Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 100
- 试验地点
- 4
- 主要终点
- Area under the curve ROC of Residual plasma level of emicizumab
研究概览
简要总结
Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult or child with Clinical diagnosis of severe hemophilia A (FVIII activity < 1%) with or without inhibitor
- •Clinical indication to emicizumab therapy
排除标准
- •Refusal to give informed consent
- •acquired hemophilia A
- •other inherited or acquired bleeding disorder
- •bodyweight < 10 kgs
研究组 & 干预措施
Severe haemophila A patients with or without inhibitors
结局指标
主要结局
Area under the curve ROC of Residual plasma level of emicizumab
时间窗: At Week 5 (end of emicizumab loading period)
At least one clinically significant bleeding (defined as any bleeding treated with FVIII, rFVIIa or aPCC) from loading period completion (week 5) to the end of study, an average of 1 year
次要结局
- Residual plasma level of emicizumab (UPLC-MS/MS dosing)(At Week 5 and at each breakthrough bleeding until end of study)
- Residual plasma level of emicizumab measured by UPLC-MS/MS(At each breakthrough bleeding until end of study)
- Residual plasma level of emicizumab measured by UPLC-MS/MS(At Week 5 (end of emicizumab loading period))