SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, double-blind, placebo-controlled trial testing a single IV infusion of the AAV-based gene therapy SGT-003 in ambulant pediatric males with genetically confirmed DMD. Participants are randomized 1:1 to receive SGT-003 in Part 1 followed by placebo in Part 2, or placebo in Part 1 followed by SGT-003 in Part 2, with each participant observed across both parts. Key eligibility includes ambulatory status, weight ≤50 kg, negative anti-AAV antibodies, and stable corticosteroid treatment, and participants will be followed for safety and efficacy measures with at least five years of long-term follow-up after SGT-003 dosing. Primary outcomes focus on the effect of the single infusion on motor function while secondary outcomes and safety endpoints capture additional clinical effects and adverse events.

Why it matters

Who can join: the full rules

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 serotype 9 (AAV9).
* 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.

Where it's running

• The Children's Hospital of Westmead
  Sydney, New South Wales, Australia Recruiting
• Alberta Children's Hospital
  Calgary, Alberta, Canada Recruiting
• BC Children's Hospital
  Vancouver, British Columbia, Canada Recruiting
• The Hospital for Sick Children (SickKids)
  Toronto, Ontario, Canada Recruiting
• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Not yet recruiting
• Neurology Rare Disease Center
  Flower Mound, Texas, United States Not yet recruiting

Study contacts

• Study coordinator: Solid Bio Clinical Trials
• Email: clinicaltrials@solidbio.com
• Phone: 6173374680

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT07766980

• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

  This study tests whether KER-065 is safe and can help with symptoms in males with Duchenne muscular dystrophy, including both ambulatory and nonambulatory participants who are on standard care.

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• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

  This study tests a gene therapy (GNR-097) to see if it is safe, tolerable, and potentially beneficial for ambulatory boys with Duchenne muscular dystrophy.

  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

• ELEVIDYS safety in Duchenne patients treated after market approval

  This study tests the safety of ELEVIDYS, with sirolimus as a protective treatment, in male children and young adults with Duchenne Muscular Dystrophy who have previously received ELEVIDYS or are eligible to receive it commercially.

  Recruiting Phase 4 Sarepta Therapeutics, Inc. · Houston, Texas · NCT07542314

• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

  This study is testing a new drug called Givinostat to see if it's safe and how it works in young children aged 2 to 6 with Duchenne Muscular Dystrophy.

  Recruiting at 7 sites Phase 2 Italfarmaco · Brussels and 8 other locations · NCT06769633

• Evaluating respiratory functions and exercise capacity in children with neuromuscular diseases

  This study is trying to see how well children with Duchenne Muscular Dystrophy and Spinal Muscular Atrophy can breathe and exercise compared to healthy kids.

  Recruiting Lokman Hekim University · Ankara, Çankaya · NCT06756633

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.