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

NCT ID: NCT05042440

Last Updated: 2025-09-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-08-11
• Study Completion Date: 2021-11-24

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

Detailed Description

This is a Phase 1, single center, open-label, sequential treatment, 3-period fixed sequence study to assess PK profiles of BIVV001, SHL and EHL rFVIII after a single IV injection in male, previously treated patients, 18-65 years of age, with severe hemophilia A (defined as <1 IU/dL [<1%] endogenous FVIII).

The expected duration of the study is up to approximately 67 days including

• Screening and washout up to 28 days,
• Advate® dosing, PK sampling, including washout: approximately 4 days,
• Adynovi® dosing and PK sampling, including washout: approximately 7 days,
• BIVV001 dosing and PK sampling: approximately 14 days, Remainder of Safety Observation Period: approximately 14 days.

Conditions

Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

efanesoctocog alfa (BIVV001)

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

Group Type: EXPERIMENTAL

Efanesoctocog alfa

Intervention Type: DRUG

Solution for injection Intravenous

Octocog alfa

Intervention Type: DRUG

Solution for injection Intravenous

Rurioctocog alfa pegol

Intervention Type: DRUG

Solution for injection Intravenous

Interventions

Efanesoctocog alfa

Solution for injection Intravenous

Intervention Type: DRUG

Octocog alfa

Solution for injection Intravenous

Intervention Type: DRUG

Rurioctocog alfa pegol

Solution for injection Intravenous

Intervention Type: DRUG

Other Intervention Names

BIVV001; Advate®; Adynovi®

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences and Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational Site Number :1000001

Sofia, , Bulgaria

Countries

Bulgaria

References

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.

Reference Type: BACKGROUND

PMID: 37538505 (View on PubMed)

Related Links

https://www.trialsummaries.com/Study/StudyDetails?id=25325&tenant=MT_SNY_9011

PKM17085 Plain Language Results Summary

Other Identifiers

2021-000228-37

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1256-9661

Identifier Type: REGISTRY

Identifier Source: secondary_id

PKM17085

Identifier Type: -

Identifier Source: org_study_id