Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- FVIII inhibitor development in inhibitor negative subjects
研究概览
简要总结
The goal of this observational study is to learn about the changes of antibodies and inhibitors against the coagulation factor VIII in patients with severe hemophilia A receiving emicizumab therapy. No additional visits or procedures are planned. Patients in this study will continue to receive their routine care and analysis will be done from left over samples from routine visits.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Severe congenital hemophilia A (CHA)
- •Treatment with emicizumab irrespective of any other treatment
- •Informed consent
排除标准
- •No therapy with emicizumab
- •Immunosuppressive therapy
- •HIV-infection with CD4 (cluster of differentiation 4) cells <200/µl
结局指标
主要结局
FVIII inhibitor development in inhibitor negative subjects
时间窗: 3 years
Rate of FVIII inhibitor development during three years of emicizumab prophylaxis in inhibitor negative subjects. Assessed with Bethesda Assay (BU/ml). Number of patients who develop an FVIII inhibitor within the study period, but were FVIII inhibitor negative at start of the study.
FVIII antibody development in inhibitor negative subjects
时间窗: 3 years
Rate of FVIII antibody development during three years of emicizumab prophylaxis in inhibitor negative subjects. FVIII anti drug antibody (ADA) is assessed by FVIII specific ELISA (OD=Optical Density). Number of patients who develop an FVIII antibody (ADA) within the study period, but were FVIII inhibitor negative at start of the study.
FVIII inhibitor disappearance in inhibitor positive subjects
时间窗: 3 years
Rate of FVIII inhibitor disappearance during three years of emicizumab prophylaxis in inhibitor positive subjects. Assessed with Bethesda Assay (BU/ml). Number of patients who loose an FVIII inhibitor within the study period, but were FVIII inhibitor positive at start of the study.
FVIII antibody disappearance in inhibitor positive subjects
时间窗: 3 years
Rate of FVIII antibody disappearance during three years of emicizumab prophylaxis in inhibitor positive subjects. FVIII anti drug antibody (ADA) is assessed by FVIII specific ELISA (OD=Optical Density). Number of patients who develop an FVIII antibody within the study period, but were FVIII inhibitor positive at start of the study.
次要结局
- Anti-FVIII antibody development(3 years)
- Quality of the antibody response (FVIII epitopes)(3 years)
- Response to treatment(3 years)
- Quality of the antibody response (IgG subclasses)(3 years)
- Anti-FVIII inhibitor development(3 years)
- Treatment of bleeds(3 years)
- Time to negative inhibitor titers(3 years)
研究者
Christoph Königs
Principal Investigator, Head of molecular and clinical Haemostasis
Goethe University