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

Other Sites

Site Name City Country Status
Ospedale Vito Fazzi Lecce Lecce Italy
Centre Hospitalier Universitaire Rouen Rouen France
Noe LGA Gesundheit Region Mitte GmbH St. Poelten Austria
Fondazione Toscana Gabriele Monasterio Pisa Italy
Region Oestergoetland Linkoping Sweden
Universitair Ziekenhuis Gent Gent Belgium
Centre Hospitalier Regional De La Citadelle Liege Belgium
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Klinikum Wels-Grieskirchen GmbH Wels Austria
Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy Warsaw Poland
Algemeen Ziekenhuis Delta Roeselare Belgium
Instytut Centrum Zdrowia Matki Polki Lodz Poland
AZ Sint-Lucas & Volkskliniek Gent Belgium
Hospital Universitario De Jaen Jaen Spain
Hospital Universitario Basurto Bilbao Spain
A.O. Krankenhaus St. Josef Braunau GmbH Braunau Am Inn Austria
Unidade Local De Saude Do Alto Ave E.P.E. Guimaraes Portugal
O.L.Vrouw van Troost Dendermonde Belgium
Unidade Local De Saude De Tras-Os-Montes E Alto Douro E.P.E. Vila Real Portugal
Institutul De Urgenta Pentru Boli Cardiovasculare Prof. Dr. C. C. Iliescu Bucharest Romania
Institutul Inimii De Urgenta Pentru Boli Cardiovasculare Niculae Stancioiu Cluj Napoca Romania
Azienda Ospedaliera di Padova Padua Italy
Hippokration Hospital Athens Greece
University General Hospital Of Thessaloniki Ahepa Thessaloniki Greece
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
Region Skane Skanes Universitetssjukhus Lund Sweden
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Alexandra Hospital Athens Greece
Hospital Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Catharina Ziekenhuis Stichting Eindhoven The Netherlands
Karolinska University Hospital Solna Sweden
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Semmelweis University Budapest Hungary
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Centre Hospitalier Universitaire De Montpellier Montpellier France
Universita’ Politecnica Delle Marche Ancona Italy
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida Lleida Spain
Hospital Son Llatzer Palma Spain
Klinik Favoriten Vienna Austria
Jessa Ziekenhuis Hasselt Belgium
Gasthuiszusters Antwerpen Antwerp Belgium
Fakultni Nemocnice U Sv Anny V Brne Brno-Stred Czechia
Centre Hospitalier Universitaire De Nantes Nantes France
Region Midtjylland Aarhus Denmark
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Uniwersytecki Szpital Kliniczny W Poznaniu Poznan Poland
Centre Hospitalier Universitaire De Rennes Rennes France
Ziekenhuis Oost Limburg Genk Belgium
Rigshospitalet Copenhagen Denmark
Azienda Unita Sanitaria Locale Della Romagna Faenza Italy
Hopital Beaujon Clichy France
Universita Degli Studi Di Brescia Brescia Italy
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Stadt Wien Wiener Gesundheitsverbund Vienna Austria
University Hospital Ostrava Ostrava Czechia
Vychodoslovensky Ustav Srdcovych A Cievnych Chorob a.s. Kosice Slovakia
CHU De Martinique Fort De France France
Otto Von Guericke Universitaet Magdeburg Magdeburg Germany
Azorg Aalst Belgium
Conlssoez Udohuazayyobia Sjbwjzysg Woluwe-Saint-Lambert Belgium
Crxrjq Hoapdrqaloq Ucauszyiiiaof Rplwx Reims France
Ijgdylurc Fbm Cmnlqfeb Aqr Eyhkgcjloeyc Mgpnnkba Prague Czechia
Umtuidaoqmts Mxgdtyf Cpgljpo Ghocgylwv Groningen The Netherlands
Cjyvkqzi Hwruvdlwjkqz Ugsgcqcnubvff Dh Vlog Vigo Spain
Uhdnsxxsto Hlqvhqev Cyhldeu Cologne Germany
Cjhtju Hoktgepeylg Ubrbswmmmfjix Dr Diinr Dijon France
Abcqxtwbax Pqdnvacw Hpfsoldu Dn Mjbnnmhmq Marseille France
Atwluocf Umhrioxoqx Hcnhklnc Lorenskog Norway
Evazygd Urlqnklffaks Moygqgf Cqdhmej Rgavwcsxf (pfhtswq Mii Rotterdam The Netherlands
Cjznqp Hrqosonwhdw Rdwvlhyc Ubixtsorzuqvh Dv Timdi Tours France
Aaliekn Ovjdfcraymn Ughpkklnafevg Cxoelatfdhaf Ddimv Sgabwg E Dybzp Sclrwya Dk Tfrqod Turin Italy
Unlzxaldvlntkljbdaqur Mwdadsbc Akl Munster Germany
Kwzxpzcb dwk Ujjejjaymorf Mnjgsqxa Ajo Munich Germany
Ugupotoqtwvtjgchrufle Wugfglczy Asb Wuerzburg Germany
Apcoonw Utwcs Sptqzjuzu Lwzrex Df Bdacveg Bologna Italy
Hqydy Metyz On Reuqzvl Hz Aalesund Norway
Bnkfskshzmnuzz Augsfv Swabsgwujb Balatonfured Hungary
Umpqudikuf Ot Armmojd Edegem Belgium
Uszvvxgkmi Dpndu Slkko Do Rsrm Lp Sgudeazy Rome Italy
Flfyuljxv Peow Lb Ilhbwlerhvesm Bjgzunxrr Dbm Hcracagc Umtzqbkzvmhae Lb Pxy Madrid Spain
Hdzbnlsu Vhqn dnvtvnen Barcelona Spain
Hltzhkqp Uipicmwkgwsso dl A Cdphep A Coruna Galicia Spain
Hvgtiavn Unvczsddbrheob Sbogfhggdv &sjagcj Hyawbqz dv Hsjropbfvvo 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 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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