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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Aarhus Universitets Hospital
Aarhus N, Palle Juul-Jensens, 8200, Denmark
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Alexandra General Hospital of Athens
Athens, Attica, 11528, Greece
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Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan
Bruges, West Vlaanderen, 8000, Belgium
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Azienda Ospedaliero - Universitaria Careggi
Florence, Florence, 50134, Italy
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Boston University Medical Center General Clinical Research Unit
Boston, Massachusetts, 02118, United States
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Box Hill Hospital
Box Hill, Victoria, 3128, Australia
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Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
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Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, H2X 0A9, Canada
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Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED
Ribeirão Preto, São Paulo, 14026-900, Brazil
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Centro Hospitalar Universitário Lisboa Norte EPE- Hospital Santa Maria
Lisbon, Lisbon District, 1649-035, Portugal
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Cleveland Clinic Main Campus
Cleveland, Ohio, 44195, United States
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Clinica Universidad de Navarra Madrid
Madrid, Madrid, 28027, Spain
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Clínica Universidad de Navarra
Pamplona, Navarre, 31008, Spain
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Columbia University Irving Medical Center
New York, New York, 10032, United States
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Complejo Hospitalario Universitario de Santiago (CHUS)
Santiago de Compostela, La Coruña, 15706, Spain
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Duke University Health System - Duke Clinic
Durham, North Carolina, 27710, United States
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Fakultní Nemocnice u sv. Anny v Brně
Brno, South Moravian, 656 91, Czechia
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Fiona Stanley Hospital
Murdoch, Western Australia, 6150, Australia
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Fondazione IRCCS Policlinico San Matteo
Pavia, 27100, Italy
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Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica
Pisa, Pisa, 56124, Italy
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Hadassah University Hospital Ein Kerem
Jerusalem, 9112001, Israel
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Hospital Clínico Universitario de Valencia
Valencia, Valencia, 46010, Spain
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Hospital Son Llàtzer
Palma de Mallorca, Balearic Islands, 7198, Spain
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Hospital Universitario Puerta de Hierro - Majadahonda
Majadahonda, Madrid, 28222, Spain
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Hôpital du Sacré-Coeur de Montréal
Montreal, Quebec, H4J 1C5, Canada
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Hôpital régional de Rimouski
Rimouski, Quebec, G5L 5T1, Canada
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Institut Klinické a Experimentální Medicíny
Prague, Prague, 140 21, Czechia
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Instituto de Cardiologia do Rio Grande do Sul
Porto Alegre, Rio Grande do Sul, 90620-001, Brazil
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Instituto do Coração de São Paulo
São Paulo, São Paulo, 05403-000, Brazil
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L' Institut de Cardiologie de Montréal
Montreal, Quebec, H1T 1C8, Canada
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Laurelton Heart Specialists
Rosedale, New York, 11422, United States
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Mater Misericordiae University Hospital
Dublin, Dublin, 7, Ireland
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Mayo Clinic - Rochester
Rochester, Minnesota, 55905, United States
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MedStar Medical Group Cardiology at Franklin Square
Baltimore, Maryland, 21237, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Middlemore Clinical Trials - Middlemore Hospital
Papatoetoe, Auckland, 2025, New Zealand
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Montefiore Medical Center at Hutchinson
The Bronx, New York, 10461, United States
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National Amyloidosis Centre, Division of Medicine - Royal Free
London, England, NW3 2PF, United Kingdom
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New York University Langone Health
New York, New York, 10016, United States
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NorthShore University Health System
Evanston, Illinois, 60201, United States
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Northwestern University
Chicago, Illinois, 60611, United States
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Oregon Health & Science University
Portland, Oregon, 97239, United States
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Ospedale San Donato
Arezzo, Arezzo, 52100, Italy
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Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104, United States
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Princess Alexandra Hospital
Woolloongabba, Queensland, 4102, Australia
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Providence Sacred Heart Children's Hospital
Spokane, Washington, 99204, United States
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Queen Elizabeth II Health Sciences Centre - Halifax Infirmary
Halifax, Nova Scotia, B3H 3A7, Canada
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Richmond Pharmacology Ltd
London, England, SE1 1YR, United Kingdom
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Roanoke Heart Institute
Roanoke, Virginia, 24014, United States
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Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
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Royal Hobart Hospital
Hobart, Tasmania, 7000, Australia
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Saint Boniface Hospital
Winnipeg, Manitoba, R2H 2A6, Canada
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Saint Luke's Hospital of Kansas City
Kansas City, Missouri, 64111, United States
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Saint Vincent's Hospital Sydney
Darlinghurst, New South Wales, 2010, Australia
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Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, 13620, South Korea
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Seoul National University Hospital
Seoul, Gyeonggi-do, 3080, South Korea
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The Chaim Sheba Medical Center
Ramat Gan, Tel Aviv, 5265601, Israel
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Toronto Heart Centre
Toronto, Ontario, M4P IE4, Canada
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Universitair Medisch Centrum Groningen
Groningen, Provincie Groningen, 9713 GZ, Netherlands
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Universitair Medisch Centrum Utrecht
Utrecht, Utrecht, 3584 CX, Netherlands
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University of Chicago Medical Center
Chicago, Illinois, 60637, United States
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University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
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University of Utah
Salt Lake City, Utah, 84132, United States
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University of Washington School of Medicine
Seattle, Washington, 98195, United States
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Vancouver General Hospital
Vancouver, British Columbia, V5Z 1M9, Canada
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Virginia Commonwealth University Health System
Richmond, Virginia, 23219, United States
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Vseobecna Fakultni Nemocnice v Praze
Prague, Prague, 128 08, Czechia
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Waikato Hospital
Hamilton, Waikato Region, 3240, New Zealand
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Washington University
St Louis, Missouri, 63110, United States
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Ziekenhuis Oost-Limburg
Genk, Limburg, 3600, Belgium
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