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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03368742 | Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | PHASE1 | ACTIVE NOT_RECRUITING | 12 | — | — | Dec 6, 2017 | Oct 15, 2026 | Jun 2, 2026 | 2 | United States |
| Arm | Type | Description |
|---|---|---|
| SGT-001 - Dose Level 1 | EXPERIMENTAL | Single IV infusion of SGT-001 at starting dose |
| SGT-001 - Dose Level 2 | EXPERIMENTAL | Single IV infusion of SGT-001 at next ascending dose |
| Untreated Control | NO_INTERVENTION | Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose. |
| Name | Type | Description |
|---|---|---|
| SGT-001 | GENETIC | AAV9 vector containing muscle-specific promoter and microdystrophin construct |
Inclusion Criteria: * Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype * Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants) * Anti-AAV9 antibodies below protocol-specified thresholds * Stable cardiac and ...