A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Purpose
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.
Condition
- Duchenne Muscular Dystrophy
Eligibility
- Eligible Ages
- Between 0 Years and 17 Years
- Eligible Sex
- Male
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
Arkansas Children's Hospital
Little Rock, Arkansas 72202
Little Rock, Arkansas 72202
University of California, Los Angeles Medical Center
Los Angeles, California 90095
Los Angeles, California 90095
University of California, Davis
Sacramento, California 95817
Sacramento, California 95817
University of California
San Diego, California 92037
San Diego, California 92037
Rare Disease Research
Atlanta, Georgia 30329
Atlanta, Georgia 30329
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611-2605
Chicago, Illinois 60611-2605
Washington University in St. Louis
St Louis, Missouri 63110
St Louis, Missouri 63110
Nationwide Children's Hospital
Columbus, Ohio 43215
Columbus, Ohio 43215
Oregon Health and Sciences University
Portland, Oregon 97239
Portland, Oregon 97239
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
Children's Hospital of the King's Daughters
Norfolk, Virginia 23510
Norfolk, Virginia 23510
Seattle Children's Hospital
Seattle, Washington 98105
Seattle, Washington 98105
More Details
- Status
- Recruiting
- Sponsor
- Solid Biosciences Inc.