Status:
COMPLETED
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Lead Sponsor:
Bioverativ, a Sanofi company
Conditions:
Hemophilia A
Eligibility:
MALE
Up to 11 years
Phase:
PHASE3
Brief Summary
Primary Objective: \- To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis ...
Detailed Description
Study duration per participants was approximately 60 weeks (maximum 8 weeks for screening and 52 weeks of treatment). All participants completing or remaining at the end of study were offered partici...
Eligibility Criteria
Inclusion
- 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 international units per deciliter (IU/dL) (\<1 percent \[%\]) endogenous Factor VIII (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 exposure days (EDs) for participants aged 6 to \<12 years and above 50 EDs for participants 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 greater than or equal to (\>=) 0.6 Bethesda units (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 would not exclude the participant.
- Positive inhibitor test result, defined as \>=0.6 BU/mL at Screening.
- The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Exclusion
Key Trial Info
Start Date :
February 19 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 18 2023
Estimated Enrollment :
74 Patients enrolled
Trial Details
Trial ID
NCT04759131
Start Date
February 19 2021
End Date
January 18 2023
Last Update
September 11 2025
Active Locations (40)
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1
Children's Hospital Los Angeles-Site Number:8400006
Los Angeles, California, United States, 90027
2
University of Florida-Site Number:8400009
Gainesville, Florida, United States, 32610
3
Children's Healthcare of Atlanta-Site Number:8400019
Atlanta, Georgia, United States, 30322
4
Rush University Medical Center-Site Number:8400001
Chicago, Illinois, United States, 60612-3833