An Investigator-initiated Study on rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors - European Initiative to Prevent Joint Damage in Hemophilia A Children With Inhibitors
试验速览
- 阶段
- 2 期
- 入组人数
- 50
- 试验地点
- 15
- 主要终点
- Total number of joint bleeds.
研究概览
简要总结
The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors.
详细描述
This is a multicentre, randomised, controlled study designed to gain evidence of the advantage of the prophylactic, daily treatment with recombinant activated FVII as compared to the conventional on demand therapy in reducing the bleeding frequency and preserving the orthopaedic status in hemophilic children with inhibitors.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 8 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patients with hemophilia A who have been treated with factor VIII on demand or on prophylaxis and who have developed inhibitors to factor VIII
- •≤ 2 years from the time of first inhibitor detection.
- •High-responding inhibitors (historical peak > 5 BU/mL)and known anamnestic response in case of negative inhibitor titre.
- •Candidates to start daily ITI with FVIII doses ranging from 50 IU/Kg/day to 200 IU/Kg/day
- •Maximal two bleedings in the same joint within the last 6 months before entering the study or maximal six joint bleeds in the same joint within 2 years
- •Adequate venous access for daily infusion and capable (caregiver) of reconstituting and injecting the study drug
- •Informed consent by parents or legal guardians.
排除标准
- •ITI already started
- •Known or suspected hypersensitivity to the active substance or to any of the excipients of the study drug
- •Administration of any investigational product within 30 days prior to randomisation
- •Other coagulation disorders than congenital hemophilia A.
- •Family history of thrombosis at an early age (< 40 years), known thrombophilia, any previous thrombosis including catheter-related deep vein thrombosis, previous neonatal thrombosis.
- •Known pseudo tumours
- •Known severe liver disease
- •Platelet count < 50,000 platelets/µL at screening
- •Surgery within one month or planned major and/or orthopaedic surgery.
研究组 & 干预措施
prophylaxis
prophylaxis with recombinant activated FVII 90 µg/kg/day i.v.
干预措施: recombinant activated factor VII (Drug)
on demand treatment
treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution
干预措施: recombinant activated factor VII (Drug)
结局指标
主要结局
Total number of joint bleeds.
时间窗: 18 months
次要结局
- Number of adverse events and serious adverse events.(18 months)
- Joint status evaluated by the Hemophilia Joint Health Score(18 months)
研究者
Elena Santagostino
MD
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico