This study is currently not recruiting participants.

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

Investigating Safety and Efficacy of Investigational Medication in Pediatrics

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.

This study investigates hemophilia A. It focuses on evaluating the safety of an investigational medication in children who have previously been treated for this condition.

The study evaluates the effectiveness of the investigational medication in preventing and treating bleeding episodes, as well as its impact on joint health and quality of life. It also looks at how the body processes the medication, known as pharmacokinetics.

  • Who can participate: Children under 12 years old with severe hemophilia A can participate. They must have a history of treatment with certain medications and weigh at least 10 kg.
  • Study details: Participants will receive an investigational medication. Some participants may receive a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine.
  • Study timelines: The study will last 60 weeks.
Updated on 11 Sep 2025. Study ID: NCT04759131