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

Study at a glance

From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

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

ClinicalTrials.gov ID

NCT07358078

Acronym / sponsor ID

DemonsTTRate / ALN-TTRSC02-008

Conditions

Transthyretin Amyloidosis With Cardiomyopathy

Keywords

ATTR-CM, Transthyretin, Amyloidosis, ATTR, Cardiomyopathy

Study type

Observational

Recruitment status

Recruiting

Enrolment

2,000 participants (estimated)

Lead sponsor

Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)

Study start

9 Jan 2026 (actual)

Primary completion

23 Oct 2030 (estimated)

Study completion

23 Oct 2030 (estimated)

First posted

22 Jan 2026 (actual)

Results

Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model

Cohort

Time perspective

Prospective

Number of arms

1

Who may qualify

Ages

18 years and older

Sex

All

Healthy volunteers

Not accepted

Age groups

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

1. 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.

2. 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.

3. 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).

4. 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).

5. 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."

6. 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."

7. 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.

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
• Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

27 sites in 3 countries: see every site and its status.