RecruitingNot Applicable

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

United States2,000 participantsStarted 2026-01-09

Plain-language 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

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

New York Heart Association (NYHA) Class

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

Outpatient Diuretic Intensification

Timeframe: From time of enrollment for up to 5 years

Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score

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

Kansas City Cardiomyopathy Questionnaire (KCCQ)

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

Patient Global Impressions - Change (PGI-C)

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

Patient Global Impressions - Severity (PGI-S);

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

Treatment Satisfaction Questionnaire for Medication version 9 (TSQM-9)

Trial details

NCT IDNCT07358078

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2030-10-23

Contact for this trial

Clinical Trial Information Line

1-877-ALNYLAM clinicaltrials@alnylam.com

View on ClinicalTrials.gov More Transthyretin Amyloidosis With Cardiomyopathy trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

New York Heart Association (NYHA) Class

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

Outpatient Diuretic Intensification

Timeframe: From time of enrollment for up to 5 years

Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Score

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

Kansas City Cardiomyopathy Questionnaire (KCCQ)

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

Patient Global Impressions - Change (PGI-C)

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

Patient Global Impressions - Severity (PGI-S);

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

Treatment Satisfaction Questionnaire for Medication version 9 (TSQM-9)