NCT01311648已完成3 期
A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 94
- 试验地点
- 43
- 主要终点
- Annualized Number of Total Bleeds Within 48 h
研究概览
简要总结
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
The secondary objectives were
- To assess the safety and efficacy of BAY81-8973 during surgeries.
- To assess incremental recovery of BAY81-8973.
- To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated patients [PTPs] and previously untreated patients [PUPs] / minimally treated patients [MTPs] - participation in PK sampling was voluntary and required consent).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •PTPs (previously treated patients): aged <= 12 years
- •PUPs (previously untreated patients) / MTPs (minimally treated patients): aged < 6 years
- •Severe hemophilia A defined as < 1% FVIII concentration (FVIII:C)
- •PTPs: >= 50 exposure days (EDs) with any FVIII concentrate, no current evidence of inhibitory antibody, and no history of FVIII inhibitor formation
- •PUPs: no prior exposure to any FVIII product
- •MTPs: having no more than 3 EDs with any FVIII product, no current evidence of inhibitory antibody and no history of FVIII inhibitor formation
排除标准
- •With another bleeding disorder that is different from Hemophilia A
- •With thrombocytopenia (platelet count < 100 000/mm^3)
- •Creatinine > 2x upper limit of normal or Aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) > 5x upper limit of normal
- •Without a negative inhibitor testing at screening (except for PUPs)
- •Receiving chemotherapy, immune modulatory drugs, has received another investigational FVIII product within the last month, or received another experimental drug within the last 3 months
- •Requires any pre-medication to tolerate FVIII treatment
- •Known hypersensitivity to active substance, mouse, or hamster protein
结局指标
主要结局
Annualized Number of Total Bleeds Within 48 h
时间窗: Within 48 hours post infusion
Annualized number (mean +/- standard deviation) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days \[EDs\]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
次要结局
- Half-life (t1/2) of BAY81-8973 in Plasma(Pre-infusion and until 24 hours post infusion)
- Number of Participants With Inhibitor Development in Main Study(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Factor VIII Recovery Values(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Number of Infusions Per Bleed(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Response to Treatment of Bleeds(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Hemostatic Control During Major and Minor Surgeries(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Number of Participants With New Inhibitor Development in Extension Study(From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years))
- Consumption of Factor VIII in All Infusions(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Consumption of FVIII in Infusions for Prophylaxis(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Consumption of FVIII in Infusions for the Treatment of Bleeds(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
- Annualized Number of Total Bleeds During Prophylaxis Treatment(Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months))
研究者
研究点 (43)
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