To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
- Conditions
- Von Willebrand's Disease (VWD)
- Interventions
- Drug: efanesoctocog alfa (BIVV001)
- Registration Number
- NCT04770935
- Lead Sponsor
- Bioverativ, a Sanofi company
- Brief 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).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 6
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efanesoctocog alfa (BIVV001) efanesoctocog alfa (BIVV001) A single IV dose of BIVV001 will be administered to each patient
- Primary Outcome Measures
Name Time Method Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax) Day 1 to Day 10 Pharmacokinetic Parameter: Terminal half-life (t½z) Day 1 to Day 10 Pharmacokinetic Parameter: Total Clearance (CL) Day 1 to Day 10 Pharmacokinetic Parameter: Volume of distribution at steady state (Vss) Day 1 to Day 10 Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞) Day 1 to Day 10 Pharmacokinetic Parameter: Mean residence time (MRT) Day 1 to Day 10 Pharmacokinetic Parameter: Incremental recovery (IR) Day 1 to Day 10
- Secondary Outcome Measures
Name Time Method Number of participants with adverse events Up to Day 29
Trial Locations
- Locations (4)
Investigational Site Number :2500001
🇫🇷Lille, France
Investigational Site Number :2500002
🇫🇷Nantes, France
University of Iowa_Investigational Site Number :8400002
🇺🇸Iowa City, Iowa, United States
Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
🇺🇸Pittsburgh, Pennsylvania, United States