Observational Study of ATTR-CM Treatments
This is an observational study called DemonsTTRate for people with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where abnormal protein deposits affect the heart. The study aims to understand how vutrisiran and other approved ATTR-CM treatments work in real-world settings. Researchers will look at your heart health (using NYHA Class), how often you need changes to your diuretic medicine, and your quality of life over time. You can join if you have ATTR-CM and are starting treatment with vutrisiran or another approved ATTR-CM medicine. The study is currently unclear on its recruitment status and plans to include about 2000 participants.
- Study design
- This is an observational study, meaning researchers will watch and collect information about your treatment and health over time. It is not a randomized or blinded study.
- What's involved
- You will be followed for up to 5 years, or until you withdraw, are lost to follow-up, or the study ends. The specific visits or procedures are not detailed.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 5 years from the time of ATTR-CM diagnosis or enrollment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
At a glance
Conditions
Where it's being run
4 sites across 4 statesStudy leadership
- Medical Director · STUDY_DIRECTOR · Alnylam Pharmaceuticals
Who to contact
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Do you actually qualify for this trial?
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Inclusion
Exclusion
What this trial measures
- New York Heart Association (NYHA) ClassFrom 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 IntensificationFrom 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 ScoreFrom 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)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)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);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)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.