Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

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.

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Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Aurora, Colorado, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

BENEFIX

Drug

Primary outcomes

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 outcomes

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

Sponsors and collaborators

Lead sponsor

Wyeth is now a wholly owned subsidiary of Pfizer

Industry

Registry information

Official study 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

Important dates

Study start: 2002
Study completion: 2007
First posted: May 20, 2002
Registry last updated: Dec 27, 2007

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed