NCT01981837已完成2 期
A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 26 人开始时间: 2013年12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
- 26
- 试验地点
- 1
- 主要终点
- The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
研究概览
简要总结
The purpose of this study is to determine the pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •TTR cardiac amyloidosis;
- •Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 2 highly effective methods of contraception;
- •Male subjects agree to use appropriate contraception;
- •Adequate blood counts, liver, renal and heart function;
- •Adequate Karnofsky performance status;
- •Adequate New York Heart Association (NYHA) Classification Score;
- •Clinically stable on heart medications;
- •Adequate 6-minute walk test;
- •Willing to give written informed consent and are willing to comply with the study requirements.
排除标准
- •Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
- •Subjects with a history of multiple drug allergies or intolerance to SC injection;
- •Received an investigational agent other than tafamidis, diflunisal, doxycycline or tauroursodeoxycholic acid, or an investigational device within 30 days prior to first dose of study;
- •Uncontrolled hypertension, ischemic heart disease or cardiac arrhythmia;
- •Untreated hypo- or hyperthyroidism;
- •Prior major organ transplant;
- •Considered unfit for the study by the Principal Investigator.
研究组 & 干预措施
ALN-TTRSC (revusiran)
Experimental
干预措施: ALN-TTRSC (revusiran) for subcutaneous administration (Drug)
结局指标
主要结局
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
时间窗: Up to 63 days
次要结局
- Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)(Up to 90 days)
- Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)(Up to 90 days)
研究者
研究点 (1)
Loading locations...
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