Brief Title
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Official Title
A Phase 1, Single-Site, Open-Label Study to Assess Pharmacokinetics of Efanesoctocog Alfa (BIVV001), Standard Half-Life and Extended Half-Life FVIII After Each Single Intravenous Injection in a Fixed Sequence, in Previously Treated Adults With Severe Hemophilia A
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.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Half-life of BIVV001
Secondary Outcome
Assessment of pharmacokinetic (PK) parameter : maximum activity (Cmax)
Condition
Hemophilia A
Intervention
Efanesoctocog alfa
Study Arms / Comparison Groups
efanesoctocog alfa (BIVV001)
Description: 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
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
13
Start Date
August 11, 2021
Completion Date
November 24, 2021
Primary Completion Date
November 24, 2021
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.
Gender
Male
Ages
18 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Clinical Sciences and Operations, ,
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT05042440
Organization ID
PKM17085
Secondary IDs
2021-000228-37
Responsible Party
Sponsor
Study Sponsor
Sanofi
Study Sponsor
Clinical Sciences and Operations, Study Director, Sanofi
Verification Date
April 6, 2022