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

NCT ID NCT04988386

Disease control Sponsor: Eidos Therapeutics, a BridgeBio company Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

389 people

The number who actually took part.

Started

Oct 2021

Expected to finish

May 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 90 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Completed 30 months of the blinded study treatment in Study AG10-301 and the Study AG10-301 Month 30 visit including assessments and procedures. 2. Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures. 3. Female participants of childbearing potential who engage in heterosexual intercourse must agree to use a highly effective method of contraception beginning with enrollment and continuing for 30 days after the last dose of acoramidis. Female participants using oral contraceptives must agree to use an additional birth control method. While not considered highly effective, a double-barrier method is acceptable. A male participant who is sexually active with a female of childbearing potential and has not had a vasectomy must agree to use a double-barrier method of birth control. Exclusion Criteria: 1. Acute myocardial infarction, acute coronary syndrome or coronary revascularization within 90 days prior to Day 1 stroke or transient ischemic attack (TIA) within 90 days prior to Day 1. 2. Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study. 3. Has had a heart and/or liver transplant or is on the heart transplantation list within the year prior to Day 1 4. Has had implantation of a cardiac mechanical assist device (CMAD) or is scheduled for implantation of a CMAD 5. Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301. 6. Has estimated glomerular filtration rate (eGFR) by modification of diet for renal disease (MDRD) formula \< 15 mL/min/1.73 m2 at Month 27 of Study AG10-301 or at any subsequent central lab value prior to Day 1. 7. Known hypersensitivity to acoramidis, its metabolites, or formulation excipients. 8. At the end of Study AG10-301 or at Day 1 of Study AG10-304 (or any time during the study), participant is on prohibited medication. 9. Females who are pregnant or breastfeeding. A negative urine pregnancy test at the Day 1 visit and at each study visit are required for female participants of childbearing potential. 10. In the judgment of the Investigator or Medical Monitor, has any clinically important ongoing medical condition or laboratory abnormality or condition that might jeopardize the participant's safety, increase their risk from participation, or interfere with the study. 11. Participation in another interventional clinical trial (with the exception of Study AG10-301) within 30 days prior to dosing. Participation in observational and/or registry studies should be discussed with the Medical Monitor. 12. Has any condition that in the opinion of the Investigator or Medical Monitor would preclude compliance with the study protocol such as a history of substance abuse, alcoholism, or a psychiatric condition.

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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.

Amyloid cardiomyopathy, transthyretin-related

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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