Clinical trial NCT00037557: Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia B
Keywords: Hemophilia B
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer
Sponsor class: INDUSTRY
First posted: 2002-05-20
Enrollment: 20
Sex: ALL
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: 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:
measure: To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.
Other outcomes:
Locations:
city: Aurora
country: United States
state: Colorado
city: Detroit
country: United States
state: Michigan
city: New Brunswick
country: United States
state: New Jersey
city: Chapel Hill
country: United States
state: North Carolina
city: Dayton
country: United States
state: Ohio
city: Houston
country: United States
state: Texas
Study officials:
affiliation: Wyeth is now a wholly owned subsidiary of Pfizer
name: Medical Monitor, MD
role: STUDY_DIRECTOR
References:
citation: 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.