Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

Phase 3

Completed

• Conditions: Hemophilia B

• Registration Number: NCT00037557
• Lead Sponsor: Wyeth is now a wholly owned subsidiary of Pfizer

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2002-05-17
• Study First Submitted QC: 2002-05-17
• Study First Posted: 2002-05-20
• Study Start: 2002-09
• Study Completion: 2007-11
• Status Verified: 2007-12
• Last Update Submitted: 2007-12-18
• Last Update Posted: 2007-12-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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 Measures

Name	Time	Method
To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.