A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period
研究概览
简要总结
This trial is conducted in Europe, Japan and the United States of America (USA).
The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with haemophilia B (baseline level of Factor IX less than or equal to 2%)
- •History of at least 150 exposure days to any Factor IX products
- •Body Mass Index (BMI) below 30.0 kg/m2 (inclusive)
排除标准
- •History of Factor IX inhibitors
- •Platelet count less than 50,000 platelets/microlitre (assessed by laboratory)
- •Kidney or liver dysfunction
- •Scheduled surgery requiring Factor IX replacement therapy, during the trial period
研究组 & 干预措施
25U/kg
干预措施: nonacog beta pegol (Drug)
50U/kg
干预措施: nonacog beta pegol (Drug)
100U/kg
干预措施: nonacog beta pegol (Drug)
结局指标
主要结局
Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period
时间窗: assessed up to five weeks after trial product administration
Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda)
时间窗: assessed up to five weeks after trial product administration
次要结局
- AUC, CL, T½, Incremental recovery (first sample) from 0 to 48 hours after trial product administration(assessed up to five weeks after trial product administration)
- AUC, CL, T½, Incremental recovery (first sample) from 0 to 168 hours after trial product administration(assessed up to five weeks after trial product administration)