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Study to Evaluate the Safety and Effectiveness of Vutrisiran in Patients with Transthyretin Amyloidosis and Heart Disease

Verified siteRegistered 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, often referred to as ATTR Amyloidosis with Cardiomyopathy. This is a disease where abnormal proteins build up in the heart, affecting its function. The study will use a treatment called Vutrisiran, which is a solution for injection. Vutrisiran is also known by its code name ALN-TTRSC02. The purpose of the study is to gather information on the safety of Vutrisiran in patients who have this condition.

Participants in the study will receive Vutrisiran through an injection under the skin. The study will observe patients who are continuing their treatment with Vutrisiran or those who are switching from another medication called Patisiran. The study will last for a period of time, during which the safety of the treatment will be closely monitored. Patients will be asked to take vitamin A supplements as part of the study protocol.

The study will track how often any side effects occur and will also look at changes in heart-related health measures. These include changes in a questionnaire score that assesses heart failure symptoms, changes in heart function class, and levels of certain proteins in the blood that indicate heart stress. The study will also monitor overall survival and any heart-related hospital visits. The goal is to better understand how Vutrisiran affects patients with ATTR Amyloidosis with Cardiomyopathy over an extended period.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing the required parent studies or the 24-month period in the vutrisiran parent study.

    Written informed consent is necessary to proceed.

  2. Step 2

    Medication administration

    Receive Amvuttra (vutrisiran) 25 mg solution for injection.

    The medication is administered through a subcutaneous injection, which means it is injected under the skin.

  3. Step 3

    Supplementation

    Adhere to vitamin A supplementation as outlined in the study protocol.

  4. Step 4

    Monitoring and assessments

    Regular monitoring for any adverse events is conducted to ensure safety.

    Assessments include changes from baseline in heart-related health scores and biomarkers.

    Monitoring of overall health outcomes, including mortality and cardiovascular events, is performed.

  5. Step 5

    Study duration

    The study is expected to continue until June 1, 2027.

    Participation involves ongoing assessments and adherence to the study protocol throughout this period.

Who can join the trial?

5 criteria

  • Patients must have **ATTR amyloidosis with cardiomyopathy**. This is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients should be participating in specific studies related to the condition, such as the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014, or have completed a 24-month period in the vutrisiran parent study ALN-TTRSC02-003.
  • Patients must be willing to take **vitamin A supplements** as part of the study requirements.
  • The patient or their legal guardian must be able to understand the study requirements and agree to participate by signing a written consent form.
  • Both male and female patients are eligible to participate.

Who cannot join the trial?

5 criteria

  • Patients who have a different type of amyloidosis other than Transthyretin Amyloidosis with Cardiomyopathy. This is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients who are not in the specified age range for the study. The study is open to certain age groups only.
  • Patients who are not able to continue using the study medication, vutrisiran, or who have not switched from another medication called patisiran.
  • Patients who are not able to provide informed consent, which means they cannot understand the study and agree to participate.
  • Patients who are part of a vulnerable population, meaning they might need special protection or care.
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Investigated drugs

  • Vutrisiran

    is a medication being studied for its safety and effectiveness in patients with a condition called transthyretin amyloidosis with cardiomyopathy. This condition affects the heart and is caused by abnormal proteins. Vutrisiran is designed to help reduce the production of these abnormal proteins, potentially improving heart function and symptoms in affected patients.

  • Patisiran

    is another medication used in the study. It is also aimed at treating transthyretin amyloidosis by reducing the levels of abnormal proteins in the body. Some patients in the trial may have switched from using patisiran to vutrisiran to see if there are differences in safety or effectiveness between the two treatments.

What is already known about the treatment

Vutrisiran – Vutrisiran is administered through subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials for its safety and 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 – 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. The condition progresses as the amyloid deposits increase, further impairing heart function. It is often associated with genetic mutations that affect the transthyretin protein. The disease primarily affects older adults and can significantly impact quality of life.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-518343-38-00Protocol codeALN-TTRSC02-007Estimated enrolment734 patientsSponsorAlnylam Pharmaceuticals Inc.

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