ClinicalTrials.gov record NCT06679946

A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

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

An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

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.

StatusEnrolling by invitationThe study enrols only people the study team invites, for example patients already at a particular clinic. It is not open to the public.

Study typeInterventionalA 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.

PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.

Age18 years old and older

SexAll

LocationArgentina, Austria, Belgium, Brazil, Czechia, Denmark, France, Italy, Netherlands, Norway, Portugal, South Korea, Spain, Sweden, Taiwan, United Kingdom, 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 is an open-label extension Phase 3 study collecting safety, efficacy, and pharmacodynamic data on vutrisiran in people with ATTR amyloidosis with cardiomyopathy. It includes people who continued extended vutrisiran use or who switched from patisiran in specified parent studies. Participants receive vutrisiran 25 mg by subcutaneous injection every 3 months. Safety (adverse events) and cardiac measures are followed for up to 36 months.

What it is studying. The study is testing the long-term safety profile and effects of vutrisiran given every three months in people with transthyretin amyloidosis with cardiomyopathy, and it will collect measures of heart symptoms, heart-failure class, cardiac biomarkers, mortality and cardiovascular events, and changes in serum TTR levels.

What the researchers hope to learn. To obtain safety, efficacy, and pharmacodynamic data for vutrisiran in transthyretin amyloidosis with cardiomyopathy. To track patient-reported heart-failure status (KCCQ), changes in New York Heart Association (NYHA) class, cardiac biomarkers (NT-proBNP and troponin I), all-cause mortality and cardiovascular events, and serum transthyretin (TTR) levels at specified time points.

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 obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: Vutrisiran

Eligibility

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

Inclusion criteria

Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003

Exclusion criteria

Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
Future or current participation in another investigational device or drug study, scheduled to occur during this study
Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

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.

Participants receive vutrisiran 25 mg by subcutaneous injection once every 3 months. They will attend regular study visits with safety monitoring and adverse event reporting for up to 36 months. Visits include questionnaires about symptoms and quality of life (Kansas City Cardiomyopathy Questionnaire), a clinical assessment of NYHA class, and blood tests for cardiac biomarkers (NT‑proBNP, troponin I) and serum TTR (measured at baseline, Month 6, and Month 12). Hospitalizations and urgent heart-failure visits will also be recorded.

How the study is structured. Phase 3, open-label, single-group interventional study. All participants receive vutrisiran 25 mg by subcutaneous injection once every 3 months. There is no masking; outcomes and safety are recorded over follow-up of up to 36 months.

Questions you could ask

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

The registry says participants are enrolled by invitation. Who decides who is invited, and how?
The listed sites are in Argentina, Austria, Belgium, Brazil, Czechia, Denmark, France, Italy, Netherlands, Norway, Portugal, South Korea, Spain, Sweden, Taiwan, United Kingdom, 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 wild-type ATTR amyloidosis

This trial is also listed on this site under ATTRv amyloidosis.

Clinical trials121 trials listed for wild-type ATTR amyloidosisEvery trial the registry lists for wild-type ATTR amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in wild-type ATTR amyloidosis researchLatest monthly update: September 2026. Includes: Frailty and support needs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Dec 3, 2024
Primary completion: Mar 31, 2028 (estimated)
Study completion: Mar 31, 2028 (estimated)
Target enrollment: 700 participants (estimated)
Last updated on registry: Feb 20, 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

Participating sites (87)

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Clinical Trial Site
Phoenix, Arizona, 85054 · United States
Clinical Trial Site
Beverly Hills, California, 90211 · United States
Clinical Trial Site
La Jolla, California, 92037 · United States
Clinical Trial Site
Stanford, California, 94305 · United States
Clinical Trial Site
Washington D.C., District of Columbia, 20010 · United States
Clinical Trial Site
Chicago, Illinois, 60637 · United States
Clinical Trial Site
Glenview, Illinois, 60026 · United States
Clinical Trial Site
Indianapolis, Indiana, 46202 · United States
Clinical Trial Site
Kansas City, Kansas, 66160 · United States
Clinical Trial Site
Baltimore, Maryland, 21287 · United States

Terms used on this page · explanations by MedicalUpdates.ai

pharmacodynamic: What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
study drug: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
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.

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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