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Recruiting Phase 1/2

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639 · tracked via the Priya Life Science UK tracker
Phase
Phase 1/2
Started
2024-05-06
Last updated
2026-07-08

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

SGT-003

SGT-003: Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

Study 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 dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Eligibility

Sex
MALE
Min age
0 Years
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Incidence of treatment-emergent adverse events (AEs) — Day 360
  • Change from baseline in Microdystrophin Protein Levels — Day 90
    Microdystrophin expression evaluation in muscle biopsies

Trial sites (15)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas Recruiting
University of California, Los Angeles Medical Center Los Angeles California Recruiting
University of California, Davis Sacramento California Recruiting
University of California San Diego California Recruiting
Rare Disease Research Atlanta Georgia Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
Washington University in St. Louis St Louis Missouri Recruiting
Nationwide Children's Hospital Columbus Ohio Recruiting
Oregon Health and Sciences University Portland Oregon Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Children's Hospital of the King's Daughters Norfolk Virginia Recruiting
Seattle Children's Hospital Seattle Washington Recruiting
The Hospital for Sick Children Toronto Ontario Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy Recruiting
Great Ormond Street Hospital London United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06138639 on ClinicalTrials.gov ↗ ← All trials in the UK