Observatory of Patients With Haemophilia B Treated by IdElvion®
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- CSL Behring
- 入组人数
- 222
- 试验地点
- 28
- 主要终点
- Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events
研究概览
简要总结
OrPHEe is a non-interventional, prospective and national study which aim is to record real life data in haemophilia B French patients treated with Idelvion® to confirm the efficacy and safety of this product established in clinical development studies.
详细描述
Haemophilia B is a congenital coagulation disorder caused by a deficiency or anomaly of coagulation factor IX (FIX).
The severity of the haemophilia depends on the extent of the FIX deficiency with clinical manifestations differing depending on the location of the bleed.
Treatment of this disease involves the administration of the deficient factor, i.e. FIX, to patients. Depending on the severity of the disease and context, a range of treatment regimens are available (long-term prophylactic treatment for the prevention of non-surgical bleeds, short-term prophylactic treatment for high-risk periods, treatment for the prevention of surgical bleeds or on-demand curative treatment).
CSL Behring has developed a long-acting recombinant FIX, i.e. rIX-FP (Idelvion®), to extend the intervals between the administrations of treatment while also providing a therapeutic benefit. Data from clinical studies clearly confirm the therapeutic benefit of Idelvion® in adult and paediatric patients with haemophilia B previously receiving an on-demand treatment for bleeding episodes, for long-term prophylaxis, as well as in patients undergoing surgery.
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 agreed to participate in the observatory after receiving written information on the purpose of the study and the personal data to be collected (agreement of parents for under-age patients);
- •Present with haemophilia B and are currently being treated or have previously been treated with Idelvion® as a long-term prophylactic treatment, on-demand treatment or short-term treatment for surgical procedures or to cover periods with a high-risk of bleeding.
- •Do not have FIX targeted antibodies at the time of the inclusion visit
排除标准
- •Refusal by the patient or his/her legal representative to participate in the study;
- •Existence of a contraindication to the use of Idelvion® treatment (known hypersensitivity to FIX or hamster proteins);
- •Simultaneous participation in an interventional clinical study on a drug
结局指标
主要结局
Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events
时间窗: Up to 36 months
Incidence of non-surgical bleeding episodes
时间窗: Up to 36 months
Number of spontaneous bleeding events per patient
时间窗: Up to 36 months
Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes
时间窗: Up to 36 months
次要结局
- The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding(up to 36 months)
- The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®(up to 36 months)
- Incidence of surgical bleeding episodes(up to 36 months)
- The number of infusions and total dose of Idelvion®(up to 36 months)
- The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes(up to 36 months)
- Completion of the following questionnaire: EQ-5D-3L for adults(up to 36 months)
- The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion®(up to 36 months)
- Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years(up to 36 months)