Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (XTEND-Kids)

Brief description of study

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

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.

Clinical Study Identifier: NCT04759131

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hemophilia A
  • Age: - 12 Years
  • 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 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.

Last updated on 12 May 2022