Clinical trial NCT00866606: Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects
Official title: An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia B
Keywords: On-demand treatment
Hemostatic efficacy
Factor IX activity
Factor IX recovery
Factor IX inhibitor
Chinese
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer
Sponsor class: INDUSTRY
First posted: 2009-03-20
Enrollment: 35
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.


Eligibility criteria: Inclusion Criteria:

* Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia B (FIX activity: more than 5%, 1-5%, or less than 1%, respectively)
* Subjects with previous exposure to FIX replacement therapy
* If HIV positive, documented CD4 count more than 200/µL within 6 months of study entry

Exclusion Criteria:

* Diagnosed with any bleeding disorder in addition to hemophilia B
* Current FIX inhibitor or history of FIX inhibitor (defined as \>ULN of the reporting laboratory)
* Subject has no history of exposure to FIX products (previously untreated patient \[PUP\])
* Subject is currently utilizing primary FIX prophylaxis
* Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry
* Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation
* Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation
* Subjects with a known hypersensitivity to hamster protein
* Significant hepatic or renal impairment (ALT and AST \>5 x ULN, bilirubin \>2 mg/dL or serum creatinine \>1.25 x ULN)
* Prothrombin Time \>1.5 x ULN
* Platelet count \<80,000/µL
* Pregnant or breastfeeding women
* Unwilling or unable to follow the terms of the protocol
* Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor

Primary outcomes:
measure: Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion
timeFrame: 8 hours post infusion
measure: Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion
timeFrame: 24 hours post infusion
measure: Percentage of Participants With FIX Inhibitor Development
timeFrame: Baseline up to 6 months
Secondary outcomes:
measure: Number of Infusions Required to Treat Each Bleed
timeFrame: Baseline up to 6 months
measure: FIX Incremental Recovery
timeFrame: Baseline (Visit 2) up to 6 months (Visit 4)
measure: Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)
timeFrame: Baseline up to 6 months
measure: Percentage of Participants With Allergic-Type Allergic Reactions
timeFrame: Baseline up to 6 months
measure: Percentage of Participants With Thrombosis
timeFrame: Baseline up to 6 months
measure: Percentage of Participants With Red Blood Cell (RBC) Agglutination
timeFrame: Baseline up to 6 months
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Pfizer Investigational Site
state: Guangzhou
city: Suzhou
country: China
facility: Pfizer Investigational Site
state: Jiangsu
city: Tianjin
country: China
facility: Pfizer Investigational Site
state: Tianjin
city: Hangzhou
country: China
facility: Pfizer Investigational Site
state: Zhejiang
city: Beijing
country: China
facility: Pfizer Investigational Site
city: Shanghai
country: China
facility: Pfizer Investigational Site
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Yang R, Zhao Y, Wang X, Sun J, Jin J, Wu D, Charnigo R, O'Brien A, Zhong Z, Rendo P. Evaluation of the safety and efficacy of recombinant factor IX (nonacog alfa) in minimally treated and previously treated Chinese patients with haemophilia B. Haemophilia. 2012 Sep;18(5):e374-8. doi: 10.1111/j.1365-2516.2012.02907.x. Epub 2012 Jul 9. No abstract available.