Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (NCT03368742)
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
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: David Geffen School of Medicine at UCLA — Los Angeles, California
Age range: 4 Years to 17 Years
🧪 Interventions in This Study
- SGT-001 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (2 Sites)
- David Geffen School of Medicine at UCLA — Los Angeles, California
- University of Florida — Gainesville, Florida
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Duchenne Muscular Dystrophy
- Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy — Little Rock, Arkansas
- Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD — Columbus, Ohio
- NS-050/NCNP-03 in Boys With DMD (Meteor50) — Los Angeles, California
- A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) — Little Rock, Arkansas
- Pediatric Radio Frequency Coils Generic — Cincinnati, Ohio
Explore by condition: All Duchenne Muscular Dystrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.