clinical trial · NCT03368742
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Duchenne Muscular DystrophySGT-001
brief summary
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.
started
Dec 6, 2017
primary completion
Oct 15, 2026
completion
Oct 15, 2026
last updated
Mar 3, 2026
official title
A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol