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

September 4, 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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Germany
      • Heidelberg, Germany
        • Recruiting
        • Clinical Trial Site
      • Munich, Germany
        • Recruiting
        • Clinical Trial Site
      • Münster, Germany
        • Recruiting
        • Clinical Trial Site
  • Japan
      • Bunkyō City, Japan
        • Recruiting
        • Clinical Trial Site
      • Kumamoto, Japan
        • Recruiting
        • Clinical Trial Site
      • Kurume, Japan
        • Recruiting
        • Clinical Trial Site
      • Matsumoto, Japan
        • Recruiting
        • Clinical Trial Site
      • Sendai, Japan
        • Recruiting
        • Clinical Trial Site
      • Shinjuku, Japan
        • Recruiting
        • Clinical Trial Site
      • Suita, Japan
        • Recruiting
        • Clinical Trial Site
      • Tsukuba, Japan
        • Recruiting
        • Clinical Trial Site
      • Ube, Japan
        • Recruiting
        • Clinical Trial Site
      • Yufu, Japan
        • Recruiting
        • Clinical Trial Site
  • United States
    • Arizona
      • Tucson, Arizona, United States, 85711
        • Recruiting
        • Clinical Trial Site
    • California
      • Beverly Hills, California, United States, 90211
        • Recruiting
        • Clinical Trial Site
    • Florida
      • Naples, Florida, United States, 34102
        • Recruiting
        • Clinical Trial Site
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Clinical Trial Site
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Clinical Trial Site
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Recruiting
        • Clinical Trial Site
    • North Carolina
      • Charlotte, North Carolina, United States, 28270
        • Recruiting
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Clinical Trial Site
    • Tennessee
      • Memphis, Tennessee, United States, 38120
        • Recruiting
        • Clinical Trial Site
    • Texas
      • Dallas, Texas, United States, 75390
        • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Patients with ATTR-CM
Patients with ATTR-CM.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alnylam Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Alnylam Pharmaceuticals

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 9, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 23, 2030

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 23, 2030

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 13, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 13, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 22, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 9, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 4, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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