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

Recruiting now · Phase 1/Phase 2

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID: NCT06138639
Run by: Solid Biosciences Inc.
People needed: 60
Starts: 2024-05-06
Expected to finish: 2031-05-06
Last updated by the study team: 2026-07-08

Who can join

Age: any, up to 17. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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 is running

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

Full record on ClinicalTrials.gov

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