1 completed, 1 terminated. 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 1, Terminated 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 |
|---|---|---|---|---|---|---|
| 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 |
Limb-girdle muscular dystrophy, type 2B (1) · Muscular Dystrophies, Limb-Girdle (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Muscular Diseases (1) · Muscular Disorders, Atrophic (1) · Muscular Dystrophies (1) · Musculoskeletal Diseases (1) · Nervous System Diseases (1) · Neuromuscular Diseases (1)
Bortezomib (1)
Sarepta Therapeutics, Inc. (1) · University Hospital, Basel, Switzerland (1)
Switzerland (1) · United States (1)
Jerry R Mendell, MD (1) · Michael Sinnreich, Prof. Dr. MD (1)