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

Last updated: June 12, 2025
Sponsor: Solid Biosciences Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Treatment

SGT-003

Clinical Study ID

NCT06138639
SGT-003-101
  • Ages < 17
  • Male

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 Criteria

Inclusion

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, asdefined 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 mutationpredictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where agenotype may be predictive of residual dystrophin production and/or a clear clinicaldiagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levelsin baseline muscle biopsies may be required to determine eligibility under thiscriterion.

  • Negative for AAV antibodies.

  • Steroid regimen:

  • Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior toScreening Part A or Rescreening, allowing for weight-based modificationsconsistent 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

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 toscreening or 5 half-lives since last administration, whichever is longer.

  • Established clinical diagnosis of DMD that is associated with any deletion mutationin exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a geneticreport and confirmed by Sponsor genetic testing.

Other inclusion or exclusion criteria apply.

Study Design

Total Participants: 40
Treatment Group(s): 1
Primary Treatment: SGT-003
Phase: 1/2
Study Start date:
May 06, 2024
Estimated Completion Date:
May 06, 2031

Connect with a study center

  • The Hospital for Sick Children

    Toronto, Ontario M5G 0A4
    Canada

    Active - Recruiting

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Rome, 00168
    Italy

    Active - Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Active - Recruiting

  • University of California, Los Angeles Medical Center

    Los Angeles, California 90095
    United States

    Active - Recruiting

  • University of California, Davis

    Sacramento, California 95817
    United States

    Active - Recruiting

  • Rare Disease Research

    Atlanta, Georgia 30329
    United States

    Active - Recruiting

  • Washington University in St. Louis

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio 43215
    United States

    Active - Recruiting

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia 23510
    United States

    Active - Recruiting

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