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

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 18 years and older
• All sexes
• No healthy volunteers
• Adult, Older adult

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.

Trial locations and contacts

27 sites in 3 countries. Contact the study team or a site near you to ask about taking part.

About this study

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

Conditions

Transthyretin Amyloidosis With Cardiomyopathy

Keywords

ATTR-CM, Transthyretin, Amyloidosis, ATTR, Cardiomyopathy

Medical subject headings

Amyloidosis, Cardiomyopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

What is being measured

The primary outcome is the main question the study is designed to answer.

1. 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.

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

2. 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.

   Measured: From time of enrollment for up to 5 years

3. 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).

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

4. 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).

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

5. 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."

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

6. 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."

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

7. 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.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted22 Jan 2026
2. Study start9 Jan 2026
3. Primary completion23 Oct 2030 (estimated)
4. Study completion23 Oct 2030 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Other trials for Transthyretin Amyloidosis With CardiomyopathyStudies of the same condition, open and under way.
• More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.