24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A (A-SURE)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 361
- 试验地点
- 4
- 主要终点
- Annualised factor consumption (International Unit [IU])
研究概览
简要总结
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.
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
- •Male patients with a diagnosis of haemophilia A
- •Received prophylactic treatment with a factor product for management of haemophilia A in the 12 months prior to enrolment
- •At enrolment prescribed prophylactic treatment with Elocta or conventional factor product regardless of participation in the study
- •Having at least 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and bleeding episodes prior to the baseline visit
- •Signed and dated informed consent provided by the patient or the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
排除标准
- •Enrolment in a concurrent clinical interventional study involving intake of an investigational medicinal product (IMP), within 1 year prior to enrolment
- •Previously treated with commercially available extended half-life products other than Elocta
- •Presence of factor VIII inhibitors (≥0.60 Bethesda Unit [BU]/mL ) at the latest available inhibitor test using the Nijmegen modified Bethesda assay
研究组 & 干预措施
Elocta
Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice
干预措施: efmoroctocog alfa (Drug)
Conventional factor VIII product
Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice
干预措施: Factor VIII (Drug)
结局指标
主要结局
Annualised factor consumption (International Unit [IU])
时间窗: 24 months
Annualised bleeding rate (ABR)
时间窗: 24 months
Annualised injection frequency
时间窗: 24 months
次要结局
未报告次要终点