3 completed, 1 enrolling by invitation, 1 terminated, 1 unknown. 2 Phase 1, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 1, Terminated 1 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow Dysferlinopathy: new trials by email Literature Review of Dysferlinopathy trials Research Report on Dysferlinopathy
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 |
|---|---|---|---|---|---|---|
| Observational Cohort Study of Dysferlinopathy in China Dysferlinopathy | Completed | Peking University First Hospital | 160 | 2024-05-01 | 2025-06-24 | |
| 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 |
| Proteasomal Inhibition for Patients With Mis-sense Mutated Dysferlin Bortezomib - Dysferlinopathy | Phase 1 | Terminated | University Hospital, Basel, Switzerland | 3 | 2012-12-01 | 2017-09-21 |
| 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 | |
| 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 |
Limb-girdle muscular dystrophy, type 2B (4) · Miyoshi myopathy (3) · Muscular Dystrophies (2) · Muscular Dystrophies, Limb-Girdle (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Muscular Diseases (3) · Muscular Disorders, Atrophic (3) · Musculoskeletal Diseases (3) · Nervous System Diseases (3) · Neuromuscular Diseases (3) · Muscular Dystrophies (1)
Adrenal Cortex Hormones (1) · Bortezomib (1) · deflazacort (1)
Artgen Biotech (1) · Ludwig-Maximilians - University of Munich (1) · Newcastle-upon-Tyne Hospitals NHS Trust (1) · Peking University First Hospital (1) · Sarepta Therapeutics, Inc. (1) · University Hospital, Basel, Switzerland (1)
United States (2) · Chile (1) · China (1) · Denmark (1) · France (1) · Germany (1) · Italy (1) · Japan (1) · Russia (1) · South Korea (1) · Spain (1) · Switzerland (1) · United Kingdom (1)
Jerry R Mendell, MD (1) · Maggie C. Walter, MD (1) · Meredith K James (1) · Michael Sinnreich, Prof. Dr. MD (1) · Roman Deev, PhD (1) · Volker Straub (1)
PMID 22174839, PMID 22318734, PMID 22736764, PMID 23406536, PMID 29378789, PMID 29735511, PMID 30626655, PMID 35047756, PMID 37553796, PMID 38365661, PMID 39973465