Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 71
- 试验地点
- 1
- 主要终点
- Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units)
研究概览
简要总结
This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of nonacog beta pegol (NNC-0156-0000-0009) after long-term exposure in patients with haemophilia B.
This trial is an extension to trials NN7999-3747 (NCT01333111/paradigm™ 2) and NN7999-3773 (NCT01386528/paradigm™ 3).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 13 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Previous participation in NN7999-3747 (NCT01333111) and/or NN7999-3773
排除标准
- •Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
- •Current FIX inhibitors above or equal to 0.6 BU (Bethesda Units)
- •Congenital or acquired coagulation disorders other than haemophilia B
- •Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
- •Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's (trial physician) judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome
研究组 & 干预措施
Prophylaxis, high dose (once weekly)
干预措施: nonacog beta pegol (Drug)
Prophylaxis, low dose (once weekly)
干预措施: nonacog beta pegol (Drug)
On-demand
干预措施: nonacog beta pegol (Drug)
Prophylaxis, high dose (every second week)
干预措施: nonacog beta pegol (Drug)
结局指标
主要结局
Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units)
时间窗: From Day 1 up to 2 years
The primary endpoint was incidence of inhibitors against coagulation factor nine (FIX) defined as titre ≥0.6 Bethesda unit (BU). Number of subjects who developed inhibitors against FIX are reported.
次要结局
- Number of Bleeding Episodes During Routine Prophylaxis(From Day 1 up to 2 years)
- FIX Trough Levels(From Day 1 up to 2 years)
- Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate, Poor)(From Day 1 up to 2 years)
- Incidence of Adverse Events (AEs)(From Day 1 up to 2 years)
- Incidence of Serious Adverse Events (SAEs)(From Day 1 up to 2 years)