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

NCT04770935 | Completed Phase 1 FDA Drug

• 3 other identifiers: PKM169782020-004947-10U1111-1255-4463
• Study Type: interventional
• Target: 6
• Locations: 2 countries
• Sites: 4

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

Conditions

Von Willebrand's Disease (VWD)

Outcome Measures

Primary Outcomes (7)

• Pharmacokinetic Parameter: Maximum plasma concentration observed (Cmax)

  Day 1 to Day 10

• Pharmacokinetic Parameter: Terminal half-life (t½z)

  Day 1 to Day 10

• Pharmacokinetic Parameter: Total Clearance (CL)

  Day 1 to Day 10

• Pharmacokinetic Parameter: Volume of distribution at steady state (Vss)

  Day 1 to Day 10

• Pharmacokinetic Parameter: Area under the activity time curve extrapolated to infinity (AUC∞)

  Day 1 to Day 10

• Pharmacokinetic Parameter: Mean residence time (MRT)

  Day 1 to Day 10

• Pharmacokinetic Parameter: Incremental recovery (IR)

  Day 1 to Day 10

Secondary Outcomes (1)

• Number of participants with adverse events

  Up to Day 29

Study Arms (1)

efanesoctocog alfa (BIVV001)

EXPERIMENTAL

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

Drug: efanesoctocog alfa (BIVV001)

Interventions

efanesoctocog alfa (BIVV001) DRUG

Pharmaceutical form:solution for injection Route of administration: intravenous injection

efanesoctocog alfa (BIVV001)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \-- 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bioverativ, a Sanofi company: lead

Study Sites (4)

University of Iowa_Investigational Site Number :8400002

Iowa City, Iowa, 52242, United States

Location

Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001

Pittsburgh, Pennsylvania, 15213, United States

Location

Investigational Site Number :2500001

Lille, 59037, France

Location

Investigational Site Number :2500002

Nantes, 44093, France

Location

Related Links

• PKM16978 Plain Language Results Summary

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

BIVV001

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2021
• First Posted: February 25, 2021
• Study Start: May 3, 2021
• Primary Completion: December 1, 2022
• Study Completion: December 20, 2022
• Last Updated: September 25, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

US (2); FR (2)