Back to results

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)

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

NCT ID

NCT04770935

First Posted

February 22, 2021

Last Updated

April 27, 2023

Sponsor

Bioverativ, a Sanofi company

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

Data Monitoring Committee

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

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)

We'll reach out to this number within 24 hrs