DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
DemonsTTRate
1 other identifier
observational
2,000
3 countries
24
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jan 2026
Longer than P75 for all trials
24 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 13, 2025
CompletedStudy Start
First participant enrolled
January 9, 2026
CompletedFirst Posted
Study publicly available on registry
January 22, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 23, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 23, 2030
September 9, 2026
September 1, 2026
4.8 years
November 13, 2025
September 4, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (7)
New York Heart Association (NYHA) Class
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.
From time of ATTR-CM diagnosis up to 5 years
Outpatient Diuretic Intensification
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.
From time of enrollment for up to 5 years
Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score
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).
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Kansas City Cardiomyopathy Questionnaire (KCCQ)
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).
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Patient Global Impressions - Change (PGI-C)
The PGI-C is a 7-point numerical rating scale where 1 indicates "very much improved" and 7 indicates "very much worse."
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Patient Global Impressions - Severity (PGI-S);
The PGI-S is a 7-point numerical rating scale where 1 indicates "very much improved" and 7 indicates "very much worse."
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Treatment Satisfaction Questionnaire for Medication version 9 (TSQM-9)
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.
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Study Arms (1)
Patients with ATTR-CM
Patients with ATTR-CM.
Eligibility Criteria
The study population will include adult patients with ATTR-CM who, at enrollment, are either initiating treatment with vutrisiran or are initiating treatment with another approved ATTR-CM therapy.
You may qualify if:
- Has a diagnosis of ATTR-CM
- Is initiating treatment with vutrisiran or another approved ATTR-CM therapy
You may not qualify if:
- Is enrolled in an interventional clinical trial
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (24)
Clinical Trial Site
Tucson, Arizona, 85711, United States
Clinical Trial Site
Beverly Hills, California, 90211, United States
Clinical Trial Site
Naples, Florida, 34102, United States
Clinical Trial Site
Kansas City, Kansas, 66160, United States
Clinical Trial Site
Boston, Massachusetts, 02215, United States
Clinical Trial Site
St Louis, Missouri, 63110, United States
Clinical Trial Site
New Brunswick, New Jersey, 08901, United States
Clinical Trial Site
Charlotte, North Carolina, 28270, United States
Clinical Trial Site
Philadelphia, Pennsylvania, 19104, United States
Clinical Trial Site
Memphis, Tennessee, 38120, United States
Clinical Trial Site
Dallas, Texas, 75390, United States
Clinical Trial Site
Heidelberg, Germany
Clinical Trial Site
Munich, Germany
Clinical Trial Site
Münster, Germany
Clinical Trial Site
Bunkyō City, Japan
Clinical Trial Site
Kumamoto, Japan
Clinical Trial Site
Kurume, Japan
Clinical Trial Site
Matsumoto, Japan
Clinical Trial Site
Sendai, Japan
Clinical Trial Site
Shinjuku, Japan
Clinical Trial Site
Suita, Japan
Clinical Trial Site
Tsukuba, Japan
Clinical Trial Site
Ube, Japan
Clinical Trial Site
Yufu, Japan
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Alnylam Pharmaceuticals
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 13, 2025
First Posted
January 22, 2026
Study Start
January 9, 2026
Primary Completion (Estimated)
October 23, 2030
Study Completion (Estimated)
October 23, 2030
Last Updated
September 9, 2026
Record last verified: 2026-09
Data Sharing
- IPD Sharing
- Will not share