An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 35
- 试验地点
- 6
- 主要终点
- Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion
研究概览
简要总结
This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion
时间窗: 24 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.
Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion
时间窗: 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.
Percentage of Participants With FIX Inhibitor Development
时间窗: Baseline up to 6 months
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.
次要结局
- FIX Incremental Recovery(Baseline (Visit 2) up to 6 months (Visit 4))
- Percentage of Participants With Thrombosis(Baseline up to 6 months)
- Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)(Baseline up to 6 months)
- Percentage of Participants With Allergic-Type Allergic Reactions(Baseline up to 6 months)
- Number of Infusions Required to Treat Each Bleed(Baseline up to 6 months)
- Percentage of Participants With Red Blood Cell (RBC) Agglutination(Baseline up to 6 months)