Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Phase 3CompletedNCT00866606

Wyeth is now a wholly owned subsidiary of Pfizer35 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia B

Interventions

BenefixBIOLOGICAL

BeneFIX for on-demand treatment of bleeding episodes were according to investigator prescription. FIX recovery was assessed by determining the FIX activity (FIX:C) levels in individual subjects. BeneFIX dosage for recovery assessments: single 75 IU/kg (±5 IU/kg) IV bolus infusion.

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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

Locations (6)

Pfizer Investigational Site

Guangzhou, Guangzhou, China

Pfizer Investigational Site

Suzhou, Jiangsu, China

Pfizer Investigational Site

Tianjin, Tianjin Municipality, China

Pfizer Investigational Site

Hangzhou, Zhejiang, China

Outcomes

Primary Outcomes

Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion

Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.

Time frame: 8 hours post infusion

Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion

Time frame: 24 hours post infusion

Percentage of Participants With FIX Inhibitor Development

Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result \>=0.6 Bethesda Unit (BU). Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX infusion, and \<= 100 documented Exposure Days (EDs); while Previously Treated Patients (PTPs): those who had received \>100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.