NCT00122304已完成3 期
Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 85
- 试验地点
- 96
- 主要终点
- Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
研究概览
简要总结
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •PNH > 6 months
- •Type III PNH red blood cell (RBC) clone by flow cytometry >10%
- •At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH
- •Lactate dehydrogenase (LDH) >1.5 x upper limit of normal
- •Must avoid conception
- •Willing and able to give written informed consent
排除标准
- •Platelet count of <30,000/mm3
- •Absolute neutrophil count <500/ul
- •Active bacterial infection
- •Hereditary complement deficiency
- •History of bone marrow transplantation
- •Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
- •Pregnant, breast-feeding, or intending to conceive
- •History of meningococcal disease
结局指标
主要结局
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
次要结局
- Hemolysis measured by the change of LDH from baseline;
- Quality of Life
研究者
研究点 (96)
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