6 completed, 3 recruiting, 2 active not recruiting, 1 terminated. 2 Phase 1, 2 Phase 2, 2 Phase 3, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 2 |
| Phase 2 | 2 | Completed 2 |
| Phase 3 | 2 | Active not recruiting 1, Terminated 1 |
| Not applicable | 1 | Recruiting 1 |
Follow Limb-Girdle Muscular Dystrophy: new trials by email Literature Review of Limb-Girdle Muscular Dystrophy trials Research Report on Limb-Girdle Muscular Dystrophy
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 |
|---|---|---|---|---|---|---|
| Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular Dystrophy Neuro-psychosocial teleassistance program - Limb-Girdle Muscular Dystrophy, Social Competence | Not applicable | Recruiting | University of Deusto | 30 | 2026-03-01 | 2026-07-17 |
| The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy Muscle ultrasound - Limb-girdle Muscular Dystrophy | Unknown | Assiut University | 50 | 2024-06-01 | 2024-05-06 | |
| A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 SRP-9003, Glucocorticoid - Limb-girdle Muscular Dystrophy | Phase 3 | Active not recruiting | Sarepta Therapeutics, Inc. | 17 | 2024-01-15 | 2026-03-02 |
| A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice Limb-girdle Muscular Dystrophy | Active not recruiting | Sarepta Therapeutics, Inc. | 205 | 2021-04-22 | 2025-11-06 | |
| A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) Deflazacort - Limb-Girdle Muscular Dystrophy | Phase 3 | Terminated | PTC Therapeutics | 11 | 2019-10-31 | 2022-06-27 |
| Weekly Steroids in Muscular Dystrophy Prednisone - Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | Completed | Northwestern University | 20 | 2019-07-01 | 2023-09-21 |
| MRI on Persons With Mutations in POMT2 Gene (LGMD2N) Limb-girdle Muscular Dystrophy | Completed | Copenhagen University Hospital, Rigshospitalet | 12 | 2016-04-01 | 2017-04-11 | |
| MRI and Muscle Involvement in Patients With Mutations in GMPPB Limb-girdle Muscular Dystrophy | Completed | Copenhagen University Hospital, Rigshospitalet | 4 | 2015-11-01 | 2016-04-06 | |
| Congenital Muscle Disease Study of Patient and Family Reported Medical Information Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) | Recruiting | Cure CMD | 4000 | 2009-09-01 | 2021-08-09 | |
| Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy Retrograde high pressure transvenous perfusion with normal saline - Muscular Dystrophies, Duchenne Muscular Dystrophy | Phase 1 | Completed | University of North Carolina, Chapel Hill | 16 | 2009-03-01 | 2015-03-09 |
| Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy Limb-Girdle Muscular Dystrophy | Completed | Nationwide Children's Hospital | 277 | 2005-06-01 | 2018-01-25 | |
| Study Evaluating MYO-029 in Adult Muscular Dystrophy MYO-029 - Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy | Phase 1 / Phase 2 | Completed | Wyeth is now a wholly owned subsidiary of Pfizer | 108 | 2005-02-01 | 2007-12-28 |
| Molecular Analysis of Patients With Neuromuscular Disease Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy | Recruiting | Boston Children's Hospital | 1000 | 2002-01-01 | 2023-04-24 |
Muscular Dystrophies, Limb-Girdle (13) · Muscular Dystrophy, Duchenne (4) · Muscular Dystrophies (2) · Neuromuscular Diseases (2) · Epilepsy (1) · Intellectual Disability (1) · Muscle Weakness (1) · Muscular Atrophy (1) · Muscular Dystrophy, Congenital, Due To Integrin Alpha-7 Deficiency (1) · Muscular Dystrophy, Emery-Dreifuss (1) · Muscular Dystrophy, Facioscapulohumeral (1) · Muscular Dystrophy, Limb-Girdle, Type 2G (1) · Muscular Dystrophy, Limb-Girdle, Type 2M (1) · Musculoskeletal Diseases (1) · Myasthenic Syndromes, Congenital (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Genetic Diseases, Inborn (13) · Muscular Diseases (13) · Muscular Disorders, Atrophic (13) · Nervous System Diseases (13) · Musculoskeletal Diseases (12) · Muscular Dystrophies (11) · Neuromuscular Diseases (11) · Genetic Diseases, X-Linked (5) · Neurologic Manifestations (2) · Pathological Conditions, Signs and Symptoms (2) · Signs and Symptoms (2) · Atrophy (1) · Behavior (1) · Brain Diseases (1)
Prednisone (2) · Glucocorticoids (1) · Prednisolone (1) · Saline Solution (1) · Stamulumab (1) · deflazacort (1)
Copenhagen University Hospital, Rigshospitalet (2) · Sarepta Therapeutics, Inc. (2) · Assiut University (1) · Boston Children's Hospital (1) · Cure CMD (1) · Nationwide Children's Hospital (1) · Northwestern University (1) · PTC Therapeutics (1) · University of Deusto (1) · University of North Carolina, Chapel Hill (1) · Wyeth is now a wholly owned subsidiary of Pfizer (1)
United States (9) · Denmark (3) · Germany (3) · Spain (3) · Belgium (2) · Canada (2) · Italy (2) · Sweden (2) · United Kingdom (2) · Brazil (1) · France (1) · Norway (1) · Russia (1) · Turkey (Türkiye) (1)
Cristobal Passalacqua, MD (1) · Gustavo Dziewczapolski, PhD (1) · Jerry R. Mendell, M.D. (1) · Louis M Kunkel, PhD (1) · Senda Ajroud-Driss, MD (1) · William J. Powers, MD (1)
PMID 27708273, PMID 28533404, PMID 29175898
R01NS080929 (1), U54AR056953 (1)