NCT04161495

A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A

A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients ≥12 Years of Age With Severe Hemophilia A

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 159 (actual)
• Sponsor: Bioverativ, a Sanofi company · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: \- To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. 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 the 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 outcomes. * To evaluate the efficacy of BIVV001 for perioperative management. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the pharmacokinetics (PK) of BIVV001 based on the 1-stage activated partial thromboplastin time (aPTT) and 2-stage chromogenic coagulation factor VIII (FVIII) activity assays.

Detailed description

Participants in prophylaxis arm received a weekly prophylactic dose of BIVV001 for 52 weeks. Participants in on-demand arm received BIVV001 on demand for 26 weeks followed by a switch to weekly prophylaxis for another 26 weeks. The Sponsor planned to perform a long-term safety trial. Enrollment in this open-label extension study would be offered to participants completing the treatment period based on eligibility criteria.

Conditions

• Factor VIII Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	efanesoctocog alfa (BIVV001)	Pharmaceutical form: solution for injection Route of administration: IV injection

Timeline

• Start date: 2019-11-19
• Primary completion: 2022-02-03
• Completion: 2022-02-03
• First posted: 2019-11-13
• Last updated: 2025-09-17
• Results posted: 2023-05-24

Locations

47 sites across 19 countries: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Greece, Hungary, Italy, Japan, Mexico, Netherlands, South Korea, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study