Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Atypical Hemolytic Uremic Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- AO GENERIUM
- 入组人数
- 50
- 试验地点
- 8
- 主要终点
- Change in platelet count compared to the screening level
研究概览
简要总结
It is a multicenter observational non-comparative study of the efficacy and safety of long-term pathogenetic Elizaria® therapy in patients with atypical Hemolytic Uremic Syndrome
详细描述
After screening, patients meeting all of the inclusion / non-inclusion criteria and vaccinated against meningococcal infections were treated by Elizaria®.
The study is planned to include at least 50 patients receiving Elizaria® for the aHUS treatment.
The study will consist of a screening period of up to 4 weeks, including, if necessary, immunization with meningococcal vaccine, a treatment period of 52 weeks.
Medication will be prescribed in accordance with routine medical practice. Accordingly to minimize the risks and subjectivity of assessments the methods adopted in the routine practice of treating patients with aHUS will be used.
Investigators enroll patients with aHUS diagnosis who have indications for pathogenetic therapy and who are receiving Elizaria® under the government program. Patients will receive medication in accordance with the established requirements of national standards and protocols for the treatment of patients with aHUS. The registration of the amount of the drug used will be carried out on the basis of information in the Patient Diaries, as well as primary documentation.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Elizaria®
Eculizumab
干预措施: Elizaria® (Drug)
结局指标
主要结局
Change in platelet count compared to the screening level
时间窗: 52 week
Change in platelet count at 52 week after treatment with study drug compared to baseline at screening
次要结局
- Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2(52 week)
- Proportion of patients with normalized platelet levels(52 week)
- Proportion of TMA-related interventions(52 week)
- Proportion of patients with an improvement in glomerular filtration rate (eGFR) of 15 ml / min / 1.73m2 or more compared to the baseline level at screening.(52 week)
- The frequency and severity of adverse events (AEs)(52 weeks)
- Proportion of patients with antidrug antibodies(52 weeks)
- Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to baseline at screening.(52 week)
- Proportion of patients with no thrombotic microangiopathy (TMA) events(52 week)
- Change in lactate dehydrogenase (LDH) levels from baseline at screening(52 week)
- Change in eGFR (ml / min. / 1.73m2) compared with the baseline level at screening;(52 week)
- Proportion of patients with complete TMA response(52 week)
- Proportion of patients with an increase in hemoglobin level of more than 20 g / l compared to the baseline level at screening.(52 week)