A Phase II/III, Randomized, Cross-over, Open-label Trial to Demonstrate Superiority of Prophylaxis Over On-demand Therapy in Previously Treated Subjects With Severe Hemophilia A Treated With Plasma Protein-free Recombinant FVIII Formulated With Sucrose (BAY 81-8973)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 80
- 试验地点
- 47
- 主要终点
- Annualized Number of All Bleeds
研究概览
简要总结
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male, aged 12 to 65 years
- •Severe hemophilia A
- •History of more than 150 exposure days (ED) with clotting factor concentrates
- •Currently receiving episodic treatment with FVIII; no regular prophylaxis for more than 6 consecutive months in the past 5 years
- •No current Factor VIII inhibitor or history of inhibitor
- •Willing to use electronic patient diary
排除标准
- •Presence of another bleeding disease that is different from hemophilia A
- •Thrombocytopenia
- •Abnormal renal function
- •Presence of active liver disease
- •Known hypersensitivity to FVIII
研究组 & 干预措施
Arm 1: rFVIII on demand first CS/EP then CS/ADJ
Participants received on-demand treatment with recombinant factor VIII (rFVIII, BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months, followed by cross-over to study drug assayed by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months.
干预措施: rFVIII (BAY81-8973) on demand (Biological)
Arm 2: rFVIII on demand first CS/ADJ then CS/EP
Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/ADJ for 6 months, followed by cross-over to study drug assayed by CS/EP for 6 months.
干预措施: rFVIII (BAY81-8973) on demand (Biological)
Arm 3: rFVIII prophylaxis low-dose first CS/EP then CS/ADJ
Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII(BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.
干预措施: rFVIII (BAY81-8973) prophylaxis low-dose (Biological)
Arm 4: rFVIII prophylaxis low-dose first CS/ADJ then CS/EP
Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.
干预措施: rFVIII (BAY81-8973) prophylaxis low-dose (Biological)
Arm 5: rFVIII prophylaxis high-dose first CS/EP then CS/ADJ
Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.
干预措施: rFVIII (BAY81-8973) prophylaxis high-dose (Biological)
Arm 6: rFVIII prophylaxis high-dose first CS/ADJ then CS/EP
Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII(BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.
干预措施: rFVIII (BAY81-8973) prophylaxis high-dose (Biological)
结局指标
主要结局
Annualized Number of All Bleeds
时间窗: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)
The annualized number of bleeds experienced by participants
次要结局
- Annualized Number of All Bleeds During CS/ADJ Period(Up to 6 months (6 months on CS/ADJ potency assignment))
- Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973)(Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design))
- Annualized Number of All Bleeds During CS/EP Period(Up to 6 months (6 months on CS/EP potency assignment))