4 completed, 2 active not recruiting, 2 recruiting, 2 unknown. 11 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 11 | Completed 4, Active not recruiting 2, Recruiting 2, Unknown 2, Terminated 1 |
Follow Muscular Dystrophies: new trials by email Literature Review of Muscular Dystrophies trials Research Report on Muscular Dystrophies
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 |
|---|---|---|---|---|---|---|
| Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting ELEVIDYS, Sirolimus, Glucocorticoids - Duchenne Muscular Dystrophy | Phase 4 | Recruiting | Sarepta Therapeutics, Inc. | 20 | 2026-10-30 | 2026-10-06 |
| A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy vamorolone 40 mg/mL oral suspension - Duchenne Muscular Dystrophy | Phase 4 | Active not recruiting | Santhera Pharmaceuticals | 80 | 2024-11-10 | 2026-04-23 |
| Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) Viltolarsen - Duchenne Muscular Dystrophy | Phase 4 | Active not recruiting | NS Pharma, Inc. | 9 | 2021-06-10 | 2022-10-19 |
| Once Weekly Infant Corticosteroid Trial for DMD Prednisolone - Duchenne Muscular Dystrophy | Phase 4 | Recruiting | Anne M. Connolly | 26 | 2021-04-30 | 2026-08-14 |
| An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53] - Duchenne Muscular Dystrophy | Phase 4 | Terminated | Rare Disease Research, LLC | 2 | 2020-10-31 | 2026-04-03 |
| Stacking Exercises Aid the Decline in FVC and Sick Time Lung Volume Recruitment (LVR), Conventional Treatment - Duchenne Muscular Dystrophy | Phase 4 | Completed | Children's Hospital of Eastern Ontario | 70 | 2013-03-01 | 2025-01-09 |
| Flu Vaccine Study in Neuromuscular Patients 2011 2011-2012 seasonal flu vaccine Subcutaneous, 2011-2012 seasonal flu vaccine Intramuscular - Duchenne Muscular Dystrophy, Spinal Muscular Atrophy | Phase 4 | Completed | Children's Hospital Medical Center, Cincinnati | 22 | 2011-08-01 | 2020-10-19 |
| Tadalafil in Becker Muscular Dystrophy Tadalafil, Placebo - Becker Muscular Dystrophy | Phase 4 | Completed | Cedars-Sinai Medical Center | 48 | 2010-01-01 | 2013-08-20 |
| Ramipril Versus Carvedilol in Duchenne and Becker Patients carvedilol, ramipril - Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 4 | Unknown | Catholic University, Italy | 194 | 2008-12-01 | 2016-01-28 |
| The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy Carvedilol - Duchenne Muscular Dystrophy, Cardiomyopathies | Phase 4 | Unknown | Suzuka Hospital | 60 | 2007-12-01 | 2008-02-05 |
| Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy Bisphosphonate treatment - Osteoporosis, Muscular Dystrophy | Phase 4 | Completed | Gilles Boire | 11 | 2001-05-01 | 2018-01-09 |
Muscular Dystrophy, Duchenne (10) · Cardiomyopathies (1) · Cystic Fibrosis (1) · Influenza, Human (1) · Muscular Atrophy, Spinal (1) · Osteoporosis (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Genetic Diseases, Inborn (11) · Muscular Diseases (11) · Muscular Disorders, Atrophic (11) · Musculoskeletal Diseases (11) · Nervous System Diseases (11) · Neuromuscular Diseases (11) · Genetic Diseases, X-Linked (10) · Respiratory Tract Diseases (2) · Bone Diseases (1) · Bone Diseases, Metabolic (1) · Cardiovascular Diseases (1) · Central Nervous System Diseases (1) · Digestive System Diseases (1)
Carvedilol (2) · Adrenal Cortex Hormones (1) · Adrenergic beta-Antagonists (1) · Alendronate (1) · Angiotensin-Converting Enzyme Inhibitors (1) · Anti-Bacterial Agents (1) · Glucocorticoids (1) · Influenza Vaccines (1) · Prednisolone (1) · Ramipril (1) · Risedronic Acid (1) · Sirolimus (1) · Suspensions (1) · Tadalafil (1) · VBP15 compound (1)
Anne M. Connolly (1) · Catholic University, Italy (1) · Cedars-Sinai Medical Center (1) · Children's Hospital Medical Center, Cincinnati (1) · Children's Hospital of Eastern Ontario (1) · Gilles Boire (1) · NS Pharma, Inc. (1) · Rare Disease Research, LLC (1) · Santhera Pharmaceuticals (1) · Sarepta Therapeutics, Inc. (1) · Suzuka Hospital (1)
United States (5) · Canada (3) · Belgium (1) · Czechia (1) · Greece (1) · Ireland (1) · Israel (1) · Italy (1) · Japan (1) · Netherlands (1) · New Zealand (1) · Spain (1) · United Kingdom (1)
Anne Connolly, MD (1) · Brenda Wong, MD (1) · Gilles Boire, MD, MSc (1) · Ian MacLusky, MD (1) · Nicholas Barrowman, PhD (1) · Ronald G Victor, MD (1) · Sherri Katz, MD (1) · Takao Nishizawa, MD, PhD (1) · Vincenzo Giglio, MD, PhD (1)
PMID 33887060, PMID 35236763, PMID 36625789, PMID 41147267, PMID 41671094