ClinicalTrials.gov record NCT07358078

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.

Age18 years old and older

SexAll

LocationGermany, Japan, United States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational study follows adults with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) who are starting vutrisiran or another approved ATTR-CM therapy. Sites will invite eligible patients and follow them for up to 5 years. The study collects clinical measures, patient questionnaires, treatment patterns, and healthcare use, and compares long-term outcomes between vutrisiran and other approved treatments.

What it is studying. The study is trying to describe clinical characteristics and treatment patterns for adults with ATTR-CM treated in routine care, measure health-related quality of life and healthcare resource use, and compare long-term outcomes for patients starting vutrisiran versus other approved ATTR-CM therapies.

What the researchers hope to learn. To describe the clinical characteristics of adults with ATTR-CM treated with vutrisiran in routine care; to describe treatment patterns; to assess health-related quality of life and healthcare resource use; and to compare long-term outcomes of vutrisiran versus other approved ATTR-CM treatments.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Each participating site will invite eligible patients and follow them for up to 5 years (or until withdrawal, loss to follow-up, death, or study end). The study will collect routine clinical information such as New York Heart Association (NYHA) heart‑failure class, any outpatient diuretic changes, the ATTR‑CM treatment started (vutrisiran or another approved therapy), and healthcare use. You may be asked to complete questionnaires about symptoms and quality of life when available (Norfolk QOL‑DN, Kansas City Cardiomyopathy Questionnaire, Patient Global Impression scales, and TSQM‑9). The registry does not list any invasive procedures (for example, biopsies or lumbar punctures) or other extra tests beyond collecting clinical information and these questionnaires.

How the study is structured. This is a prospective observational cohort study. Patients who are initiating vutrisiran or another approved ATTR-CM treatment are followed by their usual care teams for up to 5 years. The study collects clinical status (for example New York Heart Association class and outpatient diuretic changes), patient-reported questionnaires when available (Norfolk QOL‑DN, KCCQ, PGI‑C, PGI‑S, TSQM‑9), and information on healthcare use. The study does not assign treatments; it records data from routine clinical care.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The registry lists this study under Transthyretin Amyloidosis With Cardiomyopathy. Does it apply to my diagnosis?
This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
The listed sites are in Germany, Japan, United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on ATTRv amyloidosis

This trial is also listed on this site under wild-type ATTR amyloidosis.

Clinical trials106 trials listed for ATTRv amyloidosisEvery trial the registry lists for ATTRv amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in ATTRv amyloidosis researchLatest monthly update: September 2026. Includes: Gene-silencing drugs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Jan 9, 2026
Primary completion: Oct 23, 2030 (estimated)
Study completion: Oct 23, 2030 (estimated)
Target enrollment: 2000 participants (estimated)
Last updated on registry: Oct 9, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Alnylam Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Clinical Trial Information Line 📞 1-877-ALNYLAM ✉ clinicaltrials@alnylam.com
Clinical Trial Information Line 📞 1-877-256-9526 ✉ clinicaltrials@alnylam.com

Participating sites (27)

Filter by country:

Clinical Trial SiteRecruiting
Tucson, Arizona, 85711 · United States
Clinical Trial SiteRecruiting
Beverly Hills, California, 90211 · United States
Clinical Trial SiteRecruiting
La Jolla, California, 92037 · United States
Clinical Trial SiteRecruiting
Naples, Florida, 34102 · United States
Clinical Trial SiteRecruiting
Honolulu, Hawaii, 96813 · United States
Clinical Trial SiteRecruiting
Kansas City, Kansas, 66160 · United States
Clinical Trial SiteRecruiting
Boston, Massachusetts, 02215 · United States
Clinical Trial SiteRecruiting
St Louis, Missouri, 63110 · United States
Clinical Trial SiteRecruiting
New Brunswick, New Jersey, 08901 · United States
Clinical Trial SiteRecruiting
Charlotte, North Carolina, 28270 · United States

Terms used on this page · explanations by MedicalUpdates.ai

interventional: A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
follow-up: The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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