Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A

NCT05042440 | Completed Phase 1

• 3 other identifiers: PKM170852021-000228-37U1111-1256-9661
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

Primary objective

• To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives
• To characterize additional pharmacokinetic (PK) parameters of BIVV001, SHL rFVIII and EHL rFVIII after a single IV injection
• To evaluate the safety and tolerability of a single IV injection of BIVV001

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (3)

• Half-life of BIVV001

  BIVV001 period: Predose, and post-dose from 0.17 hr to 336 hr, and at day 28

• Half-life of SHL rFVIII

  Advate® period: Predose, and post-dose from 0.17 hr to 72 hr

• Half-Life of EHL rFVIII

  Adynovi® period: Predose, and post-dose from 0.17 hr to 120 hr

Secondary Outcomes (8)

• Assessment of pharmacokinetic (PK) parameter : maximum activity (Cmax)

  Predose of each period, 0.17 hr to 72 hr for Advate® ; 0.17 hr to 120 hr for Adynovi® ; 0.17 hr to 336 hr and at day 28 for BIVV001

• Assessment of PK parameter : clearance (CL)

  Predose of each period, 0.17 hr to 72 hr for Advate® ; 0.17 hr to 120 hr for Adynovi® ; 0.17 hr to 336 hr and at day 28 for BIVV001

• Assessment of PK parameter : volume of distribution at steady state (Vss)

  Predose of each period, 0.17 hr to 72 hr for Advate® ; 0.17 hr to 120 hr for Adynovi® ; 0.17 hr to 336 hr and at day 28 for BIVV001

• Assessment of PK parameter : area under the activity time curve extrapolated to infinity (AUC∞)

  Predose of each period, 0.17 hr to 72 hr for Advate® ; 0.17 hr to 120 hr for Adynovi® ; 0.17 hr to 336 hr and at day 28 for BIVV001

• Assessment of PK parameter : mean residence time (MRT)

  Predose of each period, 0.17 hr to 72 hr for Advate® ; 0.17 hr to 120 hr for Adynovi® ; 0.17 hr to 336 hr and at day 28 for BIVV001

Study Arms (1)

efanesoctocog alfa (BIVV001)

EXPERIMENTAL

Each participant will be sequentially dosed with three single intravenous (IV) doses of first rFVIII (Advate®), second Polyethylene Glycol (PEG)-rFVIII (Adynovi® or Adynovate®), and lastly, BIVV001

Drug: Efanesoctocog alfa; Drug: Octocog alfa; Drug: Rurioctocog alfa pegol

Interventions

Efanesoctocog alfa DRUG

Solution for injection Intravenous

Also known as: BIVV001

efanesoctocog alfa (BIVV001)

Octocog alfa DRUG

Solution for injection Intravenous

Also known as: Advate®

efanesoctocog alfa (BIVV001)

Rurioctocog alfa pegol DRUG

Solution for injection Intravenous

Also known as: Adynovi®

efanesoctocog alfa (BIVV001)

Eligibility Criteria

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

You may qualify if:

• Severe hemophilia A, defined as \<1 IU/dL (\<1%) endogenous FVIII activity.
• Previous treatment for hemophilia A, defined as at least 150 days documented prior exposure to any recombinant and/or plasma-derived FVIII and/or cryoprecipitate products at Day 1.
• Platelet count ≥100,000 cells/µL at Screening.
• A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment. (CD4 lymphocyte count \>200 cells/mm³ - Viral load of \<400 copies/mL).

You may not qualify if:

• Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis.
• Serious active bacterial, fungal or viral infection (other than chronic hepatitis or HIV) present within 30 days of Screening.
• Other known coagulation disorder(s) in addition to hemophilia A.
• History of hypersensitivity or anaphylaxis associated with any FVIII product.
• History of a positive inhibitor test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant.
• Positive inhibitor result, defined as ≥0.6 BU/mL at Screening.
• Major surgery within 8 weeks of Screening.
• Sensitivity to any of the study interventions, or components thereof, or drug or other allergy.
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Investigational Site Number :1000001

Sofia, 1756, Bulgaria

Location

Related Publications (1)

• Lissitchkov T, Willemze A, Jan C, Zilberstein M, Katragadda S. Pharmacokinetics of recombinant factor VIII in adults with severe hemophilia A: fixed-sequence single-dose study of octocog alfa, rurioctocog alfa pegol, and efanesoctocog alfa. Res Pract Thromb Haemost. 2023 May 13;7(4):100176. doi: 10.1016/j.rpth.2023.100176. eCollection 2023 May.

  PMID: 37538505 BACKGROUND

Related Links

• PKM17085 Plain Language Results Summary

MeSH Terms

Conditions

Hemophilia A

Interventions

BIVV001; F8 protein, human; Factor VIII; BAX 855

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Clinical Sciences and Operations

  Sanofi

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: August 12, 2021
• First Posted: September 13, 2021
• Study Start: August 11, 2021
• Primary Completion: November 24, 2021
• Study Completion: November 24, 2021
• Last Updated: September 18, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

BU (1)