NCT06679946Enrolling By Invitation3 期
An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Alnylam Pharmaceuticals88 个研究点 分布在 7 个国家目标入组 700 人开始时间: 2024年12月3日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 700
- 试验地点
- 88
- 主要终点
- Frequency of Adverse Events (AEs)
研究概览
简要总结
The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.
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
- •Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003
排除标准
- •Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
- •Future or current participation in another investigational device or drug study, scheduled to occur during this study
- •Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
研究组 & 干预措施
Vutrisiran 25 mg
Experimental
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).
干预措施: Vutrisiran (Drug)
结局指标
主要结局
Frequency of Adverse Events (AEs)
时间窗: Up to 36 Months
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
次要结局
- Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)(Baseline up to 36 Months)
- Change from Baseline in New York Heart Association (NYHA) Class(Baseline up to 36 Months)
- Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)(Baseline up to 36 Months)
- Change From Baseline in the Cardiac Biomarker: Troponin I(Baseline up to 36 Months)
- All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)(Up to 36 Months)
- Change from baseline in serum TTR level(Baseline, Month 6 and Month 12)
研究者
研究点 (88)
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