Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Active Phase 1 study of Duchenne Muscular Dystrophy, sponsored by Solid Biosciences.
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. According to ClinicalTrials.gov, the official US trial registry.
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 |
What the registry record for NCT03368742 still lists
NCT03368742 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower).
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 a single indexed study location in 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
Similar trials for Duchenne Muscular Dystrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03368742's enrollment target sits among peer trials
12 41st of 48 higher than 7 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
RECRUITING · Phase 1
-
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
RECRUITING · Phase 1
-
NS-050/NCNP-03 in Boys With DMD (Meteor50)
RECRUITING · Phase 1
-
Trial of Cell Based Therapy for DMD
RECRUITING · Phase 1
-
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
RECRUITING · Phase 1
-
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
COMPLETED · Phase 1
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02602769 · 12 participants
Whole Transcriptome Profiling and Metabolic Phenotyping in Children With ROHHAD Syndrome
- NCT02605421 · 12 participants · Phase 2
Myeloablative Consolidation Therapy and Tandem Autologous Stem Cell Rescue in Patients With High-Risk Neuroblastoma
- NCT02619448 · 12 participants · Early Phase 1
Concurrent Chemotherapy Plus HFR Radiation Therapy in Inoperable NSCLC
- NCT03311503 · 12 participants · Phase 1
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI