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active, not recruiting

Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy

key information

study id #: NCT03375164

condition: Duchenne Muscular Dystrophy

status: active, not recruiting


The proposed clinical trial is a single-dose controlled trial using rAAVrh74.MHCK7.micro-dystrophin for DMD subjects. Cohort A will include six subjects ages 3 months to 3 years, and Cohort B will include six subjects ages 4 to 7 years old. All subjects will receive intravenous micro-dystrophin vector (2X10e14 vg/kg in 10mL/kg)

intervention: rAAVrh74.MHCK7.micro-dystrophin

mechanism of action: Gene therapy to introduce a version of dystrophin

results: https://clinicaltrials.gov/ct2/show/results/NCT03375164

last updated: April 08, 2020