Menu
Recruiting NCT07160634

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

Phase III Interventional Duchenne Muscular Dystrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: SGT-003, Placebo.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: 7 years — 11 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Canada
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy

Overview

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.

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

Primary outcome measures

  • Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 [Time frame: Baseline, Day 540]
Secondary outcome measures (12)
  • Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540 [Time frame: Baseline, Day 540]
  • Change From Baseline in 4-Stair Climb (4SC) Velocity (tasks/s) at Day 540 [Time frame: Baseline, Day 540]
  • Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540 [Time frame: Baseline, Day 540]
  • Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540 [Time frame: Baseline, Day 540]
  • Cumulative Loss of Function in NSAA Items at Day 540 [Time frame: At Day 540]
  • Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90 [Time frame: Baseline, Day 90]
  • Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90 [Time frame: Baseline, Day 90]
  • Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540 [Time frame: Baseline, Day 540]
  • Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540 [Time frame: Baseline, Day 540]
  • Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540 [Time frame: Baseline, Day 540]
  • Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540 [Time frame: Baseline, Day 540]
  • Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of special interest (AESIs), and clinically significant changes in Electrocardiogram (ECG) and Echocardiography (ECHO) [Time frame: From first dose up to Day 540]

Eligibility criteria

Inclusion criteria

  • Participant is ambulatory.
  • Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
  • Negative for antibodies against adeno-associated virus.
  • On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice.
  • Meet 10-meter walk/run time criteria.
  • Meet time to rise from supine criteria.
  • Participant has bodyweight ≤50 kg.

Exclusion criteria

  • Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.
  • Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment.
  • Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report.

Other Inclusion/Exclusion criteria to be applied as per protocol.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 3 centers
  • Arkansas Children's Hospital — Little Rock
  • Neurology Rare Disease Center — Flower Mound
  • Children's Hospital of the King's Daughters — Norfolk
Canada · 2 centers
  • Alberta Children's Hospital — Calgary
  • BC Children's Hospital — Vancouver
Australia · 1 center
  • The Children's Hospital of Westmead — Sydney

Identifiers

NCT: NCT07160634 · SGT-003-301 · 2025-522949-22 · 1013075

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗