NCT02256917已完成3 期
Prospective, Open-label, Multi-centre Phase 3b Study to Assess the Efficacy and Safety of Personalized Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 58
- 试验地点
- 37
- 主要终点
- Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
研究概览
简要总结
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses. Because trough FVIII levels are likely to be important predictors of the efficacy of prophylaxis, the focus of this study is on pharmacokinetic (PK) data.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Severe Haemophilia A (FVIII:C < 1%)
- •Male patients >= 18 years of age
- •Previous treatment with a FVIII concentrate for at least 150 EDs
- •Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
- •Immunocompetence (CD4+ count > 200/uL)
排除标准
- •Any coagulation disorder other than Haemophilia A
- •Present of past FVIII inhibitor activity
- •Severe liver or kidney disease
结局指标
主要结局
Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
时间窗: 6 months
Total annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
次要结局
- Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis(6 months)
- Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis(6 months)
- Mean Prophylactic Dosing Interval(6 months)
- Mean Residence Time (MRT) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
- AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
- Clearance (CL) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
- In-vivo Recovery (IVR) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
- Number of Patients With Adverse Events (AEs)(At each study visit over the study duration (7-9 months))
- Median Prophylactic Dosing Interval(6 months)
- Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
- Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient)(6 months)
- Half Life (t1/2) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
研究者
研究点 (37)
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