Prospective, Open-Label, Multi-Center, Phase III CLinical Study to Investigate the Efficacy and Safety of Human Factor VWF/FVIII Concentrate (Wilate) in Subjects With Inherited Von Willebrand Disease Who Undergo Surgical Procedures
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 30
- 试验地点
- 30
- 主要终点
- Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale.
研究概览
简要总结
Proportion of surgeries in which the primary endpoint (overall assessment) is classified as success.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with congenital VWD (von Willebrand Disease)
- •Require therapy with a VWF (von Willebrand Factor) product to treat any major surgical procedure
排除标准
- •Known coagulation disorder other than VWD
- •Known history of, or suspected VWF or FVIII inhibitors
- •Subjects with hepatic liver disease
- •Known or suspected hypersensitivity or previous evidence of severe side effects to wilate or other VWF/FVIII concentrates
- •Pregnant women in the first 20 weeks of gestation
结局指标
主要结局
Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale.
时间窗: 30 Days
Efficacy of Wilate in surgical procedures was assessed intra-operatively by the surgeon and post-operatively by the investigator. The IDMC additionally conducted an independent adjudication of all hemostatic efficacy results ('secondary adjudication') and adjudicated the surgeons'/investigators' assessments of the intra- and post-operative assessments where there were discrepancies between the two assessments ('primary adjudication'). It was specified in the SAP that the study will be terminated early and success claimed if the two-sided 98.75% confidence interval (CI) for the overall success rate excludes and is greater than 0.60 (equivalent to 25 or more successes out of the 30 procedures).
次要结局
- Post-operative Efficacy Assessment(up to 30 days)
- Assessment of Intra-operative Hemostatic Efficacy(1 Day)