An Open-label, Multi-centre, Un-controlled Trial to Assess Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Patients With Haemophilia B
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Haemostatic Effect During Surgery Evaluated by the Four-point Response Scale (Excellent, Good, Moderate, Poor)
研究概览
简要总结
This trial is conducted in Africa, Asia, Europe and the United States of America (USA).
The aim of the trial is to evaluate the safety and efficacy of NNC-0156-0000-0009 (nonacog beta pegol) during surgical procedures in 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
- 13 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patients with haemophilia B with a FIX activity below or equal to 2%
- •Male patients with moderately severe or severe congenital haemophilia B with a FIX activity below or equal to 2% according to medical records
- •History of at least 150 exposure days to other FIX products
- •Scheduled major surgery
排除标准
- •Known history of FIX (coagulation factor nine) inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
- •Current FIX (coagulation factor nine) inhibitors above or equal to 0.6 Bethesda Units (central laboratory)
- •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)
- •ALT (alanine aminotransferase) 3 times the upper limit of normal reference ranges at screening (central laboratory)
- •Immune modulating or chemotherapeutic medication
研究组 & 干预措施
Patients enrolled in trial
New patients will be included as well as transferred patients from Paradigm™ 2 or Paradigm™ 4 trial
干预措施: nonacog beta pegol (Drug)
结局指标
主要结局
Haemostatic Effect During Surgery Evaluated by the Four-point Response Scale (Excellent, Good, Moderate, Poor)
时间窗: At the day of surgery
Haemostatic effect during surgery was evaluated immediately after surgery (last stitch) using a fourpoint response scale: - Four-point response scale: Excellent, good, moderate, poor. The evaluation was done by the surgeon, anaesthesiologist and/or investigator based on experience as follows: 1. Excellent: Better than expected/predicted in this type of procedure. 2. Good: As expected in this type of procedure. 3. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. 4. Poor: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
次要结局
- Incidence of Adverse Events (AEs)(during the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) until 4 weeks after post-operative period (day 1 to day 13))
- Incidence of Serious Adverse Events (SAE)(During the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) until 4 weeks after post-operative period (day 1 to day 13))
- Haemoglobin Pre- and Post-surgery Start(0, 1 hour, 24 hours.)
- Consumption of NNC-0156-0000-0009 (U/kg Body Weight)(During surgery (the time from knife to skin until last stitch) and post-operative period (day 1 to day 13))
- Transfusion Requirements (Fulfilling Transfusion Criteria)(during surgery (the time from knife to skin until last stitch) and post-operative period (day 1 to day 13))
- Incidence of Inhibitors Against FIX (Coagulation Factor Nine)(During the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) and every 4 weeks after post-operative period (day 1 to day 13))