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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07180355 | A Study of SGT-212 Gene Therapy in Friedreich's Ataxia | PHASE1 | RECRUITING | 10 | — | — | Oct 22, 2025 | Feb 29, 2032 | Apr 27, 2026 | 3 | United States |
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (Non-Ambulatory) | EXPERIMENTAL | Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion. |
| Cohort 2 (Ambulatory) | EXPERIMENTAL | Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion. |
| Cohort 3 (Ambulatory and Non-Ambulatory) | EXPERIMENTAL | Participants will receive bilateral IDN followed by systemic IV infusion. |
| Name | Type | Description |
|---|---|---|
| SGT-212 | DRUG | Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA) |
Inclusion Criteria: * Has history of FA symptom onset ≤25 years of age * Has a clinical and genetic diagnosis of FA * Has a staging score of ≥1 but \<6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score * Is willing to agree to the following rules for use of omavelox...