15 terminated. 15 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 15 | Terminated 15 |
| Phase 2 | 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 to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. fordadistrogene movaparvovec - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Pfizer | 7 | 2023-03-13 | 2025-10-21 |
| Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD Pamrevlumab, Placebo, Corticosteroids - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Kyntra Bio | 73 | 2021-03-03 | 2024-08-26 |
| Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) Pamrevlumab, Placebo, Corticosteroids - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Kyntra Bio | 98 | 2020-08-10 | 2024-03-12 |
| 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 |
| An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy Edasalonexent - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Catabasis Pharmaceuticals | 130 | 2019-03-14 | 2020-11-23 |
| An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy Casimersen, Golodirsen - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Sarepta Therapeutics, Inc. | 171 | 2018-08-02 | 2024-09-19 |
| Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) idebenone 150 mg film-coated tablets - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Santhera Pharmaceuticals | 161 | 2018-07-04 | 2021-12-03 |
| A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids Idebenone 150 mg film-coated tablets, placebo - Duchenne Muscular Dystrophy (DMD) | Phase 3 | Terminated | Santhera Pharmaceuticals | 255 | 2016-09-01 | 2021-12-03 |
| An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy Ataluren - Muscular Dystrophy, Duchenne, Muscular Dystrophies | Phase 3 | Terminated | PTC Therapeutics | 219 | 2014-03-20 | 2020-08-11 |
| A Study of Tadalafil for Duchenne Muscular Dystrophy Tadalafil, Placebo - Muscular Dystrophy, Duchenne | Phase 3 | Terminated | Eli Lilly and Company | 331 | 2013-09-01 | 2019-10-09 |
| A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects Drisapersen - Muscular Dystrophies | Phase 3 | Terminated | BioMarin Pharmaceutical | 53 | 2013-05-01 | 2018-06-14 |
| Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy GSK2402968 - Muscular Dystrophies | Phase 3 | Terminated | GlaxoSmithKline | 233 | 2011-09-01 | 2017-03-23 |
| Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) Ataluren - Duchenne Muscular Dystrophy | Phase 3 | Terminated | PTC Therapeutics | 265 | 2010-11-22 | 2026-10-08 |
| Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders mechanical insufflation - exsufflation, Standard respiratory physiotherapy - Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis | Phase 3 | Terminated | Assistance Publique - Hôpitaux de Paris | 14 | 2009-06-01 | 2015-05-28 |
| CoQ10 and Prednisone in Non-Ambulatory DMD Prednisone, Coenzyme Q10 - Duchenne Muscular Dystrophy | Phase 3 | Terminated | Cooperative International Neuromuscular Research Group | 3 | 2007-04-01 | 2013-11-08 |
Muscular Dystrophies (6) · Neuromuscular Diseases (4) · Musculoskeletal Diseases (3) · Genetic Diseases, Inborn (2) · Genetic Diseases, X-Linked (2) · Muscular Diseases (2) · Muscular Disorders, Atrophic (2) · Nervous System Diseases (2) · Amyotrophic Lateral Sclerosis (1) · Myotonia Congenita (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Genetic Diseases, Inborn (13) · Genetic Diseases, X-Linked (13) · Muscular Diseases (13) · Muscular Disorders, Atrophic (13) · Nervous System Diseases (13) · Musculoskeletal Diseases (12) · Neuromuscular Diseases (11) · Muscular Dystrophies (9) · Central Nervous System Diseases (1) · Heredodegenerative Disorders, Nervous System (1) · Metabolic Diseases (1) · Motor Neuron Disease (1) · Myotonic Disorders (1) · Neurodegenerative Diseases (1)
Adrenal Cortex Hormones (2) · ataluren (2) · idebenone (2) · pamrevlumab (2) · PRO051 (1) · Prednisone (1) · Tadalafil (1) · casimersen (1) · coenzyme Q10 (1) · edasalonexent (1) · golodirsen (1)
Kyntra Bio (2) · PTC Therapeutics (2) · Santhera Pharmaceuticals (2) · Assistance Publique - Hôpitaux de Paris (1) · BioMarin Pharmaceutical (1) · Catabasis Pharmaceuticals (1) · Cooperative International Neuromuscular Research Group (1) · Eli Lilly and Company (1) · GlaxoSmithKline (1) · Pfizer (1) · Sarepta Therapeutics, Inc. (1) · Wave Life Sciences USA, Inc. (1)
United States (13) · Canada (10) · France (10) · United Kingdom (10) · Belgium (9) · Italy (9) · Spain (8) · Germany (7) · Australia (5) · Czechia (5) · Israel (5) · Netherlands (5) · Sweden (5) · Austria (4) · Bulgaria (4)
Francesco Bibbiani, M.D. (1) · Joanne M Donovan, MD, PhD (1) · Michael A Panzara, MD, MPH (1) · Paula R Clemens, M.D. (1) · Vinay Penematsa, MD (1)
PMID 17389552, PMID 17450125, PMID 28972192, PMID 30135256, PMID 40217158
2017 (1)