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SGT-003 · 2 trials · 1 indication
Microdystrophin expression evaluation in muscle biopsies
| Arm | Type | Description |
|---|---|---|
| SGT-003 in Part 1, Placebo in Part 2 | EXPERIMENTAL | Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria. |
| Placebo in Part 1, SGT-003 in Part 2 | EXPERIMENTAL | Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2 per protocol-defined eligibility criteria. |
| Cohort 1: SGT-003 | EXPERIMENTAL | All ambulatory participants from age 4 to \< 7 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 2: SGT-003 | EXPERIMENTAL | All ambulatory participants from age 7 to \< 12 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 3: SGT-003 | EXPERIMENTAL | All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 4: SGT-003 | EXPERIMENTAL | All ambulatory participants from age 12 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 5: SGT-003 | EXPERIMENTAL | All non-ambulatory participants from age 10 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1. |
| Name | Type | Description |
|---|---|---|
| SGT-003 | DRUG | Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5) |
| Placebo | DRUG | IV infusion |
Inclusion Criteria: * Participant is ambulatory. * Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype. * Negative for antibodies against adeno-associated virus serotype 9 (AAV9). * On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or...
SGT-003 is an investigational gene therapy being developed by Solid Biosciences Inc. for Duchenne Muscular Dystrophy, a neuromuscular condition. It is designed to deliver genetic material aimed at addressing the underlying cause of the disease and is currently in clinical development, including a Phase 3 study.
SGT-003 is being developed as a treatment for Duchenne Muscular Dystrophy, a genetic muscle-wasting disorder. It is an investigational gene therapy intended for males with the condition. The Phase 3 IMPACT DUCHENNE study enrolls ambulant males, while the Phase 1 INSPIRE DUCHENNE study includes younger patients.
SGT-003 is a gene therapy that targets integrin receptors. By directing its therapeutic payload toward integrin receptors, the therapy aims to address the molecular mechanisms involved in Duchenne Muscular Dystrophy. This target-based approach distinguishes it from therapies acting on other pathways.
SGT-003 is being developed by Solid Biosciences Inc., a biotechnology company that trades under the ticker symbol SLDB. The company is advancing the gene therapy through clinical development for Duchenne Muscular Dystrophy, including a Phase 3 trial and a Phase 1 trial.
SGT-003 is in Phase 3 clinical development for Duchenne Muscular Dystrophy. The Phase 3 IMPACT DUCHENNE trial is currently recruiting, and an earlier Phase 1 INSPIRE DUCHENNE trial is also ongoing. SGT-003 is investigational and has not been approved by the FDA.
SGT-003 is being evaluated in two recruiting clinical trials. The Phase 3 IMPACT DUCHENNE study, NCT07160634, enrolls 80 ambulant males across the United States, Australia, and Canada. The Phase 1 INSPIRE DUCHENNE study, NCT06138639, enrolls 60 males across the United States, Canada, Italy, and the United Kingdom.