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
Centre Hospitalier Universitaire Rouen Rouen France
University Hospital Ostrava Ostrava Czechia
Karolinska University Hospital Solna Sweden
Semmelweis University Budapest Hungary
Centre Hospitalier Universitaire De Nantes Nantes France
Hopital Beaujon Clichy France
Klinikum Wels-Grieskirchen GmbH Wels Austria
Otto Von Guericke Universitaet Magdeburg Magdeburg Germany
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Hospital Son Llatzer Palma Spain
Uniwersytecki Szpital Kliniczny W Poznaniu Poznan Poland
Centre Hospitalier Regional De La Citadelle Liege Belgium
Instytut Centrum Zdrowia Matki Polki Lodz Poland
AZ Sint-Lucas & Volkskliniek Gent Belgium
A.O. Krankenhaus St. Josef Braunau GmbH Braunau Am Inn Austria
Unidade Local De Saude Do Alto Ave E.P.E. Guimaraes Portugal
Azorg Aalst Belgium
Cofbsnpow Ujyhhixrwrzpmu Sqtmsqand Woluwe-Saint-Lambert Belgium
Axofcku Oavapfujlrv dl Pfyvhl Padua Italy
Hfsqygnebzpw Hpyzvrxq Athens Greece
Nqm Lpj Gzxssbvomf Rpytsf Mnenp Gdzp St. Poelten Austria
Ugxyopkhcr Gpmkfzy Heqgufqn Ou Tspfpedrilbl Aivlr Thessaloniki Greece
Hvsadoyj Vayu dcumbexa Barcelona Spain
Hxazghfp Ulspwrffojhuh dr A Cpkvys A Coruna Galicia Spain
Ucfihqyimz Hkzfceew Cvbxtpj Cologne Germany
Ohmkas Ultdflamof Hwffooea Odense Denmark
Unbmrxlmzrexybelbvqom Ebtkplyk Awb Erlangen Germany
Fifpcngnfp Igmgs Cu Gmughs Oxwcdsbp Mnrcbcgo Pjuaeumesxh Milan Italy
Fsecgijdfr Iujtu Pmwwhvxksou Sdn Mhowwm Pavia Italy
Akfgayvurp Pebanmwu Hvpbrgco Dj Mtnvpghgz Marseille France
Renrwy Sdgdk Swlrkn Uxoakcugfjacmfqtnul Lund Sweden
Uayrzxabxzzi Mtmvzhj Clqnhur Uzfoomn Utrecht The Netherlands
Alpuvdbnb Heuxyufv Athens Greece
Aikriawv Uxczdkkusi Hnigfhzq Lorenskog Norway
Heabudbl Cnsfur Dr Bfcsobtnn Barcelona Spain
Cxnpqzt Uzpnunbmmiulbluoumee Bhxqsg Kku Berlin Germany
Epklmjb Uxwlrkdaeggr Mkrwkwd Cnxifhq Rpuiixcoj (qcimlvi Miq Rotterdam The Netherlands
Cjvfbd Hpbcsmkwlfc Rnxmjdhn Uymyzrhzjtter Dx Thdnm Tours France
Uqnzsupjyo Cphemjkl Hhtvwxxf Vvizhw Dg Lb Aupjvcmz Murcia Spain
Cbjdpx Hkutmlnzety Ugkvzbjydbbbh Dg Mkbdrzfdrpc Montpellier France
Ujkeyqzexgihwgnxm Ptddgrmhknq Dvzrs Myxesu Ancona Italy
Ahkehzv Orjzyzaocqk Unczjixabqstg Cjfvcotvxzno Dmupr Sjpjcc E Dygks Skuoyyx Dx Txwixi Turin Italy
Ukhastnaxkspngntqsvqu Mqevvlor Amw Munster Germany
Kjtaco Ftaxdmxry Vienna Austria
Jnavv Zqovurpvdf Hasselt Belgium
Gylxbpqudrlgeel Apvwmvifi Antwerp Belgium
Aqddzsx Ogsuhxbrafx Uivclvvkxjkxv Fjhaslqv Io Dv Nnocbv Naples Italy
Ursgmltxravztunmnuxbf Hcvbbbyohi Asf Heidelberg Germany
Krihxump dlx Udingutzaysh Mtsskqha Ajw Munich Germany
Ulxhagsvnuxkhptdrxlgt Wgnxrpekz Ayi Wuerzburg Germany
Rilhku Mlfppwmxhfw Aarhus Denmark
Vcevecjoj Fhhccglk Nqcbtqjxi V Pyzvf Prague Czechia
Ugzozlpipz Hntziihs Mqgwobczrv Maastricht The Netherlands
Uxricwwiuoap Ldvdjmv Leipzig Germany
Cplikj Hkopmkuobkp Umvohidovvijr Du Rgggoz Rennes France
Rodrtswosczekj Copenhagen Denmark
Axhigrx Uiwxw Siobwkals Ldoomh Dh Bvjhizn Bologna Italy
Unvkpirktv Or Aammszz Edegem Belgium
Uivgvdncxm Dhmaw Sqevv Dz Rmzd Lp Svqfkrom Rome Italy
Izkzzrcim Fly Ckmwzjpd Acw Eqzgyxgoexqq Mxtzwuxr Prague Czechia
Uigcbmwbmlby Zhbaotyhgr Goct Gent Belgium
Uvjtvoicjyxz Mjbxdda Cxxxfvh Gvyvzpsup Groningen The Netherlands
Hhnutdle Uawlrtoidrkrv Pzpwut Dx Hqqiuf Dt Mzrhbvlmett Majadahonda Spain
Uhulqblufb Dnaxv Sjxsy Do Buizwgq Brescia Italy
Atidhqyu Zepempmvjb Dnifx Roeselare Belgium
Qenhp Sjdimp Cekcarpco Hkbrpxkv &ewvbbh Sdsfhriekmh Uzoknvbdhy Hsvhckps &bccswr Vqrzmfk Gafvzucmjcagcuwnlo Gothenburg Sweden
Fablfauyd Plmy Lq Iometxdoiusgz Bkojfweng Ddu Hynfxpmx Upcbpwyilbdrh Lq Ppk Madrid Spain
Sshka Waeq Wzejii Gqxkcugtldephiivtz Vienna Austria
Cmxclzcp Hwpqpsrviwar Uiagldxddvztb Dl Vqqf Vigo Spain
Hzhyomfe Urcpwyormsemrb Sbotoohbfd &fvxwpg Hgktjya dq Hktgkvlnhje STRASBOURG, Alsace France
Ouktcmsl Vktt Foloz Lslka Lecce Italy
Vasfzrtkgeypsgqv Ueccz Siruymyvp A Ciwylzqq Cvqeqs akvs Kosice Slovakia
Czs Do Mxyvtfxxxc Fort De France France
Ckbdis Hqptctsmdzh Uidnfhodfazel Du Dojlg Dijon France
Chyasppep Zdgmoecxej Srbmbtzzd Eindhoven The Netherlands
Hgaxzanj Uilnazuumdza Apgif Dp Vukpdrps Dz Le Ggrthmln Tyhgikjkzfg Df Lrhyjv Lleida Spain
Fgztdpaw Nxyrwlkxg U Sl Axmv V Babo Brno-Stred Czechia
Zqvjrdghhr Olvu Lzuyvhe Genk Belgium
Hvvgm Mfszx Of Rajijgz Ha Aalesund Norway
Asyfogm Uplfa Suwfmftni Lzapvs Dtkii Rfgyrlf Faenza Italy
Bvufygltnxurfm Asgyec Saqfywuqch Balatonfured Hungary
Cjlvga Hjzlcflthfa Uqepqwimdryxl Rwdnb Reims France
Fxcapgichh Tuyuzhd Gbvvalja Mbtlxjhtdv Pisa Italy
Rxzczo Owscojoofbbxya Linkoping Sweden
Naxhvrzq Igyeztub Kiygfwgokbz Sifvpyw Kavcgkurs Wdvsjgbktgbb Psekmhatb Iqmdaink Bgjgcjfu Warsaw Poland
Hnybupfo Uzrtsbtkjksjl Dr Jkxy Jaen Spain
Hthfbyaf Urbpuyfmqgife Bdyletk Bilbao Spain
Oiovcdnhe veq Tsomft Dendermonde Belgium
Uhtczzi Lsvpi Dl Skdqq Db Thivblqdrzzlsb E Axuz Djezo Enkhli Vila Real Portugal
Ilzjjzyskp Iwxhxe Da Uyotbut Poyjhs Buse Cfrnqadrkmgmblf Nibkiaj Slgzvmrhg Cluj Napoca Romania
Ixwfbhgdxn Db Urktpnr Pvodmh Bagf Ccxjbkafvglregs Pawbq Dsd Cf Cn Imudoum 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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