NCT04770935

To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)

A Phase 1, Open-Label Study to Assess the Pharmacokinetics, and Safety and Tolerability of a Single Intravenous Injection of rFVIIIFc-VWF-XTEN (BIVV001) in Adults With Type 2N and 3 Von Willebrand Disease (VWD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Bioverativ, a Sanofi company · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: -To characterize the pharmacokinetics (PK) of BIVV001 after a single intravenous (IV) administration, as assessed by factor VIII (FVIII) activity determined by the one-stage activated partial thromboplastin time (aPPT) clotting assay, as well as, BIVV001 capture chromogenic Coatest FVIII activity assay Secondary Objective: -To assess the safety and tolerability of a single IV dose of BIVV001 in adult patients with type 2N and 3 VWD

Detailed description

Duration of each part of the study for one participant: Total study duration: Up to 57 days. * Screening: up to 28 days. * Up to 29 days of safety observation following the IV BIVV001 dose administration (this period includes PK sampling up to the first 10 days following administration).

Conditions

• Von Willebrand's Disease (VWD)

Interventions

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

Timeline

• Start date: 2021-05-03
• Primary completion: 2022-12-01
• Completion: 2022-12-20
• First posted: 2021-02-25
• Last updated: 2025-09-25

Locations

4 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study