NCT06138639
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Recruiting
PHASE1Ages 0–17InterventionalTreatmentSolid Biosciences Inc.
~60 participants
Updated 2026-07-08 on ClinicalTrials.gov
What's tested:SGT-003
At a glance
Recruiting sites
15 of 15 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of treatment-emergent adverse events (AEs)
Measured over Day 360
+1 more outcome measured
Conditions
Where it's being run
15 sites across 13 statesCalifornia3
Arkansas1
Georgia1
Illinois1
Missouri1
Ohio1
Oregon1
Pennsylvania1
Study leadership
- Solid Bio Clinical Trials · STUDY_DIRECTOR · Solid Biosciences
Who to contact
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Do you actually qualify for this trial?
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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.
What this trial measures
- Incidence of treatment-emergent adverse events (AEs)Day 360
- Change from baseline in Microdystrophin Protein LevelsDay 90
Microdystrophin expression evaluation in muscle biopsies