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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT03368742 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-08

No results posted yet for this 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

Interventions

GENETIC

SGT-001

AAV9 vector containing muscle-specific promoter and microdystrophin construct

Sponsors & Collaborators

  • Solid Biosciences Inc.

    lead INDUSTRY

Principal Investigators

  • Solid Bio Clinical Trials · Solid Biosciences

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-06
Primary Completion
2026-10-15
Completion
2026-10-15
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03368742 on ClinicalTrials.gov