A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 73
- 试验地点
- 37
- 主要终点
- Characterization of a potential immune response
研究概览
简要总结
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.
In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.
Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males < 12 years of age
- •Subjects with severe hemophilia A
- •Previously treated with FVIII for > 50 exposure days
排除标准
- •Subjects with current evidence of or history of inhibitors to FVIII
- •Any other inherited or acquired bleeding disorder
- •Platelet counts < 100,000/mm^3
- •Creatinine > 2x the upper limit of normal
- •Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal
结局指标
主要结局
Characterization of a potential immune response
时间窗: 12 weeks
Annualized number of all bleeds
时间窗: At least 50 exposure days (ED) over 6 months, on average 245 days
Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period
时间窗: Pre-dose to 72 hours post-dose
Pharmacokinetics profile includes maximum concentration (Cmax), half-life (t1/2), area under the concentration versus time curve (AUC), mean residence time (MRT), volume of distribution at steady state (Vss), and clearance (CL)
Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)
时间窗: At least 50 exposure days (ED) over 6 months, on average 245 days
Inhibitor development in the extension study
时间窗: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
次要结局
- Number of participants with adverse events as a measure of safety and tolerability(From the start of study treatment up to 7 days after the last dose (Main study: on average 245+7 days; Part 2: 12 weeks+7 days; Extension study: on average 5 years+7 days))
- Assessment of incremental recovery in main study(At least 50 exposure days (ED) over 6 months, on average 245 days)
- Inhibitor development in the main study(After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days)