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NCT Number: NCT06138639

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

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

0 year–17 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

The Hospital for Sick Children, Toronto, Ontario, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SGT-003

Genetic

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

Primary outcomes

  1. Incidence of treatment-emergent adverse events (AEs)

    Time frame: Day 360

  2. Change from baseline in Microdystrophin Protein Levels

    Time frame: Day 90

    Microdystrophin expression evaluation in muscle biopsies

Secondary outcomes

  1. Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF)

    Time frame: Day 90, Day 360

  2. Change from baseline in Microdystrophin Protein Levels

    Time frame: Day 360

    Microdystrophin expression evaluation in muscle biopsies

  3. Change from Baseline in Time to Rise Velocity

    Time frame: Day 360, Day 540

    The Time to rise from supine (TTR) (s) will be captured as part of item 12 of the North Star Ambulatory Assessment (NSAA).

    Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.

  4. Change from baseline in Stride Velocity 95th Centile (SV95C)

    Time frame: Day 360, Day 540

    Assessment of peak ambulatory performance captured by wearable activity monitoring device.

  5. Change from baseline in 10-meter walk/run velocity

    Time frame: Day 360, Day 540

    The 10MWR (s) will be captured as part of item 17 of the NSAA. Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.

  6. Change from baseline in 4-stair climb velocity

    Time frame: Day 360, Day 540

  7. Change from baseline in North Star Ambulatory Assessment (NSAA) total score

    Time frame: Day 360, Day 540

    Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.

  8. Change from baseline in 6-minute walk test (6MWT) distance

    Time frame: Day 360, Day 540

  9. Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters

    Time frame: Through Day 360 and Day 540

  10. Number of Participants with Clinically Significant Abnormalities in Vital Signs

    Time frame: Through Day 360 and Day 540

  11. Number of Participants with Clinically Significant Abnormalities in Physical Examinations

    Time frame: Through Day 360 and Day 540

  12. Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) or Echocardiography (ECHO)

    Time frame: Through Day 360 and Day 540

Study contacts

Contact information is provided by the study sponsor or research team.

Solid Bio Clinical Trials

CONTACT

[email protected]

617-337-4680

Sponsors and collaborators

Lead sponsor

Solid Biosciences Inc.

Industry

Registry information

Official study title

A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Important dates

Study start
2024
Primary completion
2027
Study completion
2031
First posted
Nov 18, 2023
Registry last updated
Jul 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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