NCT03303313撤回2 期
A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家开始时间: 2017年9月19日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- The effect of Cemdisiran on platelet count
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with 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
- •Willing to provide written informed consent and to comply with the study requirements
- •Age 18 years or older
- •Clinical diagnosis of primary aHUS
- •Clinical thrombotic microangiopathy (TMA) activity
- •Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
- •Previously vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations
- •ADAMTS13 >10% or other proven aHUS-associated mutation
排除标准
- •Clinically significant abnormal laboratory results
- •Positive Shiga toxin producing Escherichia coli test at Screening
- •Suspected secondary aHUS, in the opinion of the Investigator (unless there is a documented aHUS-associated genetic mutation)
- •Positive direct Coombs test
- •Patients who have received hemodialysis for >3 months
- •Bone marrow transplant recipients
- •Organ transplant recipients, except for kidney transplant recipients with primary aHUS (confirmed by known genetic mutation and kidney biopsy)
- •Known history or evidence of systemic lupus erythematosus or antiphospholipid antibody syndrome
- •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
- •Malignancy (except for non-melanoma skin cancers, cervical in-situ carcinoma, breast ductal carcinoma in situ, or stage 1 prostate carcinoma) within the last 5 years
- •Patients with a poor prognosis that is expected to limit their life expectancy to less than 3 months, in the opinion of the Investigator
研究组 & 干预措施
Cemdisiran
Experimental
干预措施: Cemdisiran (Drug)
结局指标
主要结局
The effect of Cemdisiran on platelet count
时间窗: Week 32
次要结局
- The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy(after 32 weeks of treatment)
- The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)(up to 84 weeks)
- The effect of Cemdisiran on hematological response as measured by platelet count(after 32 weeks of treatment)
- The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count(after 32 weeks of treatment)
- The effect of Cemdisiran on serum creatinine levels(up to 84 weeks)
- The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH(after 32 weeks of treatment)
- The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels(after 32 weeks of treatment)
- The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)(after 32 weeks of treatment)
- The effect of Cemdisiran on hematological response as measured by rescue plasma therapy(after 32 weeks of treatment)
- The effect of Cemdisiran on LDH response as measured by LDH(after 32 weeks of treatment)
- The effect of Cemdisiran on LDH response as measured by rescue plasma therapy(after 32 weeks of treatment)
- The effect of Cemdisiran on adverse events (AEs)(up to 108 weeks)
研究者
研究点 (1)
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