Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00037557

Enrollment

Registered

2002-05-20

Start date

2002-09-30

Completion date

2007-11-30

Last updated

2007-12-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia B

Keywords

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.

Interventions

DRUGBeneFIX

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 5 Years

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

Design outcomes

Primary

Measure	Time frame
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

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026