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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01687608 | Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B | PHASE1 | ACTIVE NOT_RECRUITING | 30 | — | — | Feb 11, 2013 | Jan 17, 2030 | Mar 20, 2026 | 15 | United States |
| Arm | Type | Description |
|---|---|---|
| AskBio009 Dose Escalation | EXPERIMENTAL | Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy |
| Name | Type | Description |
|---|---|---|
| AskBio009 | BIOLOGICAL | Single dose IV injection |
Inclusion Criteria: * Males age 18-75 years, inclusive * Established hemophilia B with ≥3 hemorrhages per year requiring treatment with exogenous FIX OR use of FIX prophylaxis because of history of frequent bleeding episodes * Plasma FIX activity ≤2% (\<1% for first cohort; then per protocol) * Neg...