Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Public ClinicalTrials.gov record NCT03368742. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Study identification
- NCT ID
- NCT03368742
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Solid Biosciences Inc.
- Industry
- Enrollment
- 12 participants
Conditions and interventions
Conditions
Interventions
- SGT-001 Genetic
Genetic
Eligibility (public fields only)
- Age range
- 4 Years to 17 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 5, 2017
- Primary completion
- Oct 14, 2026
- Completion
- Oct 14, 2026
- Last update posted
- May 7, 2026
2017 – 2026
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| David Geffen School of Medicine at UCLA | Los Angeles | California | 90095 | — |
| University of Florida | Gainesville | Florida | 32610 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03368742, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 7, 2026 · Synced May 10, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03368742 live on ClinicalTrials.gov.