Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07160634 | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) | PHASE3 | RECRUITING | 80 | — | — | Oct 22, 2025 | Jan 1, 2034 | Jun 2, 2026 | 5 | United States, Australia +1 |
| NCT06138639 | A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | PHASE1 | RECRUITING | 60 | — | — | May 6, 2024 | May 6, 2031 | May 8, 2026 | 15 | United States, Canada +2 |
Microdystrophin expression evaluation in muscle biopsies
| Arm | Type | Description |
|---|---|---|
| SGT-003 followed by Placebo | EXPERIMENTAL | Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2. |
| Placebo followed by SGT-003 | 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. |
| 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. * On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams p...