NCT02595983已完成2 期
An Open-label Study to Evaluate the Efficacy and Safety of Revusiran in Patients With Transthyretin-mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post-Orthotopic Liver Transplant
Alnylam Pharmaceuticals2 个研究点 分布在 2 个国家目标入组 12 人开始时间: 2015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
- 12
- 试验地点
- 2
- 主要终点
- Percentage Change From Baseline in Serum TTR at Month 6
研究概览
简要总结
The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of FAP (familial amyloidotic polyneuropathy) with documented TTR mutation
- •Received an orthotopic liver transplant ≥12 months before the date of informed consent
- •An increase in polyneuropathy disability (PND) score post-transplant
- •Polyneuropathy Disability score of ≤3b
排除标准
- •New York Heart Association (NYHA) classification of >2
- •Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)
研究组 & 干预措施
All Patients
Experimental
All patients who received at least 1 dose of revusiran (ALN-TTRSC)
干预措施: Revusiran (Drug)
结局指标
主要结局
Percentage Change From Baseline in Serum TTR at Month 6
时间窗: Month 6
A negative percentage change from baseline at Month 6 indicates a reduction in serum TTR level.
次要结局
- Percentage Change From Baseline in Serum TTR Over 18 Months(Weeks 3, 7, 12, 18, 24, 26 (Month 6), 39 (Month 9), 52 (Month 12), 57, 78 (Month 18))
- Number of Participants in Each Polyneuropathy Disability (PND) Stage Based on Worst Post-Baseline Score(Baseline, Months 6, 12, 18)
- Change From Baseline in Modified Neurological Impairment Score (mNIS +7) Composite Score Over 18 Months(Baseline, Months 6, 12, 18)
- Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Score(Baseline, Months 6, 12, 18)
研究者
研究点 (2)
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