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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

key information

study id #: NCT03368742

condition: Duchenne Muscular Dystrophy

status: suspended


This is a randomized, controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Eligible patients will be randomized to an active treatment group or an untreated control group. Patients in the active treatment group will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. Patients in the untreated control group who continue to meet treatment criteria will receive SGT-001 after 1 year on study.

The protocol was amended to drop the control arm after 4 subjects were dosed. Subjects currently enrolling are randomized to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol.

intervention: SGT-001

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

results: https://clinicaltrials.gov/ct2/show/results/NCT03368742

last updated: June 25, 2020