Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

Wyeth

Status and phase: Completed; Phase 3

Conditions: Hemophilia B

Treatments: Drug: BeneFIX

Study type: Interventional

Funder types: Industry

Identifiers

NCT00037557

3090A1-301

Details and patient eligibility

About

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.

Sex: All

Ages: Under 5 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)