Hemophilia B Clinical Trial
Official title:
An Open-Label, Single-Arm, Safety and Efficacy Study of Recombinant Human Factor IX (rFIX; BeneFIX®) in Children Less Than 6 Years of Age With Severe Hemophilia B
To characterize the safety and efficacy of rFIX in children less than 6 years of age with
severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery.
This study will provide an opportunity for systematic observation of treatment with rFIX in
children less than 6 years of age regardless of prior FIX treatment. Younger patients
exhibit a different pharmacokinetic profile and therefore may respond differently to rFIX
infusions when compared with older children and adults. This evaluation will provide data
from which recommendations can be made regarding rFIX dosing and treatment of these
patients. Surveillance for certain observations that have been made in patients treated with
rFIX in the clinical and postmarketing setting will be performed, including inhibitor
development, thrombogenicity, FIX recovery/lack of effect, allergic-type manifestations, and
RBC agglutination. Comparisons will be derived from published reports and communications
describing experience with other FIX products and protein therapeutics in general.
n/a
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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