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Study of Vutrisiran for Patients with Transthyretin Amyloidosis and Heart Disease

Fast replyRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Transthyretin Amyloidosis with Cardiomyopathy. This is a disease where abnormal proteins called amyloids build up in the heart, leading to heart problems. The study is testing a treatment called Vutrisiran, which is given as an injection under the skin. Vutrisiran is a type of medicine that belongs to a group known as nucleic acids, which are designed to target and reduce the production of these harmful proteins in the body.

The purpose of the study is to evaluate how effective Vutrisiran is compared to a placebo in reducing deaths from any cause and heart-related events, such as hospital visits due to heart problems. Participants in the study will receive either Vutrisiran or a placebo and will be monitored over a period of time to see how their condition changes. The study will also look at how Vutrisiran affects the ability to walk over a short distance, quality of life related to heart health, and overall health status.

Throughout the study, participants will undergo regular check-ups and assessments to track their health and any changes in their condition. The study aims to provide valuable information on whether Vutrisiran can be a safe and effective treatment option for people with Transthyretin Amyloidosis with Cardiomyopathy. This research could potentially lead to better management and outcomes for those affected by this challenging condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will be informed about the trial's purpose, which is to evaluate the effectiveness and safety of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.

    The patient will be required to provide written informed consent, confirming their understanding and willingness to comply with the study requirements.

  2. Step 2

    Initial assessment

    The patient will undergo an initial assessment to confirm eligibility, which includes a documented diagnosis of transthyretin amyloidosis with cardiomyopathy and a medical history of heart failure.

    The patient must be clinically stable, with no cardiovascular-related hospitalizations within six weeks prior to randomization.

  3. Step 3

    Randomization

    The patient will be randomly assigned to receive either vutrisiran or a placebo. This process is double-blind, meaning neither the patient nor the researchers will know which treatment the patient is receiving.

  4. Step 4

    Treatment administration

    The patient will receive vutrisiran as a solution for injection, administered subcutaneously. The dosage is 25 mg, provided in a pre-filled syringe.

    The frequency and duration of administration will be determined by the study protocol and communicated to the patient during the trial.

  5. Step 5

    Monitoring and follow-up

    Throughout the trial, the patient will be monitored for any changes in health status, including cardiovascular events and overall mortality.

    Regular assessments will include tests such as the 6-minute walk test and questionnaires to evaluate the impact on quality of life.

  6. Step 6

    Completion of the study

    The study is estimated to conclude by November 21, 2025. Upon completion, the patient's health outcomes will be evaluated to determine the efficacy and safety of vutrisiran.

Who can join the trial?

10 criteria

  • Age between 18 and 85 years.
  • Agree to sign a form to release medical records, if allowed by local rules, to collect information about health status and hospital visits during the study.
  • Have a confirmed diagnosis of **ATTR amyloidosis with cardiomyopathy**, which is a condition affecting the heart.
  • Have a history of **heart failure** with at least one previous hospital stay for heart failure, or show signs of heart failure that need treatment with a **diuretic** (a medicine that helps remove extra fluid from the body).
  • Meet one of these conditions: either have never used **tafamidis** (a medication) and do not plan to start it in the first year of the study, or are already using tafamidis as prescribed for heart issues.
  • Be in a stable health condition with no hospital visits related to heart problems in the last 6 weeks before starting the study.
  • Have a **NT-proBNP** level (a blood test that helps assess heart function) between 300 and 8500 ng/L, or between 600 and 8500 ng/L if you have a type of irregular heartbeat called **atrial fibrillation**.
  • Be able to walk at least 150 meters during a **6-minute walk test** at the start of the study.
  • Have a **Karnofsky performance status** of 60% or higher, which means being able to carry out daily activities with some help.
  • Understand the study requirements and be willing and able to follow them, and provide written consent to participate.

Who cannot join the trial?

8 criteria

  • Patients who have a different type of amyloidosis other than Transthyretin Amyloidosis with Cardiomyopathy cannot participate. Amyloidosis is a condition where abnormal proteins build up in organs and tissues.
  • Patients who are not within the specified age range for the study cannot participate. The age range is determined by the study organizers.
  • Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible to participate.
  • Patients who have a history of drug or alcohol abuse that could affect the study results cannot participate.
  • Patients who have allergies to the study medication or its ingredients cannot participate.
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Investigated drugs

Vutrisiran is a medication being studied for its effectiveness in treating patients with a condition called transthyretin amyloidosis with cardiomyopathy. This condition affects the heart and can lead to serious heart problems. The trial aims to see if Vutrisiran can help reduce the risk of death and cardiovascular events in these patients.

What is already known about the treatment

Vutrisiran – Vutrisiran is administered via subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials for its effectiveness in treating transthyretin amyloidosis with cardiomyopathy, a condition where abnormal protein deposits affect the heart. The medication works by reducing the production of transthyretin protein, which is involved in the disease process. Vutrisiran is classified as an RNA interference therapeutic, which means it uses small RNA molecules to interfere with the production of specific proteins.

Investigated diseases

Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy) – This is a rare disease where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits can cause the heart to become stiff, leading to difficulties in pumping blood effectively. Over time, this can result in symptoms such as fatigue, shortness of breath, and swelling in the legs and abdomen. The condition progresses as the amyloid deposits increase, further impairing heart function. Patients may experience worsening heart failure symptoms as the disease advances. The progression can vary among individuals, with some experiencing more rapid changes in heart function than others.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-518318-25-00Protocol codeALN-TTRSC02-003Estimated enrolment691 patientsSponsorAlnylam Pharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).