A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 60
- 试验地点
- 58
- 主要终点
- Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events)
研究概览
简要总结
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B. While taking part in this study, participants will receive the same treatment as given to them by their study doctor. All visits at the clinic are done in the same way as the participants are used to. During visits at the clinic, participants might be asked for some relevant tests if considered useful by their study doctor. During the visits, the participants study doctor might ask if the participants had any side effects since their last study visit. The participants will be asked to note down the number of bleeds and the treatment of their bleeds as well as their regular prophylaxis. During the visits to the clinic, the participants will be asked to answer some questionnaires about their quality of life and their ability to be physically active. The participant's participation in the study will last for 4-9 years, depending on when they join the study. Participants are free to leave the study at any time and for any reason. This will not affect their current and future medical care.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
- •Male patients at any age with haemophilia B assigned to N9-GP prophylaxis treatment
- •Decision to initiate treatment with commercially available N9-GP has been made by the patient(s)/Legally Authorised Representative(s) (LAR(s)) and the treating physician before and independently from the decision to include the patient in this study
排除标准
- •Previous participation in this study. Participation is defined as signed informed consent
- •Known or suspected hypersensitivity to N9-GP or related products
- •Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation
- •Clinical suspicion or presence of FIX inhibitor at time of inclusion.
研究组 & 干预措施
Patients with haemophilia B
Patients with haemophilia B without current inhibitors
干预措施: Nonacog beta pegol (Drug)
结局指标
主要结局
Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events)
时间窗: From start of study period (week 0) to end of study period (up to 9 years)
Count of events
次要结局
- Number of bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)(From start of study period (week 0) to up to 9 years)
- Haemostatic effect of N9-GP when used for treatment of bleeding episodes(From start of study period (week 0) to up to 9 years)
- Number of treatment requiring bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)(From start of study period (week 0) to up to 9 years)
- Number of Serious Adverse Events (SAEs)(From start of study period (week 0) to up to 9 years)
- Haemostatic response of N9-GP when used in perioperative management(From start of study period (week 0) to end of study period (up to 9 years))