Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00037557
• Other study ID #: 3090A1-301
• Status: Completed
• Phase: Phase 3
• First received: May 17, 2002
• Last updated: December 18, 2007
• Start date: September 2002
• Est. completion date: November 2007

Study information

• Verified date: December 2007
• Source: Wyeth is now a wholly owned subsidiary of Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: November 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 5 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Drug:
• BeneFIX

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Wyeth is now a wholly owned subsidiary of Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.