NCT02319005已完成3 期
A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 206 人开始时间: 2014年12月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 206
- 试验地点
- 1
- 主要终点
- 6 Minute Walk Distance (6-MWD)
研究概览
简要总结
The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented TTR mutation
- •Amyloid deposits in cardiac or non-cardiac tissue
- •Medical history of heart failure
- •Evidence of cardiac involvement by echocardiogram
排除标准
- •Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non-FAC hereditary cardiomyopathy, hypertensive cardiomyopathy, or cardiomyopathy due to valvular heart disease
- •Has known peripheral vascular disease affecting ambulation
- •Has a Polyneuropathy Disability score >2
- •Has a New York Heart Association (NYHA) classification of IV
研究组 & 干预措施
Revusiran (ALN-TTRSC)
Active Comparator
administered by subcutaneous (SC) injection
干预措施: Revusiran (ALN-TTRSC) (Drug)
Sterile Normal Saline (0.9% NaCl)
Placebo Comparator
administered by subcutaneous (SC) injection
干预措施: Sterile Normal Saline (0.9% NaCl) (Drug)
结局指标
主要结局
6 Minute Walk Distance (6-MWD)
时间窗: 18 months
The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
Serum TTR Levels
时间窗: 18 months
The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
次要结局
- Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization(18 months)
- New York Heart Association (NYHA) Class(18 months)
- Cardiovascular (CV) Mortality(18 months)
- All-cause Mortality(18 months)
- Kansas City Cardiomyopathy Questionnaire (KCCQ)(18 months)
- Cardiovascular (CV) Hospitalization(18 months)
研究者
研究点 (1)
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