Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

Study on the Safety and Efficacy of Investigational Medication in Children with Hemophilia A

Not Recruiting
11 years or below
Male
Phase 3
74 participants needed

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
  • Age: 11 years or below
  • 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.

Updated on 13 Feb 2024. Study ID: NCT04759131

This study investigates the safety and efficacy of an investigational medication in children with hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to low levels of a protein called Factor VIII. This study aims to see how safe the investigational medication is for children who have been treated for hemophilia A before.

Participants will receive the investigational medication as a regular treatment to prevent bleeding and during bleeding episodes to see how well it works. The study will also look at how the medication affects joint health and quality of life. Additionally, the study will measure how the body processes the investigational medication, known as pharmacokinetics.

  • Who can participate: Children under 12 years old with severe hemophilia A can participate. They must have been treated for hemophilia A before and weigh at least 10 kg. Children with a history of hypersensitivity to Factor VIII products cannot participate.
  • Study details: Participants will receive the investigational medication to prevent and treat bleeding episodes. The study will assess how the medication affects joint health and quality of life.
  • Study timelines: The study will last 60 weeks.