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A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 80

Last updated 2026-05-08

No results posted yet for this study

Summary

This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.

Conditions

Interventions

DRUG

SGT-003

Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)

DRUG

Placebo

IV infusion

Sponsors & Collaborators

  • Solid Biosciences Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
7 Years
Max Age
11 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-22
Primary Completion
2029-01-31
Completion
2034-01-31
FDA Drug
Yes

Countries

  • Australia
  • Canada

Study Locations

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Entities

Drugs
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 NCT07160634 on ClinicalTrials.gov