Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

This study has been completed.
Sponsor:
Information provided by:
Wyeth is now a wholly owned subsidiary of Pfizer
ClinicalTrials.gov Identifier:
NCT00037557
First received: May 17, 2002
Last updated: December 18, 2007
Last verified: December 2007
  Purpose

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 Intervention Phase
Hemophilia B
Drug: BeneFIX
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Wyeth is now a wholly owned subsidiary of Pfizer:

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.

Estimated Enrollment: 20
Study Start Date: September 2002
Study Completion Date: November 2007
  Eligibility

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00037557

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

No publications provided

ClinicalTrials.gov Identifier: NCT00037557     History of Changes
Other Study ID Numbers: 3090A1-301
Study First Received: May 17, 2002
Last Updated: December 18, 2007
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on October 20, 2014