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)
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Enrollment
- 6
- Locations
- 4
- Primary Endpoint
- Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
Study Overview
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).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Other
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
efanesoctocog alfa (BIVV001)
A single IV dose of BIVV001 will be administered to each patient
Intervention: efanesoctocog alfa (BIVV001) (Drug)
Outcomes
Primary Outcomes
Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
Time Frame: Day 1 to Day 10
Pharmacokinetic Parameter: Terminal half-life (t½z)
Time Frame: Day 1 to Day 10
Pharmacokinetic Parameter: Total Clearance (CL)
Time Frame: Day 1 to Day 10
Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)
Time Frame: Day 1 to Day 10
Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)
Time Frame: Day 1 to Day 10
Pharmacokinetic Parameter: Mean residence time (MRT)
Time Frame: Day 1 to Day 10
Pharmacokinetic Parameter: Incremental recovery (IR)
Time Frame: Day 1 to Day 10
Secondary Outcomes
- Number of participants with adverse events(Up to Day 29)