An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 3,000
- 试验地点
- 12
- 主要终点
- Proportion of patients who experience specified events
研究概览
简要总结
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.
详细描述
The study will capture post-marketing safety data on patients treated with eculizumab or ravulizumab. Additionally, the study will collect information on the progression of disease in all patients.
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
- •Male or female patients of any age, including minors, who have been diagnosed with aHUS
- •Patients with or without an identified complement pathogenic variant or anti-complement factor antibody
- •Able to give written informed consent. Patient or patient's parent/legal guardian must be willing and able to given written informed consent and the patient (if minor) must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].
- •ADAMTS13 > 5%, if performed.
排除标准
- •Hemolytic Uremic Syndrome (HUS) only due to Shiga Toxin producing Escherichia coli (STEC).
- •Unable to give written informed consent. Patient or patient's parent/legal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees [IRB/IEC]).
结局指标
主要结局
Proportion of patients who experience specified events
时间窗: 10 years
To collect and evaluate safety and effectiveness data specific to the use of eculizumab or ravulizumab in aHUS patients.
Time to first and subsequent occurrence of specified events.
时间窗: 5 years
To assess the long term manifestations of thrombotic microangiopathy (TMA) complications of aHUS as well as other clinical outcomes, including morbidity and mortality in aHUS patients, receiving eculizumab or ravulizumab treatment or other disease management approaches
次要结局
未报告次要终点