Brief Title
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Official Title
A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Pediatric Patients <12 Years of Age With Severe Hemophilia A
Brief Summary
Primary Objective:
- To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia
A
Secondary Objectives:
- To evaluate the efficacy of BIVV001 as a prophylaxis treatment
- To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes
- To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes
- To evaluate the effect of BIVV001 prophylaxis on joint health outcomes
- To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes
- To evaluate the efficacy of BIVV001 for perioperative management
- To evaluate the safety and tolerability of BIVV001 treatment
- To assess the pharmacokinetics (PK) of BIVV001
Detailed Description
Study duration per participants will be approximately 60 weeks (maximum 8 weeks for screening
and 52 weeks of treatment)
All participants completing or remaining at the end of study will be offered participation in
the planned extension trial.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Occurrence of inhibitor development
Secondary Outcome
Annualized bleeding rate (ABR) levels
Condition
Hemophilia A
Intervention
efanesoctocog alfa (BIVV001)
Study Arms / Comparison Groups
Prophylaxis
Description: Participants will receive weekly dose of BIVV001 for 52 weeks.
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
65
Start Date
February 19, 2021
Completion Date
February 2023
Primary Completion Date
February 2023
Eligibility Criteria
Inclusion criteria :
- Participant must be younger than 12 years of age, at the time of signing the informed
consent
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII as documented either by
central laboratory testing at Screening or in historical medical records from a
clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a
documented genotype known to produce severe hemophilia A.
- Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant
and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs for patients aged
6-11 years and above 50 EDs for patients aged <6 years
- Weight above or equal to 10 kg.
Exclusion criteria:
- History of hypersensitivity or anaphylaxis associated with any FVIII product.
- History of a positive inhibitor (to FVIII) 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 test result, defined as ≥0.6 BU/mL at Screening.
The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.
Gender
Male
Ages
N/A - 12 Years
Accepts Healthy Volunteers
No
Contacts
Clinical Sciences & Operations, 800-633-1610, [email protected]
Location Countries
France
Location Countries
France
Administrative Informations
NCT ID
NCT04759131
Organization ID
EFC16295
Secondary IDs
2020-000769-18
Responsible Party
Sponsor
Study Sponsor
Sanofi
Study Sponsor
Clinical Sciences & Operations, Study Director, Sanofi
Verification Date
February 2021