Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

Study of Nucresiran in Adults with Transthyretin Amyloidosis with Cardiomyopathy to Evaluate its Effects on Survival and Heart Problems

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

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

This study focuses on Transthyretin Amyloidosis with Cardiomyopathy, a condition where abnormal proteins build up in the heart muscle, affecting its function. The study will test a new medication called Nucresiran (also known as ALN-TTRSC04) compared to placebo to determine if it can reduce deaths and heart-related medical events in patients with this condition.

The medication Nucresiran is given as an injection using a pre-filled syringe. The study aims to evaluate how well this new treatment works in patients who have heart problems related to protein buildup in their heart tissue. Some participants will receive the study medication, while others will receive a placebo.

During the study, doctors will monitor patients' heart health and track any heart-related hospitalizations or urgent care visits. The study will also look at how the treatment affects patients' quality of life and overall survival. Patients may continue taking their regular heart failure medications and other approved treatments for their condition while participating in the study.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit and medication start

    You will start participating in a study comparing nucresiran with placebo (a substance with no medical effect) for treating heart problems caused by ATTR amyloidosis.

    You will receive either nucresiran or placebo as an injection solution in a pre-filled syringe.

    The study will continue until June 2032.

  2. Step 2

    Regular health monitoring

    Your heart health will be regularly monitored throughout the study.

    The medical team will track any heart-related hospitalizations or urgent heart failure visits.

    Your quality of life related to heart condition will be evaluated using special questionnaires.

  3. Step 3

    Ongoing treatment requirements

    You may continue taking your current heart failure medications during the study.

    If you are already taking medications that stabilize TTR protein, you may continue using them.

    All your current medications must remain unchanged for at least 30 days before starting the study.

  4. Step 4

    Health status monitoring

    Your heart health will be monitored through regular checks of NT-proBNP (a blood marker that indicates heart stress).

    The medical team will track your overall health status and any heart-related events throughout the study period.

Who can join the trial?

7 criteria

  • You must be between 18 and 85 years old
  • You must have a confirmed diagnosis of ATTR amyloidosis with cardiomyopathy (a heart condition where abnormal proteins build up in the heart muscle)
  • You must have a history of heart failure with either: - At least one previous hospital stay due to heart failure, or - Clinical signs of heart failure (with or without hospitalization)
  • If you are taking medications for ATTR amyloidosis or heart failure, your treatment must be stable for at least 30 days before screening
  • You must be clinically stable with no heart-related hospitalizations in the 6 weeks before starting the study
  • Your NT-proBNP blood test results (a marker that indicates heart strain) must be: - Between 300 and 8500 ng/L for most patients - Between 600 and 8500 ng/L for patients with atrial fibrillation (irregular heartbeat)
  • You must be able to understand, willing to follow study requirements, and provide written informed consent

Who cannot join the trial?

14 criteria

  • History of heart transplant or being on a waiting list for heart transplant
  • Severe liver disease (serious condition affecting the liver's function)
  • Current treatment with tafamidis or other medications for ATTR amyloidosis
  • Participation in other clinical trials within the last 30 days
  • Severe kidney disease requiring dialysis (machine-based blood filtering)
  • Blood clotting disorders or current use of blood-thinning medications
  • History of severe allergic reactions to similar medications
  • Pregnant women or women planning to become pregnant during the study
  • Active cancer or cancer treatment within the past 3 years
  • Uncontrolled high blood pressure (consistently above 160/100 mmHg)
  • Severe mental health conditions that could affect study participation
  • Drug or alcohol abuse within the past year
  • Life expectancy less than 2 years due to other medical conditions
  • Unable to follow study procedures or attend regular study visits
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

RecruitingNot yet recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Nucresiran is an investigational medication designed to treat ATTR amyloidosis with cardiomyopathy, a condition where abnormal protein deposits affect heart function. This medication works by targeting and reducing the production of a protein called transthyretin (TTR) that can build up in the heart tissue. The treatment aims to reduce deaths from all causes and cardiovascular events in patients with this heart condition.

What is already known about the treatment

Nucresiran - A novel investigational medication administered via injection that is being studied for the treatment of Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM), a rare and progressive heart condition. This drug belongs to the class of RNA-targeting therapeutics and works by reducing the production of transthyretin protein in the liver, which in its mutated or wild-type form can build up in the heart tissue causing damage. Currently undergoing Phase 3 clinical trials, Nucresiran is being evaluated for its potential to reduce mortality and cardiovascular events in patients with ATTR-CM, representing a promising therapeutic approach for this serious cardiac condition.

Investigated diseases

Transthyretin Amyloidosis with Cardiomyopathy - A rare genetic condition where abnormal proteins called transthyretin build up in the heart muscle. These protein deposits make the heart walls stiff and thick, affecting its ability to pump blood effectively. The condition develops gradually over time as more protein accumulates in the heart tissue. The disease causes the heart to work harder to maintain normal blood flow throughout the body. As the condition progresses, it affects the heart's normal rhythm and pumping function.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-519917-72-00Protocol codeALN-TTRSC04-003Estimated enrolment1 316 patientsSponsorAlnylam Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.