Reformulated BeneFIX Efficacy and Safety After Conversion From a pdFIX
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 1
- 试验地点
- 16
- 主要终点
- Number of Participants Reporting Efficacy
研究概览
简要总结
The purpose of this study is to collect data around the period of the conversion from plasma-derived Factor IX (pdFIX) to BeneFIX. The main information collected will be: a retrospective history of the bleedings in the 3-month period before the conversion, the recovery with pdFIX just before the conversion and with BeneFIX just after the conversion, and a prospective history of the bleedings in the 3 month period following the conversion.
详细描述
The switch to BeneFIX has already been decided by the investigator. Patients will be followed up to 3 months after the switch.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Moderately to severe haemophilia B patient (FIX activity < or equal to 2%) for whom the switch from pdFIX to BeneFIX has already been decided by the investigator
- •Previously treated patients (PTP) with > or equal to 150 ED to any FIX product
- •Male patients, aged > or equal to 12 years
- •Absolute CD4 count > or equal to 300/microL
- •Normal platelet count (> or equal to 100 000/microL)
- •Patient is in a non-bleeding state and has not received any coagulation FIX within five (5) days of recovery
- •Written informed consent obtained prior to study entry (for patients aged < 18 years, parents' signature or subject legally acceptable representative obtained prior to study entry)
排除标准
- •Any other known bleeding disorder in addition to haemophilia B
- •History of, or current detectable factor IX inhibitor (> or equal to 0.6 BU by Bethesda inhibitor assay)
- •History of anaphylaxis to any coagulation factor IX
- •Patient with a known hypersensitivity to hamster protein
- •Patient with a hypersensitivity to the active substance or to any of the excipients
- •Patient unable to be off FIX replacement therapy for at least 5 days without bleeding Patient with hepatic or renal impairment (ALT [SGPT] and AST [SGOT] > 5 x Upper Limit Normal (ULN), total bilirubin > 20mg/l, albumin < 25 g/l, prothrombin time > 1.25 x ULN, serum creatinine > 1.25 x ULN)
- •Treatment with any investigational drug or device within the past 30 days
- •Any condition that, in the Investigator's judgment, makes participation in the study not advisable
结局指标
主要结局
Number of Participants Reporting Efficacy
时间窗: 4 months
Clinical efficacy was measured by number/location of bleeding episodes, number of injections per bleeding, factor IX consumption, global assessment of efficacy by investigator and patient; biological efficacy (recovery) with BeneFIX was measured just after conversion.
次要结局
未报告次要终点