DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting (DemonsTTRate)

April 23, 2026 updated by: Alnylam Pharmaceuticals

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

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

Study Overview

Status

Recruiting

Conditions

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

2000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Florida
      • Naples, Florida, United States, 34102
        • Recruiting
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Clinical Trial Site
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Recruiting
        • Clinical Trial Site
    • Tennessee
      • Memphis, Tennessee, United States, 38120
        • Recruiting
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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.

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • Is enrolled in an interventional clinical trial

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Patients with ATTR-CM
Patients with ATTR-CM.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
New York Heart Association (NYHA) Class
Time Frame: 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.
From time of ATTR-CM diagnosis up to 5 years
Outpatient Diuretic Intensification
Time Frame: 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.
From time of enrollment for up to 5 years
Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score
Time Frame: 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).
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Kansas City Cardiomyopathy Questionnaire (KCCQ)
Time Frame: 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).
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Patient Global Impressions - Change (PGI-C)
Time Frame: 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."
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Patient Global Impressions - Severity (PGI-S);
Time Frame: 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."
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years
Treatment Satisfaction Questionnaire for Medication version 9 (TSQM-9)
Time Frame: 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.
From time of enrollment (if available) or ATTR-CM diagnosis up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 9, 2026

Primary Completion (Estimated)

October 23, 2030

Study Completion (Estimated)

October 23, 2030

Study Registration Dates

First Submitted

November 13, 2025

First Submitted That Met QC Criteria

January 13, 2026

First Posted (Actual)

January 22, 2026

Study Record Updates

Last Update Posted (Actual)

April 24, 2026

Last Update Submitted That Met QC Criteria

April 23, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTRSC02-008

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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