Heart drug acoramidis tested for Long-Term safety in rare amyloidosis

NCT ID NCT04988386

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study is checking the long-term safety of a drug called acoramidis in people with a rare heart condition called transthyretin amyloid cardiomyopathy. About 389 participants who finished a previous 30-month trial will take acoramidis twice daily for up to 5 years. Researchers will track side effects, deaths, and heart function to see if the drug remains safe over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
acoramidis (AG10)
What this could lead to
If successful, this could confirm acoramidis as a safe long-term treatment to slow heart damage and improve survival in people with this rare heart condition.
What could go wrong
This is an extension study focused on safety, not proof of effectiveness. Results may not show clear benefit, and side effects could emerge over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Amyloid Neuropathies, Familial wild type ATTR amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Aarhus Universitets Hospital

    Aarhus N, Palle Juul-Jensens, 8200, Denmark

  • Alexandra General Hospital of Athens

    Athens, Attica, 11528, Greece

  • Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan

    Bruges, West Vlaanderen, 8000, Belgium

  • Azienda Ospedaliero - Universitaria Careggi

    Florence, Florence, 50134, Italy

  • Boston University Medical Center General Clinical Research Unit

    Boston, Massachusetts, 02118, United States

  • Box Hill Hospital

    Box Hill, Victoria, 3128, Australia

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CHUM)

    Montreal, Quebec, H2X 0A9, Canada

  • Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED

    Ribeirão Preto, São Paulo, 14026-900, Brazil

  • Centro Hospitalar Universitário Lisboa Norte EPE- Hospital Santa Maria

    Lisbon, Lisbon District, 1649-035, Portugal

  • Cleveland Clinic Main Campus

    Cleveland, Ohio, 44195, United States

  • Clinica Universidad de Navarra Madrid

    Madrid, Madrid, 28027, Spain

  • Clínica Universidad de Navarra

    Pamplona, Navarre, 31008, Spain

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Complejo Hospitalario Universitario de Santiago (CHUS)

    Santiago de Compostela, La Coruña, 15706, Spain

  • Duke University Health System - Duke Clinic

    Durham, North Carolina, 27710, United States

  • Fakultní Nemocnice u sv. Anny v Brně

    Brno, South Moravian, 656 91, Czechia

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150, Australia

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100, Italy

  • Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica

    Pisa, Pisa, 56124, Italy

  • Hadassah University Hospital Ein Kerem

    Jerusalem, 9112001, Israel

  • Hospital Clínico Universitario de Valencia

    Valencia, Valencia, 46010, Spain

  • Hospital Son Llàtzer

    Palma de Mallorca, Balearic Islands, 7198, Spain

  • Hospital Universitario Puerta de Hierro - Majadahonda

    Majadahonda, Madrid, 28222, Spain

  • Hôpital du Sacré-Coeur de Montréal

    Montreal, Quebec, H4J 1C5, Canada

  • Hôpital régional de Rimouski

    Rimouski, Quebec, G5L 5T1, Canada

  • Institut Klinické a Experimentální Medicíny

    Prague, Prague, 140 21, Czechia

  • Instituto de Cardiologia do Rio Grande do Sul

    Porto Alegre, Rio Grande do Sul, 90620-001, Brazil

  • Instituto do Coração de São Paulo

    São Paulo, São Paulo, 05403-000, Brazil

  • L' Institut de Cardiologie de Montréal

    Montreal, Quebec, H1T 1C8, Canada

  • Laurelton Heart Specialists

    Rosedale, New York, 11422, United States

  • Mater Misericordiae University Hospital

    Dublin, Dublin, 7, Ireland

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905, United States

  • MedStar Medical Group Cardiology at Franklin Square

    Baltimore, Maryland, 21237, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Middlemore Clinical Trials - Middlemore Hospital

    Papatoetoe, Auckland, 2025, New Zealand

  • Montefiore Medical Center at Hutchinson

    The Bronx, New York, 10461, United States

  • National Amyloidosis Centre, Division of Medicine - Royal Free

    London, England, NW3 2PF, United Kingdom

  • New York University Langone Health

    New York, New York, 10016, United States

  • NorthShore University Health System

    Evanston, Illinois, 60201, United States

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Ospedale San Donato

    Arezzo, Arezzo, 52100, Italy

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania, 19104, United States

  • Princess Alexandra Hospital

    Woolloongabba, Queensland, 4102, Australia

  • Providence Sacred Heart Children's Hospital

    Spokane, Washington, 99204, United States

  • Queen Elizabeth II Health Sciences Centre - Halifax Infirmary

    Halifax, Nova Scotia, B3H 3A7, Canada

  • Richmond Pharmacology Ltd

    London, England, SE1 1YR, United Kingdom

  • Roanoke Heart Institute

    Roanoke, Virginia, 24014, United States

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Hobart Hospital

    Hobart, Tasmania, 7000, Australia

  • Saint Boniface Hospital

    Winnipeg, Manitoba, R2H 2A6, Canada

  • Saint Luke's Hospital of Kansas City

    Kansas City, Missouri, 64111, United States

  • Saint Vincent's Hospital Sydney

    Darlinghurst, New South Wales, 2010, Australia

  • Seoul National University Bundang Hospital

    Seongnam-si, Gyeonggi-do, 13620, South Korea

  • Seoul National University Hospital

    Seoul, Gyeonggi-do, 3080, South Korea

  • The Chaim Sheba Medical Center

    Ramat Gan, Tel Aviv, 5265601, Israel

  • Toronto Heart Centre

    Toronto, Ontario, M4P IE4, Canada

  • Universitair Medisch Centrum Groningen

    Groningen, Provincie Groningen, 9713 GZ, Netherlands

  • Universitair Medisch Centrum Utrecht

    Utrecht, Utrecht, 3584 CX, Netherlands

  • University of Chicago Medical Center

    Chicago, Illinois, 60637, United States

  • University of Colorado Hospital - Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • University of Washington School of Medicine

    Seattle, Washington, 98195, United States

  • Vancouver General Hospital

    Vancouver, British Columbia, V5Z 1M9, Canada

  • Virginia Commonwealth University Health System

    Richmond, Virginia, 23219, United States

  • Vseobecna Fakultni Nemocnice v Praze

    Prague, Prague, 128 08, Czechia

  • Waikato Hospital

    Hamilton, Waikato Region, 3240, New Zealand

  • Washington University

    St Louis, Missouri, 63110, United States

  • Ziekenhuis Oost-Limburg

    Genk, Limburg, 3600, Belgium

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