechnology Co Ltd (1)
China (6) · United States (2) · Australia (1)
Jiwen Wang (1) · Ronald G Victor, MD (1) · Ronald Victor, MD (1) · Wang Jiwen (1)