Gene therapy for Duchenne muscular dystrophy

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 multicenter, open-label study investigates the safety, tolerability, and efficacy of a single intravenous infusion of SGT-003 in children with Duchenne muscular dystrophy (DMD). The study includes two cohorts: participants aged 4 to less than 7 years and those aged 7 to less than 12 years. All participants will receive SGT-003 and will be followed for a total of 5 years to assess long-term outcomes.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Cohort 1: 4 to \<7 years of age
* Cohort 2: 7 to \<12 years of age
* Cohort 3: 0 to \< 4 years of age
* Cohort 4: 12 to \< 18 years of age
* Cohort 5: 10 to \< 18 years of age
* Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in \< 30 seconds:

  * Cohorts 1, 2, and 4: Ambulatory
  * Cohort 3: Either ambulatory or non-ambulatory
  * Cohort 5: Non-ambulatory, but having been previously ambulatory by history
* Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
* Negative for AAV antibodies.
* Steroid regimen:

  * Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.
  * Cohort 3: N/A
* Meet 10-meter walk/run time criteria
* Meet time to rise from supine criteria
* Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
* Participant has body weight: ≤ 90 kg

Exclusion Criteria:

* Treatment with dystrophin modifying drugs within 3 months prior to screening.
* Current or prior treatment with an approved or investigational gene transfer drug.
* Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
* Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.

Other inclusion or exclusion criteria apply.

Where it's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• University of California, Los Angeles Medical Center
  Los Angeles, California, United States Recruiting
• University of California, Davis
  Sacramento, California, United States Recruiting
• University of California
  San Diego, California, United States Recruiting
• Rare Disease Research
  Atlanta, Georgia, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting

Study contacts

• Study coordinator: Solid Bio Clinical Trials
• Email: clinicaltrials@solidbio.com
• Phone: 617-337-4680

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

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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

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

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

  Recruiting at 4 sites Phase 3 Solid Biosciences Inc. · Little Rock, Arkansas and 6 other locations · NCT07160634

• 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

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.