Observational Register of Patients With haEmophilia A tReated With Afstyla®
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 62
- 试验地点
- 17
- 主要终点
- The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.
研究概览
简要总结
Record real life data of patients with Hemophilia A and treated with Afstyla® to assess the effectiveness and the safety of the treatment used as prophylaxis, prevention of bleeding (e.g. surgery) or on-demand treatment during 3 years after patient inclusion
详细描述
Haemophilia A is a congenital coagulation disorder caused by a deficiency or abnormality of coagulation factor VIII (FVIII).
The severity of the haemophilia depends on the magnitude of the FVIII deficiency and the clinical features depend on the site of the bleed.
The therapeutic management of this disease involves administering the deficient factor, FVIII to the patient. Depending on the severity of the disease and patient background, the management regimens are different (long term prophylactic treatment to prevent non surgical bleeds, prophylactic short term treatment to prevent surgical bleeds or treatment of acute bleeds on demand).
Several recombinant or plasma derived treatments are currently available. The CSL Behring Company has designed a new recombinant single chain B1FVIII, rFVIII (Afstyla®). This has increased affinity for VWF, resulting in improved stability and an improvement in its pharmacokinetic indices. OPERA is a non-interventional, prospective and national study which aim is to record real life data in haemophilia A French patients treated with Afstyla® in order to confirm the efficacy and safety of this proprietary product established in clinical development studies.
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
- •Have given their agreement to take part in the observational registry after being informed in writing of the purposes of the study and after their data have been recorded (parent's agreement for minor patients);
- •Be suffering from haemophilia A and being treated or having been treated with Afstyla® long term prophylactically, on demand or preventatively for a surgical procedure;
- •Absence of inhibitor and/or treatment for immune tolerance at the time of inclusion
排除标准
- •Refusal of the patient or the patient's legal representative to take part in the study;
- •Existence of a contraindication to the use of Afstyla® treatment (known hypersensitivity to FVIII or hamster proteins);
- •Simultaneous participation in an interventional clinical study.
- •Presence of an inhibitor and/or ongoing immune tolerance.
结局指标
主要结局
The annualized bleeding rate (spontaneous, traumatic) during long term prophylactic and on demand regimen.
时间窗: Up to 36 months
The number of spontaneous bleeding episodes per patient
时间窗: Up to 36 months
The number of infusions of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes
时间窗: Up to 36 months
The total dose of Afstyla® required for the prevention and resolution of non-surgical bleeding episodes
时间窗: Up to 36 months
次要结局
- The number of infusions of Afstyla® required during the surgical procedures(Up to 36 months)
- The total dose of Afstyla® required during the surgical procedures(Up to 36 months)
- The incidence of adverse events (AE), severe AE, and AE related to Afstyla®(Up to 36 months)