NCT01757431已完成2 期
The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 2
- 试验地点
- 2
- 主要终点
- Adverse events and serious adverse events and their severity and relationship to the drug
研究概览
简要总结
Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients with diagnosis of aHUS who have been receiving eculizumab by personal importation (specific eligibility criteria below do not apply) Or,
- •Patients with current clinical manifestations of aHUS who meet the following criteria:
- •Patient with diagnosis of aHUS with or without an identified complement regulatory protein genetic abnormality or anti-complement factor antibody and for whom other known etiologies of hemolytic uremic syndrome (HUS) have been ruled out as confirmed in the Exclusion Criteria
- •Patient (and legal guardian if patient is not an adult) willing and able to give written informed consent and assent (or verbal assent if patient is unable to read or write)
- •Patient at least 1 month of age and body weight ≥5 kg
- •Platelet count at screening < lower limit of normal (LLN)
- •Signs or symptoms of hemolysis (i.e., lactate dehydrogenase (LDH) ≥ 1.5x upper limit of normal (ULN) and Hemoglobin ≤ LLN) at start of current aHUS event
- •Serum Creatinine (SrCr) level ≥ ULN at screening (patient requiring dialysis for acute renal failure also eligible)
- •Female patient of childbearing potential practicing an effective, reliable and medically approved contraceptive regimen during the entire duration of the study, including the Follow-up Period. At the time of the last follow-up visit, patient must agree to continue to use adequate contraception methods for up to 5 months following discontinuation of eculizumab treatment
- •Able and willing to comply with study procedures
排除标准
- •Shiga-toxin producing E. coli-HUS (STEC-HUS; shiga-toxin and/or STEC positive) History of malignancy within 5 years of screening Known human immunodeficiency virus (HIV) infection Identified drug exposure-related HUS Infection-related HUS HUS related to bone marrow transplant (BMT) HUS related to vitamin B12 deficiency Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage renal disease (ESRD)) Patients with a confirmed diagnosis of sepsis defined as positive blood cultures within 7 days of the screening visit and not treated with antibiotics to which the organism is sensitive Presence or suspicion of active and untreated systemic bacterial infection that, in the opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease Pregnancy or lactation Unresolved systemic meningococcal disease Any medical or psychological condition that, in the opinion of the investigator, could increase patient's risk by participating in the study or confound the outcome of the study Patients receiving chronic intravenous immunoglobulin (IVIG) within 8 weeks unless for unrelated medical condition (e.g., hypogammaglobulinemia) or chronic rituximab therapy within 12 weeks of the screening visit Patients receiving other immunosuppressive therapies such as steroids, calcineurin inhibitors (mTOR), (e.g., cyclosporine or tacrolimus) are excluded unless: [1] part of an established post-transplant anti-rejection regimen, or [2] patient has confirmed anti-Complement Factor Antibodies requiring immunosuppressive therapy or [3] steroids are used for a condition other than aHUS (e.g., asthma) Prior eculizumab use, hypersensitivity to eculizumab, to murine proteins or to one of the excipients Inclusion in any other investigational intervention trial except this study
研究组 & 干预措施
ECULIZUMAB
Other
干预措施: Eculizumab (Drug)
结局指标
主要结局
Adverse events and serious adverse events and their severity and relationship to the drug
时间窗: 12 weeks
次要结局
未报告次要终点
研究者
研究点 (2)
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