Status:
RECRUITING
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Lead Sponsor:
Solid Biosciences Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
Up to 17 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dy...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
May 6 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 6 2031
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT06138639
Start Date
May 6 2024
End Date
May 6 2031
Last Update
December 22 2025
Active Locations (15)
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1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
2
University of California, Los Angeles Medical Center
Los Angeles, California, United States, 90095
3
University of California, Davis
Sacramento, California, United States, 95817
4
University of California
San Diego, California, United States, 92037