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

  • STATUS
    Recruiting
  • End date
    Feb 24, 2023
  • participants needed
    65
  • sponsor
    Bioverativ, a Sanofi company
Updated on 24 July 2021
Investigator
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Summary

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

Description

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.

Details
Condition Hemophilia A, Hemophilia, hema
Treatment efanesoctocog alfa (BIVV001)
Clinical Study IdentifierNCT04759131
SponsorBioverativ, a Sanofi company
Last Modified on24 July 2021

Eligibility

Yes No Not Sure

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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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