NCT01961921已完成2 期
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 27 人开始时间: 2013年10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
- 27
- 试验地点
- 1
- 主要终点
- The Number of Participants 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 evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-
- •Adequate Karnofsky performance status, liver function, and renal function.
排除标准
- •Pregnant or nursing.
- •Has had a liver transplant.
- •Has a New York Heart Association heart failure classification >
- •Has unstable angina.
- •Has uncontrolled clinically significant cardiac arrhythmia.
研究组 & 干预措施
ALN-TTR02 (patisiran)
Experimental
干预措施: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion (Drug)
结局指标
主要结局
The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
时间窗: From Baseline up to 56 days post last dose
An AE is any untoward medical occurrence in a patient or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.
次要结局
- Change in Gait Speed With 10-meter Walk Test(Baseline, Month 24)
- Mean Change From Baseline in Hand Grip Strength(Baseline, Month 24)
- Percentage Change From Baseline in Serum TTR Levels(From Baseline up to 56 days post last dose)
- Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7)(Baseline, Month 24)
- Change From Baseline in Quality of Life and Disability as Assessed by the EuroQoL (Quality of Life)-5 Dimensions (EQ-5D), EuroQoL Visual Analog Scale (EQ-VAS) Questionnaires and Rasch-built Overall Disability Scale (R-ODS)(Baseline, Month 24)
- Change From Baseline in Nutritional Status (Modified Body Mass Index, mBMI)(Baseline, Month 24)
研究者
研究点 (1)
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