NCT03368742Active, not recruitingPhase 1/2
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
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
Source: ClinicalTrials.gov · Last updated 15 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 12 (actual)
- First submitted
- 5 Dec 2017
- Start date
- Dec 2017
- Primary completion date
- Nov 2026
- Lead sponsor
- Solid Biosciences Inc.
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT03368742
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 12
- From Dec 2017 to the primary completion date, Nov 2026
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
- Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening
- Additional inclusion/exclusion criteria may apply.
Other illness
- Impaired cardiovascular function based on cardiac MRI or ECHO
Lab values
- Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
Age
- Body mass index ≥ 95th percentile for age
Other
- Abnormal liver function
- Abnormal renal function
- Clinically significant coagulation abnormalities
- Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
- Significant spinal deformity or presence of spinal rods
Who the study is looking for
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
- Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
- Anti-AAV9 antibodies below protocol-specified thresholds
- Stable cardiac and pulmonary function
- Adolescents: non-ambulatory by protocol-specified criteria
- Children: ambulatory by protocol-specified criteria
- Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks
Age in the registry: 4 years to 17 years · Sex: Male · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Florida Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| David Geffen School of Medicine at UCLA Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.