Post-marketing Surveillance (Use Result Surveillance) With Refixia®. A Multicentre, Non-interventional Post Marketing Surveillance of Safety and Effectiveness of Refixia® in Routine Clinical Care With Haemophilia B Patients in Japan.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 28
- 试验地点
- 46
- 主要终点
- Number of Adverse Reactions (ARs)
研究概览
简要总结
The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B. The participants will get Refixia® as prescribed to them by their study doctor. The study will last up to Sep 2025 for the participant. The participants may be asked to fill in the quality of life questionnaires (if they are above age of 15). The blood samples taken from the participants as part of routine clinical practice will also be used to investigate the safety for the long-term use of Refixia®.
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
- All
- 接受健康志愿者
- 否
入选标准
- •Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
- •The decision to initiate treatment with commercially available Refixia® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. At each site of this study, all patients will be registered consecutively from the first patient after the launch of Refixia® (consecutively registered system).
- •Diagnosis of haemophilia B in males or females, no age limitation. Patients younger than 12 years old will continue to be registered for 3 years of recruitment period regardless of the target number of patients.
- •New patients who have not been previously exposed to Refixia®. Also patients previously exposed to Refixia® in NN7999-3639, -3747, -3774, -3775 or -3895 clinical trial can be enrolled in this study. The patients who have participated in NN7999 -3774 or -3895 clinical trial can be enrolled in this study as continuous cases until 30-September-2024 (one year before planned end of study date).
排除标准
- •Previous participation in this study. Participation is defined as having given informed consent in this study
- •Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation
- •Known or suspected hypersensitivity to study product or related products.
研究组 & 干预措施
Patients with haemophilia B
Both patients who have not previously been exposed to Refixia® and patients previously exposed to Refixia® in one of the clinical trials can be included.
干预措施: Refixia® (Drug)
结局指标
主要结局
Number of Adverse Reactions (ARs)
时间窗: From baseline (week 0) to end of study (up to 6 years and 10 months)
Count of events
次要结局
- Number of Serious Adverse Events (SAEs)(From baseline (week 0) to end of study (up to 6 years and 10 months))
- Number of Serious Adverse Reactions (SARs)(From baseline (week 0) to end of study (up to 6 years and 10 months))
- Number of treatment requiring bleeding episodes as assessed by ABR(From baseline (week 0) to end of study (up to 6 years and 10 months))
- Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures(From baseline (week 0) to end of study (up to 6 years and 10 months))
- Number of bleeding episodes as assessed by annualised bleeding rate (ABR)(From baseline (week 0) to end of study (up to 6 years and 10 months))
- Haemostatic response of Refixia® in treatment of bleeds(From baseline (week 0) to end of study (up to 6 years and 10 months))