NCT06099236进行中（未招募）不适用
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Chinese Atypical Hemolytic Uremic Syndrome Patients
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- AstraZeneca
- 入组人数
- 367
- 试验地点
- 25
- 主要终点
- Event free survival, where event defined as ESRD or death on or after TMA onset.
研究概览
简要总结
This is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines.
This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according to routine clinical practice defined by local institutional treatment guidelines/protocol. Those aHUS patients who will be treated with a supportive therapy, which does not contain eculizumab, will be monitored for up to 12 months since the ini-tial diagnosis. Patients initiated on eculizumab treatment anytime between aHUS diagno-sis until 12 months will be followed for additional 12 months, starting from the ecu initia-tion. Patient disposition, characteristics, outcomes and safety will be described for all pa-tients enrolled into this study
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants are eligible to be included in the study. Age
- •Patients of any age, who are diagnosed as aHUS by a professional physician (first epi-sode or relapse).
- •Type of Patient and Disease Characteristics
- •Evidence of TMA, including thrombocytopenia, evidence of hemolysis, and kidney dys-function, based on the following laboratory findings, should be recorded within 2 weeks time frame:
- •Platelet count < 150,000 per microliter (μL), and
- •Mechanic hemolytic anemia evident by LDH ≥ 1.5 × upper limit of normal (ULN), and hemoglobin ≤ lower limit of normal (LLN) for age and gender and
- •Serum creatinine level ≥ ULN in adults (≥18 years of age), or ≥ 97.5th percentile for age at screening in children (patients who require dialysis for acute kidney injury are also eligi-ble).
- •Gender: Male and/or female. Informed Consent
- •Willing and able to give written informed consent and comply with the study visit schedule as described in Section 6.2.
- •For patients < 18 years of age, patient's legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent (if applicable as determined by the central.
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Medical Conditions
- •Patients who were diagnosed with HUS only due to Shiga toxin-producing Escherichia coli (STEC).
- •Patients who were diagnosed with TTP (ADAMTS13 activity <10%). Other Exclusions
- •Unable to give written informed consent.
- •Any medical or psychological condition that, in the opinion of the Investigator, could increase the risk to the participant by participating in the study or confound the outcome of the study.
结局指标
主要结局
Event free survival, where event defined as ESRD or death on or after TMA onset.
时间窗: 12 Months
次要结局
- Death(12 Months)
- End-stage renal disease(ESRD)(12 Months)
- Complete TMA Response during observation the as evidenced by simultaneous normalization of hemato-logical parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline(12 Months)
- Complete Thrombotic Microangiopathy(TMA ) Response status over time(12 Months)
- Time to Complete Thrombotic Microangiopathy(TMA ) Response(12 Months)
- Observed value and change from baseline in estimated glomerular filtration rate (eGFR)(12 Months)
- Chronic kidney disease (CKD) stage, as evaluated by the Investigator and classified as improved, stable (no change), or worsened compared to baseline(12 Months)
- Plasma C3, C4, CH50, Factor H and I, soluble C5b-9 (sC5b-9) and CD46 expression over time.(12 Months)
- The proportion of patients with normal platelet count (≥150 x 109/L),LDH levels ≤upper limit of normal , serum creatinine < up-per limit of normal for age or an improvement > 25% compared to baseline,eGFR≥ 60 mL/min/1.73 m2, proteinuria negative.(12 Months)
- Descriptive data of events of interest (serious infec-tions (Aspergillus infections and infections due to en-capsulated bacteria such as Neisseria meningitidis), pregnancy, lactation, and follow-up-data on neonates for 3 months after delivery)(12 Months)
- Descriptive data of Serious Adverse Events (SAEs) for treatment period 2.(12 Months)
- Observed Value in Platelet Count(12 Months)
- Observed Value in Lactate Dehydrogenase (LDH) (mg/dL)(12 Months)
- Observed Value in Hemoglobin (mg/dL)(12 Months)
- Change from Baseline in Platelet Count(12 Months)
- Change from Baseline in Lactate Dehydrogenase (LDH) (mg/dL)(12 Months)
- Change from Baseline in Hemoglobin (mg/dL)(12 Months)
- End-stage renal disease(ESRD)(12 Months)
- Urine protein-to-creatinine ratio over time.(12 Months)
- Plasma C3, C4, CH50, Factor H and I, soluble C5b-9 (sC5b-9) and CD46 expression over time.(12 Months)
- Death(12 Months)
- Chronic kidney disease (CKD) stage, as evaluated by the Investigator and classified as improved, stable (no change), or worsened compared to baseline(12 Months)
- Complete TMA Response during observation the as evidenced by simultaneous normalization of hemato-logical parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline(12 Months)
- Complete Thrombotic Microangiopathy(TMA ) Response status over time(12 Months)
- Time to Complete Thrombotic Microangiopathy(TMA ) Response(12 Months)
- Thrombotic Microangiopathy(TMA ) relapse(12 Months)
- Observed value and change from baseline in estimated glomerular filtration rate (eGFR)(12 Months)
- Proteinuria over time.(12 Months)
- The proportion of patients with normal platelet count (≥150 x 109/L),LDH levels ≤upper limit of normal , serum creatinine < up-per limit of normal for age or an improvement > 25% compared to baseline,eGFR≥ 60 mL/min/1.73 m2, proteinuria negative.(12 Months)
- Descriptive data of events of interest (serious infec-tions (Aspergillus infections and infections due to en-capsulated bacteria such as Neisseria meningitidis), pregnancy, lactation, and follow-up-data on neonates for 3 months after delivery)(12 Months)
- Descriptive data of Serious Adverse Events (SAEs) for treatment period 2.(12 Months)
- Observed Value in Platelet Count(12 Months)
- Observed Value in Lactate Dehydrogenase (LDH) (mg/dL)(12 Months)
- Observed Value in Hemoglobin (mg/dL)(12 Months)
- Change from Baseline in Platelet Count(12 Months)
- Change from Baseline in Lactate Dehydrogenase (LDH) (mg/dL)(12 Months)
- Change from Baseline in Hemoglobin (mg/dL)(12 Months)
- The number of patients still on dialysis during each follow-up visit(12 Months)
研究者
研究点 (25)
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