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Vutrisiran

Clinical trials are studying Vutrisiran in people with transthyretin amyloidosis, including those with cardiomyopathy and polyneuropathy. These studies look at safety, efficacy, and heart or nerve-related outcomes in Phase 3 trials. Some trials include people who are continuing treatment, while others compare Vutrisiran with placebo or external control groups.

Table of contents

Overview of the clinical research

The trials in this set are studying Vutrisiran in people with transthyretin amyloidosis, a disease where abnormal protein builds up and can harm the heart or nerves.[1][2] All listed studies are Phase 3 trials, which are later-stage studies designed to test how well a treatment works and how safe it is in larger groups of patients.[1][2]

Two trials focus on transthyretin amyloidosis with cardiomyopathy, meaning heart muscle disease.[1][2] Two other studies focus on hereditary transthyretin amyloidosis, including one study of polyneuropathy, which means nerve damage affecting many nerves in the body.[3][4]

Trials in transthyretin amyloidosis with cardiomyopathy

One authorised Phase 3 trial with NCT ID 2024-518318-25-00 is studying Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.[1] The study has 691 planned participants and compares Vutrisiran with 0.9% sodium chloride, which is used here as a placebo-like control.[1]

The main outcome in this trial is a composite outcome, which combines several important events into one result.[1] These events include all-cause mortality, meaning death from any cause, and recurrent cardiovascular events such as hospital stays for heart problems and urgent heart failure visits.[1] The study also looks at a subgroup of patients who were not taking tafamidis at the start of the study, called the Vutrisiran monotherapy subgroup.[1]

A second authorised Phase 3 trial, NCT 2024-518343-38-00, also studies patients with transthyretin amyloidosis with cardiomyopathy.[2] This study plans to enroll 734 patients and focuses on safety, with the main outcome being the frequency of adverse events.[2]

This safety study includes patients who either continued extended use of Vutrisiran or switched from patisiran.[2] That means the trial is looking at safety in people already exposed to related treatment pathways, not only in treatment-naive patients.[2]

Trials in hereditary transthyretin amyloidosis with polyneuropathy

The completed Phase 3 HELIOS-A study, NCT03759379, evaluated Vutrisiran in patients with hereditary transthyretin amyloidosis.[3] It enrolled 135 participants and studied efficacy and safety, with a focus on neurologic impairment.[3]

Its main outcome was the change from baseline in the Modified Neurologic Impairment Score +7, or mNIS+7, compared with the placebo arm of the APOLLO study.[3] Baseline means the starting point before treatment, so this outcome checks whether nerve problems improved or worsened over time.[3]

A separate authorised Phase 3 study, 2025-522544-40-00, is evaluating Vutrisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy.[4] This trial plans to enroll 186 participants and measures change from baseline in mNIS+7 at Month 9 compared with an external placebo group from the APOLLO study.[4]

This design uses an external control group, which means the comparison group comes from another study rather than from the same trial.[4] The trial is focused on whether Vutrisiran can improve or slow nerve damage in people with hereditary disease.[4]

What the trials measure

The heart trials measure outcomes that matter to patients, such as death, hospitalisation, and urgent visits for heart failure.[1] These outcomes show whether treatment may help people live longer and avoid serious heart-related events.[1]

The safety study measures adverse events, meaning any unwanted medical problems seen during the trial.[2] Safety data are important because they help researchers understand how people respond when treatment is used over time.[2]

The nerve studies measure changes in mNIS+7, a score that tracks neurologic impairment.[3][4] Lower or improved scores can suggest less nerve damage, while higher scores can suggest worsening disease.[3][4]

Who may participate

People with ATTR amyloidosis with cardiomyopathy may be eligible for the two heart studies.[1][2] One of these trials includes patients who are continuing Vutrisiran or who switched from patisiran, so prior treatment history matters in that study.[2]

People with hereditary transthyretin amyloidosis may be eligible for the completed HELIOS-A study, and people with hereditary transthyretin-mediated amyloidosis with polyneuropathy may be eligible for the authorised TRITON-PN study.[3][4] These trials are aimed at patients whose disease affects the nerves, not only the heart.[3][4]

Study design and trial phase

All four studies are interventional, which means researchers are giving a treatment and observing what happens.[1][2][3][4] Each one is a Phase 3 trial, showing that the research is in a later stage and is designed to provide stronger evidence about benefit and safety.[1][2][3][4]

Two studies are authorised and ongoing, one study is completed, and one authorised study is focused on nerve disease with a month 9 endpoint.[1][2][3][4] Together, these trials show that Vutrisiran is being studied for both heart-related and nerve-related forms of transthyretin amyloidosis.[1][2][3][4]

Trial ID Phase Condition studied Status Enrollment
2024-518318-25-00 Phase 3 ATTR amyloidosis with cardiomyopathy Authorised 691
2024-518343-38-00 Phase 3 ATTR amyloidosis with cardiomyopathy Authorised 734
NCT03759379 Phase 3 Hereditary transthyretin amyloidosis Completed 135
2025-522544-40-00 Phase 3 Hereditary transthyretin-mediated amyloidosis with polyneuropathy Authorised 186

FAQ

  • What conditions are being studied in Vutrisiran trials? The trials in this data set study transthyretin amyloidosis with cardiomyopathy and hereditary transthyretin amyloidosis with polyneuropathy. These are serious diseases that can affect the heart, nerves, or both.
  • Who can take part in these trials? People with ATTR amyloidosis with cardiomyopathy may be included in the heart trials, and people with hereditary transthyretin amyloidosis may be included in the nerve trial. One study also includes patients who were already using Vutrisiran or who switched from patisiran.
  • What phase are the Vutrisiran trials? All trials listed here are Phase 3 studies. Phase 3 trials are larger studies that test how well a treatment works and how safe it is.
  • What are the main outcomes being measured? The studies measure outcomes such as death, hospital stays for heart problems, urgent heart failure visits, adverse events, and changes in neurologic scores. These outcomes help show whether the treatment helps and whether it is safe.
  • Are these trials still ongoing? Some trials are authorised, which means they are approved to run, and one trial is completed. The authorised trials are still in the study stage, while the completed trial already finished collecting its main results.

Ongoing Clinical Trials on Vutrisiran

  • A study to evaluate the safety and effectiveness of nucresiran and vutrisiran in patients with hereditary transthyretin amyloidosis with polyneuropathy

    Recruiting

    3 1 1 1
    Investigated drugs:
    Cyprus France Germany Greece Ireland Italy +3

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

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark France Italy +5

  • Study of Vutrisiran for Patients with Transthyretin Amyloidosis and Heart Disease

    Not recruiting

    3 1 1
    Investigated drugs:
    Austria Belgium Croatia Czechia Denmark France +10

  • Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Cyprus Italy The Netherlands Portugal +1

Glossary

  • Transthyretin amyloidosis: A disease where abnormal protein builds up in the body and can damage organs.
  • Cardiomyopathy: A disease of the heart muscle that can make the heart work less well.
  • Polyneuropathy: Damage to many nerves, often causing numbness, pain, weakness, or trouble walking.
  • Hereditary: Passed down through families through genes.
  • Phase 3: A late stage of clinical research with more participants, used to test how well a treatment works and how safe it is.
  • Interventional study: A trial where researchers give a treatment or compare treatments to see what happens.
  • Placebo: An inactive treatment used for comparison in a trial.
  • External control group: A comparison group taken from another study rather than from the same trial.
  • Adverse events: Unwanted medical problems that happen during a study.
  • Composite outcome: A main result that combines several events into one measure, such as death and hospital visits.
  • Neurologic impairment: Problems caused by nerve damage, such as weakness, numbness, or poor coordination.
  • mNIS+7: A score used to measure nerve damage and how much it changes over time.

References

  1. https://clinicaltrials.gov/study/2024-518318-25-00
  2. https://clinicaltrials.gov/study/2024-518343-38-00
  3. https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-vutrisiran-and-patisiran-for-patients-with-hereditary-transthyretin-amyloidosis/
  4. https://clinicaltrials.gov/study/2025-522544-40-00