Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

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ClinicalTrials.gov Identifier: NCT00037557

Recruitment Status : Completed

First Posted : May 20, 2002

Last Update Posted : December 27, 2007

Sponsor:

Information provided by:

Wyeth is now a wholly owned subsidiary of Pfizer

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Hemophilia B	Drug: BeneFIX	Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Enrollment :	20 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
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
Study Start Date :	September 2002
Actual Study Completion Date :	November 2007

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Hemophilia

Genetic and Rare Diseases Information Center resources: Hemophilia Hemophilia B Hemophilia A

U.S. FDA Resources

Arms and Interventions

Outcome Measures

Primary Outcome Measures :

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 :

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

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	up to 5 Years (Child)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00037557

Locations

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United States, Colorado

Aurora, Colorado, United States, 80262
United States, Michigan

Detroit, Michigan, United States, 48201
United States, New Jersey

New Brunswick, New Jersey, United States, 08903
United States, North Carolina

Chapel Hill, North Carolina, United States, 27599-7220
United States, Ohio

Dayton, Ohio, United States, 45404
United States, Texas

Houston, Texas, United States, 77030

Sponsors and Collaborators

Wyeth is now a wholly owned subsidiary of Pfizer

Investigators

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Study Director:	Medical Monitor, MD	Wyeth is now a wholly owned subsidiary of Pfizer

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Rendo P, Smith L, Lee HY, Shafer F. Nonacog alfa: an analysis of safety data from six prospective clinical studies in different patient populations with haemophilia B treated with different therapeutic modalities. Blood Coagul Fibrinolysis. 2015 Dec;26(8):912-8. doi: 10.1097/MBC.0000000000000359.

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ClinicalTrials.gov Identifier:	NCT00037557
Other Study ID Numbers:	3090A1-301
First Posted:	May 20, 2002 Key Record Dates
Last Update Posted:	December 27, 2007
Last Verified:	December 2007

Keywords provided by Wyeth is now a wholly owned subsidiary of Pfizer:


Hemophilia B

Additional relevant MeSH terms:

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Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases	Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked

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