NCT04644575

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 261 (actual)
• Sponsor: Bioverativ, a Sanofi company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Primary Objective: \- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). * To evaluate the efficacy of BIVV001 for perioperative management

Detailed description

Participants will receive BIVV001 once weekly for a total of at least 100 exposure days to BIVV001 (including exposure during a BIVV001 parent study, if applicable). 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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	efanesoctocog alfa (BIVV001)	Pharmaceutical form:Solution for Injection Route of administration: Intravenous

Timeline

• Start date: 2021-02-23
• Primary completion: 2027-01-15
• Completion: 2027-01-15
• First posted: 2020-11-25
• Last updated: 2025-08-01

Locations

85 sites across 23 countries: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Netherlands, South Korea, Spain, Sweden, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study