rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
Status:
Completed
Trial end date:
2019-07-01
Target enrollment:
Participant gender:
Summary
The proposed clinical trial is a double-blind, randomized controlled study with direct
intramuscular injection of rAAVrh.74.MHCK7.DYSF.DV gene vector to the extensor digitorum
brevis muscle (EDB). Two cohorts of subjects with dysferlin deficiency, each with proven
mutations will undergo gene transfer. A minimum of three subjects will be enrolled into each
cohort.
Phase:
Phase 1
Details
Lead Sponsor:
Nationwide Children's Hospital Sarepta Therapeutics, Inc.