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)

Status: Recruiting
Location: See all (12) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: Male
Maximum Age: 17
Healthy Volunteers: f
View:

• Cohort 1: 4 to \<7 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

Locations
United States
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
California
University of California, Los Angeles Medical Center
RECRUITING
Los Angeles
University of California, Davis
RECRUITING
Sacramento
University of California
RECRUITING
San Diego
Georgia
Rare Disease Research
RECRUITING
Atlanta
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Missouri
Washington University in St. Louis
RECRUITING
St Louis
Ohio
Nationwide Children's Hospital
RECRUITING
Columbus
Virginia
Children's Hospital of the King's Daughters
RECRUITING
Norfolk
Other Locations
Canada
The Hospital for Sick Children
RECRUITING
Toronto
Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITING
Rome
United Kingdom
Great Ormond Street Hospital
RECRUITING
London
Contact Information
Primary
Solid Bio Clinical Trials
clinicaltrials@solidbio.com
617-337-4680
Time Frame
Start Date: 2024-05-06
Estimated Completion Date: 2031-05-06
Participants
Target number of participants: 40
Treatments
Experimental: Cohort 1: SGT-003
All ambulatory participants from age 4 to \< 7 years will receive a single IV infusion of SGT-003 on Day 1.
Experimental: Cohort 2: SGT-003
All ambulatory participants from age 7 to \< 12 years will receive a single IV infusion of SGT-003 on Day 1.
Experimental: Cohort 3: SGT-003
All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1.
Experimental: Cohort 4: SGT-003
All ambulatory participants from age 12 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.
Experimental: Cohort 5: SGT-003
All non-ambulatory participants from age 10 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.
Sponsors
Leads: Solid Biosciences Inc.

This content was sourced from clinicaltrials.gov