An Open-label, Multi-center Controlled Clinical Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (AHUS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 主要终点
- Percentage of Patients With TMA Event-free Status
研究概览
简要总结
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adult patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).
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
- 未提供
排除标准
- •TTP, (defined as ADAMTS-13 activity <5%) from an historical observation (prior to initiation of plasma therapy) or as tested at the screening visit by the central laboratory.
- •Malignancy within 5 years of screening.
- •Typical HUS (Shiga toxin +).
- •Known HIV infection.
- •Identified drug exposure-related HUS.
- •Infection-related HUS.
- •HUS related to bone marrow transplant.
- •HUS related to vitamin B12 deficiency.
- •Patients with a confirmed diagnosis of sepsis.
- •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 meningococcal disease.
- •Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome.
- •Any medical or psychological 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.
- •Patients who have received previous treatment with eculizumab.
- •Patients receiving IVIg within 8 weeks or Rituximab therapy within 12 weeks of the screening visit.
- •Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors or tacrolimus are excluded unless: [1] part of an established post-transplant anti-rejection regime, [2] patient has confirmed anti-CFH antibody requiring immunosuppressive therapy, and [3] dose of such medications have been unchanged for at least 4 weeks prior to the screening period and throughout the Observation Period or [4] patient is experiencing an acute aHUS relapse immediately after transplant.
- •Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable dose for at least 4 weeks prior to the screening period, or a washout period of at least 2 weeks from the last dose of ESA therapy.
- •Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures beginning 4 weeks prior to screening and throughout the entire trial.
- •Hypersensitivity to eculizumab, to murine proteins or to one of the excipients.
研究组 & 干预措施
eculizumab
干预措施: eculizumab (Drug)
结局指标
主要结局
Percentage of Patients With TMA Event-free Status
时间窗: Through 26 weeks
TMA Event-free status is defined as the absence for at least 12 weeks of \[1\] decrease in platelet count of \> 25% from the Platelet Count Pre-PT Baseline Set Point; \[2\] PT while the patient is receiving eculizumab, and \[3\] new dialysis.
Percentage of Patients With Hematologic Normalization
时间窗: Through 26 weeks
Hematologic Normalization was defined as normalization of both platelet count and lactic dehydrogenase (LDH) sustained for at least two consecutive measurements which spanned a period of at least four weeks.
Percentage of Patients With Complete TMA Response
时间窗: Through 26 weeks
The proportion of patients who achieved a Complete TMA Response from baseline through 26 weeks of treatment with eculizumab was determined. Complete TMA Response was defined as Hematologic Normalization plus improvement in renal function (defined as (≥25% reduction from baseline in serum creatinine), which was sustained for two consecutive measurements over a period of at least four weeks.
次要结局
- Platelet Count Change From Baseline to 26 Weeks(From Baseline to 26 Weeks)
- TMA Intervention Rate(Through End of Study, Median Exposure 156 Weeks)
- Percentage of Patients With Complete TMA Response(Through End of Study, Median Exposure 156 Weeks)
- Platelet Count Change From Baseline to 156 Weeks(From Baseline to 156 Weeks)
- Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration(Induction Phase for 4 weeks followed by Maintenance Phase starting on Week 5 through 26 weeks or longer.)
- Percentage of Patients With Platelet Count Normalization(Through End of Study, Median Exposure 156 Weeks)
- Percentage of Patients With TMA Event-free Status(Through End of Study, Median Exposure 156 Weeks)
- Percentage of Patients With Hematologic Normalization(Through End of Study, Median Exposure 156 Weeks)