4 completed, 2 enrolling by invitation, 1 recruiting, 1 terminated. 3 Phase 1, 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 3 | Completed 2, Terminated 1 |
| Phase 2 | 2 | Completed 2 |
| Phase 3 | 1 | Completed 1 |
Follow Limb-girdle muscular dystrophy, type 2B: new trials by email Literature Review of Limb-girdle muscular dystrophy, type 2B trials Research Report on Limb-girdle muscular dystrophy, type 2B
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 |
|---|---|---|---|---|---|---|
| Long-Term Development of Muscular Dystrophy Outcome Assessments LGMD1B, LGMD1C | Recruiting | Virginia Commonwealth University | 1000 | 2023-10-18 | 2025-11-18 | |
| A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related) SRP-6004 - Limb Girdle Muscular Dystrophy | Phase 1 | Terminated | Sarepta Therapeutics, Inc. | 2 | 2023-05-22 | 2025-09-04 |
| A Multicenter Phenotype-Genotype Analysis of LGMD Patients in China Electromyography, IDEAL MRI - LGMD, LGMDR2 | Enrolling by invitation | Huashan Hospital | 450 | 2021-07-07 | 2023-10-24 | |
| Clinical, Immunological, Morphological and Genetic Characteristics of Patients With Dysferlinopathy (LGMD R2) in the RF Dysferlinopathy, Miyoshi Myopathy | Enrolling by invitation | Artgen Biotech | 100 | 2020-01-15 | 2025-06-27 | |
| rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies rAAVrh74.MHCK7.DYSF.DV - Dysferlinopathy | Phase 1 | Completed | Sarepta Therapeutics, Inc. | 2 | 2016-03-01 | 2021-05-13 |
| Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD) ATYR1940, Placebo - Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy | Phase 1 / Phase 2 | Completed | aTyr Pharma, Inc. | 18 | 2015-11-30 | 2023-10-19 |
| Clinical Outcome Study for Dysferlinopathy Dysferlinopathy, LGMD2B Now Classified as LGMDR2 | Unknown | Newcastle-upon-Tyne Hospitals NHS Trust | 200 | 2012-09-01 | 2022-07-26 | |
| Dysferlinopathy Protein in Peripheral Blood Monocytes. Protein analysis - Muscular Dystrophies, Limb-Girdle Muscular Dystrophy Type 2B | Completed | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 149 | 2012-02-01 | 2024-07-18 | |
| Deflazacort in Dysferlinopathies deflazacort, placebo - LGMD2B, Miyoshi Myopathy | Phase 2 / Phase 3 | Completed | Ludwig-Maximilians - University of Munich | 25 | 2003-09-01 | 2015-09-02 |
Dysferlinopathy (4) · Miyoshi myopathy (4) · Muscular Dystrophies (4) · Muscular Dystrophies, Limb-Girdle (4) · Limb-girdle muscular dystrophy type 2A (2) · Limb-Girdle Muscular Dystrophy, Type 1G (1) · Limb-girdle muscular dystrophy type 2F (1) · Limb-girdle muscular dystrophy, type 1B (1) · Limb-girdle muscular dystrophy, type 2C (1) · Limb-girdle muscular dystrophy, type 2E (1) · Muscular Dystrophy, Facioscapulohumeral (1) · Muscular Dystrophy, Limb-Girdle, Type 1C (1) · Muscular Dystrophy, Limb-Girdle, Type 1D (1) · Muscular Dystrophy, Limb-Girdle, Type 1E (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Muscular Diseases (7) · Muscular Disorders, Atrophic (7) · Musculoskeletal Diseases (7) · Nervous System Diseases (7) · Neuromuscular Diseases (7) · Muscular Dystrophies (3) · Cardiomyopathies (1) · Cardiovascular Diseases (1) · Cobblestone Lissencephaly (1) · Congenital Abnormalities (1) · Eye Diseases (1) · Eye Diseases, Hereditary (1) · Heart Diseases (1)
Adrenal Cortex Hormones (1) · Electromyography (1) · deflazacort (1)
Sarepta Therapeutics, Inc. (2) · Artgen Biotech (1) · Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau (1) · Huashan Hospital (1) · Ludwig-Maximilians - University of Munich (1) · Newcastle-upon-Tyne Hospitals NHS Trust (1) · Virginia Commonwealth University (1) · aTyr Pharma, Inc. (1)
United States (5) · Denmark (2) · France (2) · Spain (2) · Chile (1) · China (1) · Germany (1) · Italy (1) · Japan (1) · Russia (1) · South Korea (1) · United Kingdom (1)
Chongbo Zhao, PhD (1) · Jerry R Mendell, MD (1) · Maggie C. Walter, MD (1) · Meredith K James (1) · Nicholas Johnson, MD (1) · Roman Deev, PhD (1) · Volker Straub (1)
PMID 23406536, PMID 29378789, PMID 29735511, PMID 30626655, PMID 35047756, PMID 37553796, PMID 38365661, PMID 39973465
2015 (1)