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

Primary Purpose

Von Willebrand's Disease (VWD)

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
efanesoctocog alfa (BIVV001)
Sponsored by
Bioverativ, a Sanofi company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Von Willebrand's Disease (VWD)

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 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.

Sites / Locations

  • University of Iowa_Investigational Site Number :8400002
  • Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
  • Investigational Site Number :2500001
  • Investigational Site Number :2500002

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

efanesoctocog alfa (BIVV001)

Arm Description

A single IV dose of BIVV001 will be administered to each patient

Outcomes

Primary Outcome Measures

Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
Pharmacokinetic Parameter: Terminal half-life (t½z)
Pharmacokinetic Parameter: Total Clearance (CL)
Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)
Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)
Pharmacokinetic Parameter: Mean residence time (MRT)
Pharmacokinetic Parameter: Incremental recovery (IR)

Secondary Outcome Measures

Number of participants with adverse events

Full Information

First Posted
February 22, 2021
Last Updated
April 27, 2023
Sponsor
Bioverativ, a Sanofi company
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1. Study Identification

Unique Protocol Identification Number
NCT04770935
Brief Title
To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD)
Official Title
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)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
May 3, 2021 (Actual)
Primary Completion Date
December 1, 2022 (Actual)
Study Completion Date
December 20, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioverativ, a Sanofi company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Von Willebrand's Disease (VWD)

7. Study Design

Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
efanesoctocog alfa (BIVV001)
Arm Type
Experimental
Arm Description
A single IV dose of BIVV001 will be administered to each patient
Intervention Type
Drug
Intervention Name(s)
efanesoctocog alfa (BIVV001)
Intervention Description
Pharmaceutical form:solution for injection Route of administration: intravenous injection
Primary Outcome Measure Information:
Title
Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)
Time Frame
Day 1 to Day 10
Title
Pharmacokinetic Parameter: Terminal half-life (t½z)
Time Frame
Day 1 to Day 10
Title
Pharmacokinetic Parameter: Total Clearance (CL)
Time Frame
Day 1 to Day 10
Title
Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)
Time Frame
Day 1 to Day 10
Title
Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)
Time Frame
Day 1 to Day 10
Title
Pharmacokinetic Parameter: Mean residence time (MRT)
Time Frame
Day 1 to Day 10
Title
Pharmacokinetic Parameter: Incremental recovery (IR)
Time Frame
Day 1 to Day 10
Secondary Outcome Measure Information:
Title
Number of participants with adverse events
Time Frame
Up to Day 29

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
University of Iowa_Investigational Site Number :8400002
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Investigational Site Number :2500001
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Investigational Site Number :2500002
City
Nantes
ZIP/Postal Code
44093
Country
France

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Learn more about this trial

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

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