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1 Study Matches

An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B.

A study to confirm the appropriate dose of fitusiran when given to severe Hemophilia A or B patients with inhibitors between the ages of 1 and <12 years old.
Younger than 18 years old
This study is NOT accepting healthy volunteers
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Inclusion Criteria:
Severe hemophilia A or B (FVIII <1% or FIX ≤2%)
Inhibitory antibodies to FVIII or FIX
Age 1 to <12 years
Exclusion Criteria:
AT activity <60% at screening
Co-existing thrombophilic disorder
Central or peripheral indwelling catheter, with a hx of venous access complications
Anticipated need of surgery during the study
Presence of clinically significant liver disease
Blood Disorders
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Study Locations

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Hershey, PA