A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis Who Have Previously Received ALN-TTRSC
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Serum TTR Levels
研究概览
简要总结
The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.
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 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who previously received and tolerated revusiran (ALN-TTRSC) in the ALN-TTRSC-002 study
- •Adequate liver function
- •Not Pregnant or nursing
排除标准
- •Inadequate renal function
- •Uncontrolled hypertension, ischemic heart disease or clinically significant cardiac arrhythmia
- •Untreated hypo- or hyperthyroidism
- •Prior major organ transplant
研究组 & 干预措施
Revusiran (ALN-TTRSC)
干预措施: Revusiran (ALN-TTRSC) (Drug)
结局指标
主要结局
Serum TTR Levels
时间窗: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR
Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.
时间窗: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study \[drug\] discontinuation.
次要结局
- Mortality(Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months)
- Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization(Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months)
- 6-minute Walk Test Performance(Baseline, Month 6, and Month 12)