Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as PF-06838435 (formerly SPK-9001), PF-06838435
Fidanacogene elaparvovec · 2 trials · 1 indication
ABR = number of total bleeding episodes on study during the given time period) \*365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
| Arm | Type | Description |
|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | EXPERIMENTAL | - |
| PF-06838435 Dose-Escalation | EXPERIMENTAL | Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken. |
| Name | Type | Description |
|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | BIOLOGICAL | Gene Therapy |
| PF-06838435 (formerly SPK-9001) | BIOLOGICAL | Gene Therapy: A novel, bioengineered adeno-associated viral vector carrying human factor IX variant |
Inclusion Criteria * Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study. * Documented moderately severe to severe hemophilia B (Factor IX activity \< =2%) * Previous experience with FIX t...