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

Launched by SOLID BIOSCIENCES INC. · Nov 14, 2023

Keywords

ClinConnect Summary

The INSPIRE DUCHENNE trial is studying a new gene therapy called SGT-003 to see if it is safe and effective for boys with Duchenne Muscular Dystrophy (DMD). This condition affects muscle strength and movement, and the trial will involve two groups of boys: those aged 4 to under 7 years old and those aged 7 to under 12 years old. All participants will receive a single infusion of the treatment through an IV and will be followed for five years to monitor their health and progress.

To be eligible for this trial, boys need to be able to walk without help, have a confirmed diagnosis of DMD with specific genetic mutations, and weigh 50 kg or less. They should also be on a stable dose of certain medications for at least 12 weeks before joining. Participants can expect regular check-ups and assessments during the study to help researchers understand how the therapy works over time. This trial is currently recruiting participants, and it offers a potential new option for managing DMD in young boys.

Gender

MALE

Eligibility criteria

• 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 in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.
• • Other inclusion or exclusion criteria apply.