This study is currently not recruiting participants.

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

Study on How an Investigational Medication Works and Its Safety in Adults with Von Willebrand Disease

Not Recruiting
18 years - 65 years
All
Phase 1
6 participants needed

Study Overview

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

Study Details

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).

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Von Willebrand's Disease (VWD)
  • Age: 18 years - 65 years
  • Gender: All

Inclusion criteria :

-- Male and/or female participant, between 18 and 65 years of age, inclusive at the time of informed consent.

  • The participant has been diagnosed with hereditary type 3 VWD or type 2N VWD as documented in historical medical records OR a documented genotype known to produce VWD type 3 or 2N VWD.
  • Type 3 VWD participants are included if they have a medical history of at least 25 exposure days to VWF and factor VIII-containing coagulation factor concentrates
  • Type 2N VWD participants are included if the use of DDAVP is deemed insufficient or contraindicated, as assessed by the Investigator, or if they have required prior use of VWF- and FVIII- containing coagulation factor concentrates.

Exclusion criteria:

  • Hereditary or acquired coagulation disorder other than VWD (including qualitative and quantitative platelet disorders, and thrombocytopenia < 100,000 cells/uL at Screening)
  • The participant has a FVIII activity levels >20 IU/dL, at Screening
  • History or presence of a VWF inhibitor or clinical suspicion of a VWF inhibitor
  • History of a positive FVIII inhibitor test, defined as ≥0.6 BU/mL (by Nijmegen modified Bethesda assay) or a clinical suspicion of a FVIII inhibitor
  • Positive FVIII inhibitor test, defined as ≥0.6 BU/mL, at Screening
  • History of hypersensitivity or anaphylaxis associated with any FVIII- or VWFcontaining product
  • The participant has received or anticipates receiving systemic immunosuppressive or immunomodulatory treatment within 12 weeks prior to Baseline.
  • The participant requires the use of acetylsalicylic acid, non-NSAID anti-platelets, and NSAIDs above the maximum dose product
  • Patients currently on a prophylaxis regimen for the treatment of VWD that, in the Investigator's opinion, would preclude participation in the study due to the possible increased risk of bleeding associated with the requirement to withhold prophylaxis during the study.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

This study investigates the pharmacokinetics and safety of an investigational medication in adults with Type 2N and 3 Von Willebrand Disease (VWD). The purpose of this study is to understand how the investigational medication behaves in the body after a single intravenous dose, focusing on factor VIII activity, which is crucial for blood clotting.

Participants will receive a single intravenous dose of the investigational medication. The study includes monitoring factor VIII activity through blood tests using specific assays. Additionally, the safety and tolerability of the medication will be assessed, ensuring that participants do not experience adverse effects from the treatment.

  • Who can participate: Adults aged 18 to 65 with Type 3 or Type 2N Von Willebrand Disease can participate. They must meet specific medical history and treatment criteria.
  • Study details: Participants will receive a single dose of the investigational medication intravenously. They will undergo blood tests to monitor factor VIII activity and assess the medication's effects. Safety assessments will also be conducted to ensure participant well-being.
  • Study timelines: The study will last up to 57 days.
Updated on 05 Oct 2025. Study ID: NCT04770935