Brief Title
Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
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
Brief Summary
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 Phase
Phase 3
Study Type
Interventional
Primary Outcome
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.
Secondary Outcome
To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.
Condition
Hemophilia B
Intervention
BeneFIX
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
20
Start Date
September 2002
Completion Date
November 2007
Eligibility Criteria
Inclusion Criteria: - Severe hemophilia B - Less than 5 years of age - In the investigator's judgment, the patient and/or caregiver will be compliant to study procedures Exclusion Criteria: - A currently detectable FIX inhibitor. A family history of inhibitors will not exclude the patient. - Impaired liver function - Impaired renal function
Gender
All
Ages
N/A - 5 Years
Accepts Healthy Volunteers
No
Contacts
Medical Monitor, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00037557
Organization ID
3090A1-301
Study Sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Study Sponsor
Medical Monitor, MD, Study Director, Wyeth is now a wholly owned subsidiary of Pfizer
Verification Date
December 2007