Overview
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
Eligibility
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.