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.
1Initial 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.
2Regular 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.
3Ongoing 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.
4Health 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 Study?
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 Study?
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
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STRASBOURG, Alsace
France
Want to learn more about this study or check if you can participate? Contact us.
Trial status
Country
Status
Recruitment Start
Austria
Recruiting
07.07.2025
Belgium
Recruiting
07.07.2025
Czechia
Recruiting
07.07.2025
Denmark
Recruiting
07.07.2025
France
Recruiting
07.07.2025
Germany
Recruiting
07.07.2025
Greece
Recruiting
07.07.2025
Hungary
Recruiting
07.07.2025
Ireland
Recruiting
07.07.2025
Italy
Recruiting
07.07.2025
Norway
Recruiting
07.07.2025
Poland
Not yet recruiting
07.07.2025
Portugal
Recruiting
07.07.2025
Romania
Recruiting
07.07.2025
Slovakia
Recruiting
07.07.2025
Spain
Recruiting
07.07.2025
Sweden
Recruiting
07.07.2025
The Netherlands
Recruiting
07.07.2025
Trial locations
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.
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.
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