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

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What is this study about?

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.

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 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 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 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 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Cliniques Universitaires Saint-Luc Woluwe-Saint-Lambert Belgium
Azienda Ospedaliera di Padova Padua Italy
Hippokration Hospital Athens Greece
Centre Hospitalier Universitaire Rouen Rouen France
Noe LGA Gesundheit Region Mitte GmbH St. Poelten Austria
University General Hospital Of Thessaloniki Ahepa Thessaloniki Greece
Hospital Vall d’Hebron Barcelona Spain
University Hospital Ostrava Ostrava Czechia
Hospital Universitario da A Coruna A Coruna Galicia Spain
Unidade Local De Saúde De Santa Maria, E.P.E. Lisbon Portugal
University Hospital Cologne Cologne Germany
Odense University Hospital Odense Denmark
Universitaetsklinikum Erlangen AöR Erlangen Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Medical University Of Vienna Vienna Austria
Assistance Publique Hopitaux De Marseille Marseille France
Region Skane Skanes Universitetssjukhus Lund Sweden
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Alexandra Hospital Athens Greece
Akershus University Hospital Lorenskog Norway
Hospital Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Semmelweis University Budapest Hungary
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) Rotterdam The Netherlands
Centre Hospitalier Regional Universitaire De Tours Tours France
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Centre Hospitalier Universitaire De Montpellier Montpellier France
Universita’ Politecnica Delle Marche Ancona Italy
Hospital Universitario De Salamanca Salamanca Spain
Azienda Ospedaliera Universitaria Citta’ Della Salute E Della Scienza Di Torino Turin Italy
Universitaetsklinikum Muenster AöR Munster Germany
Jessa Ziekenhuis Hasselt Belgium
Gasthuiszusters Antwerpen Antwerp Belgium
Azienda Ospedaliera Universitaria Federico II Di Napoli Naples Italy
Universitaetsklinikum Heidelberg AöR Heidelberg Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Medical University Of Graz Graz Austria
Klinikum der Universitaet Muenchen AöR Munich Germany
Universitaetsklinikum Wuerzburg AöR Wuerzburg Germany
Region Midtjylland Aarhus Denmark
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
University Hospital Maastricht Maastricht The Netherlands
Universitaet Leipzig Leipzig Germany
Centre Hospitalier Universitaire De Rennes Rennes France
Rigshospitalet Copenhagen Denmark
Azienda Unita Sanitaria Locale Di Bologna Bologna Italy
University Of Antwerp Edegem Belgium
Azienda Ospedaliero Universitaria Careggi Florence Italy
Oncopole Claudius Regaud Toulouse France
Universita Degli Studi Di Roma La Sapienza Rome Italy
Technische Universitaet Dresden Dresden Germany
Katholieke Universiteit te Leuven Leuven Belgium
Institute For Clinical And Experimental Medicine Prague Czechia
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Universitair Ziekenhuis Gent Gent Belgium
Universitair Medisch Centrum Groningen Groningen The Netherlands
Medizinische Hochschule Hannover Hanover Germany
Hopital Beaujon Clichy France
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Bellvitge University Hospital L'hospitalet De Llobregat Spain
Hospital Universitario Y Politecnico La Fe Valencia Spain
Universita Degli Studi Di Brescia Brescia Italy
Algemeen Ziekenhuis Delta Roeselare Belgium
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz Madrid Spain
Stadt Wien Wiener Gesundheitsverbund Vienna Austria
Unidade Local De Saude De Lisboa Ocidental E.P.E. Carnaxide Portugal
Complexo Hospitalario Universitario De Vigo Vigo Spain
Hospital Universitario De Navarra Pamplona Spain
CHU Grenoble Alpes La Tronche France
Otto Von Guericke Universitaet Magdeburg Magdeburg Germany
Hôpitaux Universitaires Strasbourg – Hôpital de Hautepierre STRASBOURG, Alsace France
CHU De Martinique Fort De France France
Centre Hospitalier Universitaire De Dijon Dijon France
Catharina Ziekenhuis Stichting Eindhoven The Netherlands
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Universitaetsmedizin Goettingen Goettingen Germany
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida Lleida Spain
Fakultni Nemocnice U Sv Anny V Brne Brno-Stred Czechia
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu Wroclaw Poland
Uniwersytecki Szpital Kliniczny W Poznaniu Poznan Poland
Ziekenhuis Oost Limburg Genk Belgium
Helse Moere Og Romsdal HF Aalesund Norway
Azienda Unita Sanitaria Locale Della Romagna Faenza Italy
Centre Hospitalier Universitaire Reims Reims France
Fondazione Toscana Gabriele Monasterio Pisa Italy
Region Oestergoetland Linkoping Sweden
Centre Hospitalier Regional De La Citadelle Liege Belgium
Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy Warsaw Poland
Instytut Centrum Zdrowia Matki Polki Lodz Poland
AZ Sint-Lucas & Volkskliniek Gent Belgium
Hospital Universitario De Jaen Jaen Spain
Hospital Universitario Basurto Bilbao Spain
Unidade Local De Saude Do Alto Ave E.P.E. Guimaraes Portugal
Unidade Local De Saude De Tras-Os-Montes E Alto Douro E.P.E. Vila Real Portugal

Other Sites

Site Name City Country Status
Klinikum Wels-Grieskirchen GmbH Wels Austria
Hospital Son Llatzer Palma Spain
A.O. Krankenhaus St. Josef Braunau GmbH Braunau Am Inn Austria
Azorg Aalst Belgium
General Hospital Venizeleio-Pananeio Heraklion Greece
Kocqns Fgysibqcn Vienna Austria
Odlrkdyi Vxrq Ftesk Lodjp Lecce Italy
Virsmfasxcjnosae Uwxny Sfgmieryw A Cbidyyjx Cvjtxs aqeu Kosice Slovakia
Bnbqfypxbtdyek Alhdin Snacanqowo Balatonfured Hungary
Ozuynfmas vnj Tcvbzo Dendermonde Belgium
Inzrdvpevg Ifotns Dw Ufgcbqo Pvnozw Bskb Clrurphrinedcqv Nnsefnv Syxurnpqu Cluj Napoca Romania
Ihasregbvu Dl Utzucyo Pzivoz Bjco Cjumqncivzlekpk Pbxen Dgd Ct Cx Ijmfmke Bucharest Romania

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Recruiting
07.07.2025
Belgium Belgium
Recruiting
07.07.2025
Czechia Czechia
Recruiting
07.07.2025
Denmark Denmark
Recruiting
07.07.2025
France France
Recruiting
07.07.2025
Germany Germany
Recruiting
07.07.2025
Greece Greece
Recruiting
07.07.2025
Hungary Hungary
Recruiting
07.07.2025
Ireland Ireland
Recruiting
07.07.2025
Italy Italy
Recruiting
07.07.2025
Norway Norway
Recruiting
07.07.2025
Poland Poland
Not yet recruiting
07.07.2025
Portugal Portugal
Recruiting
07.07.2025
Romania Romania
Recruiting
07.07.2025
Slovakia Slovakia
Recruiting
07.07.2025
Spain Spain
Recruiting
07.07.2025
Sweden Sweden
Recruiting
07.07.2025
The Netherlands 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.

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 ID:
2024-519917-72-00
Protocol code:
ALN-TTRSC04-003
Trial Phase:
Therapeutic confirmatory (Phase III)

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