跳至主要内容
临床试验/NCT07358078
NCT07358078招募中不适用

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy in a Real-World Setting

Alnylam Pharmaceuticals24 个研究点 分布在 3 个国家目标入组 2,000 人开始时间: 2026年1月9日最近更新:
适应症

试验速览

阶段
不适用
状态
招募中
发起方
入组人数
2,000
试验地点
24
主要终点
New York Heart Association (NYHA) Class

研究概览

简要总结

The purpose of this study is to:

  • Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care
  • Describe treatment patterns of adult patients with ATTR-CM treated with vutrisiran in routine clinical care
  • Assess health-related quality of life (HRQOL) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care
  • Assess healthcare resource use (HCRU) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care
  • Compare the long-term effectiveness of vutrisiran versus other approved ATTR-CM treatments in routine clinical care

详细描述

Each site will invite all eligible patients to participate. Patients will be followed for up to 5 years or until patient withdrawal of consent, loss to follow-up, death, or the end of the study, whichever comes first.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • Has a diagnosis of ATTR-CM
  • Is initiating treatment with vutrisiran or another approved ATTR-CM therapy

排除标准

  • Is enrolled in an interventional clinical trial

研究组 & 干预措施

Patients with ATTR-CM

Patients with ATTR-CM.

结局指标

主要结局

New York Heart Association (NYHA) Class

时间窗: From time of ATTR-CM diagnosis up to 5 years

The NYHA functional classification categorizes the severity of heart failure. Patients are classified in 1 of 4 categories based on their physical limitations, as follows: * Class I No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, or shortness of breath. * Class II Slight limitation of physical activity. Comfortable at rest. Ordinary physical activity results in fatigue, palpitation, shortness of breath or chest pain. * Class III Marked limitation of physical activity. Comfortable at rest. Less than ordinary activity causes fatigue, palpitation, shortness of breath or chest pain. * Class IV Symptoms of heart failure at rest. Any physical activity causes further discomfort.

Outpatient Diuretic Intensification

时间窗: From time of enrollment for up to 5 years

The clinician-reported outcome of ODI, which measures any post-diagnosis initiation or increase in the dose of loop diuretics, may help identify patients with ATTR-CM who are at an increased risk for adverse outcomes. ODI, a marker to monitor disease progression and/or worsening heart failure, is associated with a higher risk of all-cause mortality, cardiovascular hospitalizations, and urgent heart failure visits. Clinicians monitor ODI to help manage patients with ATTR-CM.

Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score

时间窗: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years

Norfolk-QoL-DN: The Norfolk QOL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that assesses 6 domains: physical function, large-fiber neuropathy, activities of daily living, symptoms, small-fiber neuropathy, and autonomic neuropathy. The total score ranges from -4 points (best possible quality of life) to 136 points (worst possible quality of life).

Kansas City Cardiomyopathy Questionnaire (KCCQ)

时间窗: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years

The KCCQ is a 23-item self-administered questionnaire developed to independently measure the patient's perception of health status, which includes heart failure symptoms, impact on physical and social function, and how their heart failure impacts their quality of life within a 2-week recall period. The KCCQ quantifies 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary \[OS\] scores).

Patient Global Impressions - Change (PGI-C)

时间窗: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years

The PGI-C is a 7-point numerical rating scale where 1 indicates "very much improved" and 7 indicates "very much worse."

Patient Global Impressions - Severity (PGI-S);

时间窗: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years

The PGI-S is a 7-point numerical rating scale where 1 indicates "very much improved" and 7 indicates "very much worse."

Treatment Satisfaction Questionnaire for Medication version 9 (TSQM-9)

时间窗: From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years

TSQM-9 is a 9-item general instrument that measures the major dimensions of satisfaction with a medication. The questionnaire consists of 3 domains: effectiveness, convenience and side effects. The scores of each domain range from 0 to 100 with higher scores representing higher satisfaction on that domain.

次要结局

未报告次要终点

研究者

发起方
Alnylam Pharmaceuticals
申办方类型
Industry
责任方
Sponsor

研究点 (24)

Loading locations...

相似试验