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NCT03368742 · ClinicalTrials.gov registry record · Phase 1
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
A Phase 1 study of Duchenne Muscular Dystrophy, sponsored by Solid Biosciences.
- Active
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 2
- Study locations
NCT03368742 is a Phase 1 study of Duchenne Muscular Dystrophy that is active but no longer recruiting, run by Solid Biosciences. The registered enrollment target is 12 participants, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower). The trial reports 2 study locations across 2 states.
The verdict
NCT03368742, a Phase 1 study of Duchenne Muscular Dystrophy, is active but no longer recruiting, sponsored by Solid Biosciences.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 2
- Study locations
Study 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.
Conditions Studied
Interventions
- GENETIC SGT-001
Study Locations (2)
California
- David Geffen School of Medicine at UCLA - Los Angeles
Florida
- University of Florida - Gainesville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2017-12-06 |
| Est. Completion | 2026-10-15 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03368742
The ClinicalTrials.gov registry entry for NCT03368742 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower). The listed sponsor is Solid Biosciences, which has 6 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which SGT-001 is the first listed.
NCT03368742 reports 2 study locations spanning 2 distinct geographic areas - top geographies include California, Florida.
Frequently Asked Questions
What is clinical trial NCT03368742 about?
NCT03368742 is a clinical study titled "Microdystrophin Gene Transfer Study in Adolescents and Children With DMD". 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....
What is the current status of trial NCT03368742?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2017-12-06. Estimated completion is 2026-10-15.
What conditions does trial NCT03368742 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT03368742?
The interventions under investigation include: SGT-001 (GENETIC).
Who is sponsoring clinical trial NCT03368742?
This trial is sponsored by Solid Biosciences, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03368742 being conducted?
This trial has 2 study locations across California, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.