Gene therapy for Duchenne muscular dystrophy
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This study is testing a new gene therapy called SGT-003 to see if it is safe and effective for children with Duchenne muscular dystrophy.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 0 Years to 17 Years |
| Sex | Male |
| Sponsor | Solid Biosciences Inc. Industry-sponsored |
| Drugs / interventions | prednisone |
| Locations | 15 sites (Little Rock, Arkansas and 14 other locations) |
| Trial ID | NCT06138639 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are ambulatory children aged 4 to less than 12 years with a confirmed diagnosis of DMD and specific genetic mutations.
Not a fit: Patients who have received dystrophin modifying drugs or gene transfer therapies within the last three months may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could significantly improve muscle function and quality of life for children with Duchenne muscular dystrophy.
How similar studies have performed: Other studies have shown promise with gene therapy approaches for DMD, indicating potential for success in this novel intervention.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Little Rock, Arkansas and 14 other locations
- 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)
- Washington University in St. Louis — St Louis, Missouri, United States (Recruiting)
- Nationwide Children's Hospital — Columbus, Ohio, United States (Recruiting)
- Oregon Health and Sciences University — Portland, Oregon, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- Children's Hospital of the King's Daughters — Norfolk, Virginia, United States (Recruiting)
- Seattle Children's Hospital — Seattle, Washington, United States (Recruiting)
- The Hospital for Sick Children — Toronto, Ontario, Canada (Recruiting)
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome, Italy (Recruiting)
- Great Ormond Street Hospital — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Solid Bio Clinical Trials
- Email: clinicaltrials@solidbio.com
- Phone: 617-337-4680
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.