NCT01303952已完成2 期
Therapy for Chronic Cold Agglutinin Disease: A Prospective, Non-Randomized Multi-Center Study Demonstrating the Efficacy of Terminal Complement Inhibition in Patients With Cold Agglutinin Disease Using Eculizumab
University Hospital, Essen1 个研究点 分布在 1 个国家目标入组 13 人开始时间: 2011年1月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- LDH
研究概览
简要总结
Evaluation of the efficacy and safety of eculizumab in symptomatic or transfusion-dependent patients with untreated or refractory hemolytic cold agglutinin disease(CAD)
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
- •Individuals at least 18 years of age
- •Diagnosis of CAD defined by the combination of chronic hemolysis, cold agglutinin titer (IgM or IgA) >64 at 4°C, positive direct antiglobulin test (DAT) when performed with polyspecific antiserum, negative (or only weakly positive) anti-IgG, and strongly positive with anti-C3d
- •LDH level > 2 x upper limit of normal (ULN)
- •Clinical symptoms requiring treatment, such as anemia, anemia-related symptoms or hemolysis-related symptoms
- •Sufficient supplementation of iron, folic acid and vitamin B12 prior to treatment phase
- •Patient must be willing and able to give written informed consent;
- •Patients must be vaccinated against N. meningitidis at least 14 days prior treatment
- •Patient must avoid conception during the trial using a method that is most appropriate for their physical state and culture
排除标准
- •Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix
- •Liver disease with elevated LDH
- •Absolute neutrophil count < 500/µL
- •Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at treatment start or severe recurrent bacterial infections
- •Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
- •Pregnant, breast-feeding, or intending to conceive during the course of the study, including the Post-treatment Phase
- •History of bone marrow/stem cell transplantation
- •Inability to cooperate or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study
- •Hypersensitivity to eculizumab, to murine proteins or to one of the excipients
研究组 & 干预措施
Eculizumab
Experimental
干预措施: Eculizumab (Drug)
结局指标
主要结局
LDH
时间窗: From baseline to 26 weeks
Primary endpoint for this study is: lactate dehydrogenase (LDH) in U/l change from baseline to 26 weeks
次要结局
- SF-36v2 QLQ(From baseline to 26 weeks)
- Circulatory symptoms(From baseline to 26 weeks)
- Hemopexin(From baseline to 26 weeks)
- Free hemoglobin(From baseline to 26 weeks)
- Reticulocytes(From baseline to 26 weeks)
- Transfusion avoidance(From baseline to 26 weeks)
- PRBC units transfused(From baseline to 26 weeks)
- Haptoglobin(From baseline to 26 weeks)
- Change in hemoglobin levels(From baseline to 26 weeks)
- Six-Minute Walk Test(From baseline to 26 weeks)
- FACIT-F SCALE version 4(From baseline to 26 weeks)
- Thrombosis record(From baseline to 26 weeks)
研究者
Alexander Roeth, MD
MD
University Hospital, Essen
研究点 (1)
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