NCT07308574招募中4 期
Multicenter, Open-label, Single-arm, Post-Marketing Clinical Study to Evaluate the Efficacy and Safety of Ravulizumab in Participants Clinically Diagnosed as Atypical Hemolytic Uremic Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 13
- 主要终点
- Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment
研究概览
简要总结
The primary objective of this study is to assess the platelet count response to ravulizumab in participants clinically diagnosed as atypical hemolytic uremic syndrome (aHUS).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Body weight ≥20 kilograms (kg)
- •Participants clinically diagnosed as aHUS who have any of diseases/conditions listed below (including participants in whom Thrombotic microangiopathy (TMA) has not been improved even after treatment for the pathogenesis of diagnosed secondary TMA and therefore, diagnosis of aHUS was made).
- •Infection (except for pneumococcal infection and Siga toxin-producing Escherichia coli infection)
- •During pregnancy or postpartum
- •Post-renal transplantation
- •Hypertensive crisis/malignant hypertension
- •Systemic lupus erythematosus and related diseases (e.g. dermatomyositis, mixed connective tissue disease, etc.)
- •Participants with the following three signs:
- •Thrombocytopenia: Platelet count <150,000/microliter (μL)
- •Microangiopathic haemolytic anaemia: Hb < 10 grams per deciliter (g/dL) (*)
- •Acute kidney injury: one of the following is fulfilled;
- •ΔsCr ≥ 0.3 milligrams per deciliter (mg/dL) (within 48 hours),
- •1.5-fold increase from baseline sCr (within 7 days),
- •urinary output ≤ 0.5 mL/kg/hour for ≥ 6 hours.
- •No prior treatment with complement inhibitors.
- •The investigator plans to provide the participant with 26-week treatment with ravulizumab in accordance with the treatment policy in clinical practice.
- •Ravulizumab treatment is planned to be initiated within 14 days after onset of the latest TMA episode.
- •Participants consenting to meningococcal vaccine administration and appropriate antibiotic prophylaxis (if required).
排除标准
- •Participants with TTP, STEC-HUS, secondary TMA that is obviously unrelated to complement abnormality.
- •Participants with TMA caused by malignant tumors, abnormal Cobalamin C metabolism, Streptococcus pneumoniae, drugs, autoimmune diseases other than systemic lupus erythematosus and related diseases (e.g. scleroderma etc.), or hematopoietic stem cell transplantation
- •Participants with pathological complement gene variants (CFH, CFI , CD46 (MCP), C3, CFB, THBD, DGKE) associated with the development of aHUS at enrolment
- •Participants with positive anti-factor H antibodies
- •More than 14 day from onset of TMA to the planned start of ravulizumab treatment
- •Chronic kidney disease or irreversible renal impairment that requires chronic dialysis
- •Presence of unresolved meningococcal disease
- •Judgement by the investigator that the participant is not eligible for the study
研究组 & 干预措施
Ravulizumab
Experimental
Participants will receive a weight-based loading dose of ravulizumab, followed by a weight-based dose 2 weeks after loading dose administration, then weight-based maintenance doses every 8 weeks via intravenous (IV) infusion.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment
时间窗: Baseline up to Week 26
次要结局
- Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment(Baseline up to Week 26)
- Percentage of Participants who are on Dialysis on Day 1 and are Able to Withdraw From Dialysis by Week 26(Baseline (Day 1) up to Week 26)
- Percentage of Participants Showing Improvement in Renal Function(Day 4 and on Weeks 1, 2, 10, 18, and 26)
- Percentage of Participants Showing Improvement in Platelet Count(Day 4 and on Weeks 1, 2, 10, 18, and 26)
- Change From Baseline in Estimated Glomerular Filtration Rate(Baseline (Day 1), Week 26)
- Percentage of Participants Showing Improvement in Complete Thrombotic Microangiopathy (TMA) Response or Partial TMA Response(Day 4 and on Weeks 1, 2, 10, 18, and 26)
- Change from Baseline in Platelet Count(Baseline (Day 1), Week 26)
- Change From Baseline in Hemoglobin(Baseline (Day 1), Week 26)
- Change From Baseline in Lactate Dehydrogenase(Baseline (Day 1), Week 26)
研究者
研究点 (13)
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