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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04644575 | Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A | PHASE3 | ACTIVE NOT_RECRUITING | 261 | — | — | Feb 23, 2021 | Jan 15, 2027 | Aug 1, 2025 | 85 | United States, Argentina +21 |
| NCT04759131 | Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A | PHASE3 | COMPLETED | 74 | — | — | Feb 19, 2021 | Jan 18, 2023 | Sep 11, 2025 | 40 | United States, Australia +13 |
| NCT05042440 | Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A | PHASE1 | COMPLETED | 13 | — | — | Aug 11, 2021 | Nov 24, 2021 | Sep 18, 2025 | 1 | Bulgaria |
The number of participants with the occurrence of inhibitor development (neuatralizing antibodies detected against factor VIII \[FVIII\]) as determined via the Nijmegen modified Bethesda assay.
Inhibitor development was defined as an inhibitor result of greater than or equal to (\>=0.6) Bethesda units (BU/mL) that was confirmed by a second test result from a separate sample, drawn 2 to 4 weeks following the date when the original sample was drawn. Both tests must have been performed by the central laboratory using the Nijmegen modified Bethesda assay.
| Arm | Type | Description |
|---|---|---|
| Arm A: Previously treated in BIVV001 study | EXPERIMENTAL | This arm includes participants who have completed study EFC16293 or study EFC16295, participants who have completed Arm B or Arm C of this study (LTS16294) and roll over into Arm A, and participants who have completed any other potential BIVV001 study. Participants in this arm will continue receiving BIVV001 prophylaxis treatment once weekly (QW) for a total of 100 exposure days (EDs) cumulative from the parent study and this study. Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country. |
| Arm B: Newly initiated (China Only) in BIVV001 | EXPERIMENTAL | This arm includes Chinese participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) for 52 weeks. After 52 weeks of treatment in this arm B, participants will be able to roll over into arm A. |
| Arm C: Newly initiated in BIVV001 with planned major surgery | EXPERIMENTAL | This arm includes participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) and will undergo planned major surgery after at least 6 initial EDs with BIVV001, and within 26 weeks from Day 1. After 52 weeks of treatment in arm C, participants will be able to roll into arm A. |
| BIVV001: Participants aged <6 Years | EXPERIMENTAL | Participants aged less than (\<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks. |
| BIVV001: Participants aged 6 to <12 Years | EXPERIMENTAL | Participants aged 6 to \<12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks. |
| efanesoctocog alfa (BIVV001) | EXPERIMENTAL | Each participant will be sequentially dosed with three single intravenous (IV) doses of first rFVIII (Advate®), second Polyethylene Glycol (PEG)-rFVIII (Adynovi® or Adynovate®), and lastly, BIVV001 |
| Name | Type | Description |
|---|---|---|
| efanesoctocog alfa (BIVV001) | DRUG | Pharmaceutical form:Solution for Injection Route of administration: Intravenous |
| Efanesoctocog alfa | DRUG | Solution for injection Intravenous |
| Octocog alfa | DRUG | Solution for injection Intravenous |
| Rurioctocog alfa pegol | DRUG | Solution for injection Intravenous |
Inclusion criteria : * For participants rolling over into Arm A * Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study. * Male or Female * For participants new to BIVV001 (Arm B and C) * Participants who ha...