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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03861273 | A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B | PHASE3 | ACTIVE NOT_RECRUITING | 51 | — | — | Jul 29, 2019 | Feb 25, 2031 | Jun 1, 2026 | 67 | United States, Australia +14 |
| NCT03307980 | Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B | PHASE2 | ACTIVE NOT_RECRUITING | 21 | — | — | Jun 22, 2017 | Oct 25, 2029 | May 12, 2026 | 25 | United States, Australia +2 |
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...