26 terminated. 26 Phase 2, 4 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 26 | Terminated 26 |
| Phase 1 | 4 | Terminated 4 |
| Phase 3 | 1 | 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 Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment PGN-EDO51 - Duchenne Muscular Dystrophy | Phase 2 | Terminated | PepGen Inc | 7 | 2024-01-03 | 2025-12-02 |
| A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy ATL1102 25mg, ATL1102 50mg, Placebo - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Percheron Therapeutics | 48 | 2023-05-18 | 2025-02-03 |
| Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy PF-06939926 - Muscular Dystrophy, Duchenne | Phase 2 | Terminated | Pfizer | 10 | 2022-08-08 | 2025-10-21 |
| Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy WVE-210201 (suvodirsen), Placebo - Duchenne Muscular Dystrophy | Phase 2 / Phase 3 | Terminated | Wave Life Sciences USA, Inc. | 6 | 2019-09-04 | 2021-05-20 |
| Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Vesleteplirsen - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Sarepta Therapeutics, Inc. | 62 | 2019-06-26 | 2025-03-10 |
| A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) Eteplirsen - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Sarepta Therapeutics, Inc. | 15 | 2019-06-26 | 2023-08-18 |
| An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) SRP-5051 - Muscular Dystrophy, Duchenne | Phase 1 / Phase 2 | Terminated | Sarepta Therapeutics, Inc. | 15 | 2018-12-19 | 2024-09-19 |
| Safety and Efficacy of P-188 NF in DMD Patients P-188 NF - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Phrixus Pharmaceuticals, Inc. | 2 | 2018-08-08 | 2023-01-11 |
| A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy MNK-1411, Placebo - Muscular Dystrophy, Duchenne | Phase 2 | Terminated | Mallinckrodt ARD LLC | 44 | 2018-07-27 | 2021-03-16 |
| An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy PF-06252616 - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Pfizer | 59 | 2016-10-13 | 2020-11-23 |
| Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) Ezutromid - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Summit Therapeutics | 43 | 2016-06-01 | 2020-01-02 |
| Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg - Duchenne Muscular Dystrophy | Phase 2 | Terminated | BioMarin Pharmaceutical | 7 | 2016-04-01 | 2018-01-26 |
| Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) Pamrevlumab - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Kyntra Bio | 21 | 2016-01-04 | 2024-08-27 |
| HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02 HT-100 - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Akashi Therapeutics | 10 | 2015-05-01 | 2019-03-12 |
| Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg - Duchenne Muscular Dystrophy | Phase 2 | Terminated | BioMarin Pharmaceutical | 15 | 2014-12-01 | 2017-12-08 |
| A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy PF-06252616, Placebo - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Pfizer | 121 | 2014-11-24 | 2020-12-07 |
| Open Label Extension Study of HT-100 in Patients With DMD HT-100 - Duchenne Muscular Dystrophy | Phase 2 | Terminated | Processa Pharmaceuticals | 17 | 2013-10-01 | 2020-09-03 |
| A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) Regimen Selection Phase Group 2, Regimen Selection Phase Group 3, Treatment Phase Group 4 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 9 | 2013-06-01 | 2017-12-08 |
| Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy HT-100 - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Terminated | Processa Pharmaceuticals | 17 | 2013-05-01 | 2020-09-03 |
| Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week - Duchenne Muscular Dystrophy | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 15 | 2013-01-01 | 2017-12-08 |
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Muscular Dystrophies (4) · Neuromuscular Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (26) · Genetic Diseases, Inborn (26) · Genetic Diseases, X-Linked (26) · Muscular Diseases (26) · Muscular Disorders, Atrophic (26) · Musculoskeletal Diseases (26) · Nervous System Diseases (26) · Neuromuscular Diseases (25) · Muscular Dystrophies (22)
ataluren (3) · domagrozumab (2) · Cosyntropin (1) · SMT C1100 (1) · Sildenafil Citrate (1) · eteplirsen (1) · ezutromid (1) · halofuginone (1) · pamrevlumab (1)
BioMarin Pharmaceutical (4) · PTC Therapeutics (3) · Pfizer (3) · Sarepta Therapeutics, Inc. (3) · Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA (2) · Processa Pharmaceuticals (2) · Akashi Therapeutics (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · Kyntra Bio (1) · Mallinckrodt ARD LLC (1) · PepGen Inc (1) · Percheron Therapeutics (1) · Phrixus Pharmaceuticals, Inc. (1) · Summit Therapeutics (1) · Wave Life Sciences USA, Inc. (1)
United States (17) · Italy (11) · United Kingdom (11) · Canada (9) · Belgium (8) · France (5) · Netherlands (5) · Australia (4) · Sweden (4) · Bulgaria (3) · Spain (3) · Germany (2) · Israel (2) · Japan (2) · Serbia (2)
Diana M Escolar, MD (3) · Daniel P Judge, MD (1) · Kathryn R. Wagner, M.D., Ph.D. (1) · Leone Atkinson (1) · Leone Atkinson, M.D., Ph.D. (1) · Leone Atkinson, MD, PhD (1) · Michael A Panzara, MD, MPH (1) · Paulatsya Joshi (1) · T. Voit, MD PhD (1) · Thomas Ryan, MD (1) · Thomas Voit (1)
PMID 32522498, PMID 25042693, PMID 34155911, PMID 34533053, PMID 35204752, PMID 35396602, PMID 35998119, PMID 36104012, PMID 36335191, PMID 39713935
2015 (2), 2019 (2), 2009 (1), 2014 (1), 2016 (1), 2017 (1), 2023 (1)