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 — XTEND-1  

Factor VIII Deficiency Clinical Trial

Official title: 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

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Factor VIII Deficiency
• Hemophilia A

• NCT number: NCT04161495
• Study type: Interventional
• Source: Sanofi
• Status: Completed
• Phase: Phase 3
• Start date: November 19, 2019
• Completion date: February 3, 2022