Study Evaluating rFIX; BeneFIX in 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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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.
- To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.
|Study Start Date:||September 2002|
|Study Completion Date:||November 2007|
|United States, Colorado|
|Aurora, Colorado, United States, 80262|
|United States, Michigan|
|Detroit, Michigan, United States, 48201|
|United States, New Jersey|
|New Brunswick, New Jersey, United States, 08903|
|United States, North Carolina|
|Chapel Hill, North Carolina, United States, 27599-7220|
|United States, Ohio|
|Dayton, Ohio, United States, 45404|
|United States, Texas|
|Houston, Texas, United States, 77030|
|Study Director:||Medical Monitor, MD||Wyeth is now a wholly owned subsidiary of Pfizer|