Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

  • STATUS
    Recruiting
  • participants needed
    12
  • sponsor
    Genzyme, a Sanofi Company
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Updated on 4 December 2020
See if I qualify
hemophilia
severe haemophilia a
antihemophilic factor
factor ix
bethesda assay
fitusiran
haemophilia a

Summary

Primary Objective:

  • To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B

Secondary Objective:

  • To characterize the safety and tolerability
  • To characterize the pharmacokinetics (PK)

Description

Study duration per participant is approximately 160 weeks, including a 12-week fitusiran efficacy period

Details
Condition Hemophilia A, hemophilia, hemophilia
Age 1years - 11years
Treatment Fitusiran
Clinical Study IdentifierNCT03974113
SponsorGenzyme, a Sanofi Company
Last Modified on4 December 2020

Eligibility

 Yes No Not Sure

Inclusion Criteria

Male, aged 1 to <12 years at the time of enrollment
Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) 2%)
Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria
Inhibitor titer of 0.6 BU/mL at screening, OR
Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers 0.6 BU/mL, OR
Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of anamnestic response
Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
Weight requirements at the time of enrollment: 8 to <45 kg
Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements

Exclusion Criteria

Known co-existing bleeding disorders other than hemophilia A or B
Antithrombin (AT) activity <60% at Screening
Co-existing thrombophilic disorder
Clinically significant liver disease
Active Hepatitis C virus infection
Acute or chronic Hepatitis B virus infection
Acute Hepatitis A or hepatitis E infection
HIV positive with a CD4 count of <400 cells/L
History of arterial or venous thromboembolism, unrelated to an indwelling venous access
Inadequate renal function
History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy
History of intolerance to subcutaneous (SC) injection(s)
Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment
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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