A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- AstraZeneca
- 入组人数
- 80
- 试验地点
- 12
- 主要终点
- Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve)
研究概览
简要总结
This multicenter, observational cohort study uses retrospective collection of past medical history and prospective follow-up to capture longitudinal data on the management and clinical outcomes of patients with atypical hemolytic uremic syndrome (aHUS) treated with ravulizumab as part of routine clinical practice under Poland's National Drug Program (NDP).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of all ages diagnosed with atypical hemolytic uremic syndrome (aHUS) who received treatment with ravulizumab under the National Drug Program (NDP) in Poland.
- •Patients who are willing to participate in the study and have provided informed consent by signing the informed consent form (ICF).
排除标准
- •Individuals who intend to participate in a clinical trial for atypical hemolytic uremic syndrome (aHUS) on or after the date of their first ravulizumab infusion through the National Drug Program.
- •Patients with cognitive impairments, those who are unwilling to participate, or those facing language barriers that hinder adequate comprehension or cooperation.
研究组 & 干预措施
Prospective cohort
Group of patients naive to complement inhibitors
干预措施: Ravulizumab (Drug)
Retrospective cohort
Group of patients who transitioned from other complement inhibitors to ravulizumab.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve)
时间窗: Up to 24 months
In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS
Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched)
时间窗: Up to 24 months
In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS
次要结局
- Time to Complete TMA Response(Up to 24 months)
- Proportion of dialysis-free patients(Up to 24 months)
- Complete TMA response(Up to 24 months)
- Proportion of patients with lab results normalization during observation(Up to 24 months)
- Change from baseline in CKD stage, as evaluated by the physician over time(Up to 24 months)
- Change from baseline in proteinuria status over time(Up to 24 months)
- Change from baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue; adults) and Pediatric Functional Assessment of Chronic Illness Therapy - Fatigue (Peds FACIT-F; pediatric patients) score overtime (naïve)(Up to 24 months)
- Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve)(Up to 24 months)