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

  • STATUS
    Recruiting
  • End date
    May 25, 2023
  • participants needed
    9
  • sponsor
    Bioverativ, a Sanofi company
Updated on 27 May 2022
aptt
Factor VIII
coagulation factor
bleeding tendency
antihemophilic factor
coagulation studies
ddavp
type 3 vwd

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

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).

Details
Condition Von Willebrand's Disease (VWD)
Treatment efanesoctocog alfa (BIVV001)
Clinical Study IdentifierNCT04770935
SponsorBioverativ, a Sanofi company
Last Modified on27 May 2022

Eligibility

Yes No Not Sure

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

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
Hereditary or acquired coagulation disorder other than VWD (including qualitative and
quantitative platelet disorders, and thrombocytopenia < 100,000 cells/uL at Screening)
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 VWF-
containing 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
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