Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Study on the Safety and Efficacy of an Investigational Treatment in Pediatric Patients with Hemophilia A
Study Overview
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 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.
Study Details
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 participation in the planned extension trial.
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
- Conditions: Hemophilia A
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Age: 11 years or below
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Gender: Male
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.
This study investigates the safety and efficacy of an investigational treatment in children with Hemophilia A. The purpose of this study is to evaluate how well the investigational treatment works as a preventive measure and during bleeding episodes. The study also aims to assess the treatment's impact on joint health and quality of life.
Participants in the study will receive the investigational treatment and be monitored for various outcomes. The procedures include regular assessments of joint health and quality of life, as well as monitoring the treatment's effectiveness in preventing and managing bleeding episodes. Pharmacokinetics, which is the study of how the drug moves through the body, will also be evaluated.
- Who can participate: Children younger than 12 years with severe Hemophilia A can participate. They must have a history of previous treatment with Factor VIII products for a specified number of days. Participants must weigh at least 10 kg.
- Study details: Participants will receive the investigational treatment and undergo regular assessments. The study will look at how the treatment affects joint health and quality of life.
- Study timelines: The study will last approximately 60 weeks.
