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New drug aims to stop rare genetic disease before it strikes

NCT ID NCT06563895

Prevention 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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Aug 05, 2026 · Updated 4 times

Summary

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis is designed to stabilize the TTR protein and stop those plaques from forming. If successful, this could be the first preventive treatment for people at genetic risk.

What this could mean

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

Active substance
acoramidis (a drug that stabilizes the TTR protein to prevent amyloid buildup)
What this could lead to
If it works, this could be the first treatment to prevent or delay the onset of transthyretin amyloidosis in people with a genetic risk.
What could go wrong
This is a large phase 3 trial, but it's still experimental. The drug may not prevent the disease as hoped, and side effects are possible. Results will take years.

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

About 587 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2025

Expected to finish

Dec 2032

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 75 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.

Key Inclusion Criteria: * Male or female ≥ 18 to ≤ 75 years of age inclusive. * Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or all other pathogenic TTR variants). * Participant's age is within 10 years younger than or older than PADO. Key Exclusion Criteria: * Evidence of ATTR-CM or ATTR-PN. * Current or past (within last 1 to 12 months, depending on specific agent) treatment with other TTR modifying therapies. * Contraindication to or inability to undergo cardiac magnetic resonance testing. * Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy * Other diseases or conditions such has cancer within 5 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV. * Major surgery within the past 3 months or planned during the next 12 months. * Known hypersensitivity to acoramidis.

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Conditions

The condition(s) this trial relates to.

Alzheimer disease Amyloid Neuropathies, Familial amyloidosis cardiomyopathy familial amyloid neuropathy heart disorder polyneuropathy restrictive cardiomyopathy wild type ATTR amyloidosis ATTRV30M amyloidosis prevention target

As listed by the trial registrant

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

Amyloid cardiomyopathy Amyloidosis Cardiomyopathies Heart diseases Polyneuropathies Transthyretin amyloidosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    104 sites in 24 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • AP-HP Hopital Bicetre

    RECRUITING

    Le Kremlin-Bicêtre, 94270, France

  • AP-HP Hopital Henri Mondor

    RECRUITING

    Créteil, 94000, France

  • Aarhus University Hospital

    RECRUITING

    Aarhus, 8200, Denmark

  • Azienda Ospedaliero Universitaria Policlinico Umberto I

    RECRUITING

    Roma, 00161, Italy

  • Azienda Ospedaliero-Universitaria Careggi

    RECRUITING

    Florence, Italy

  • Azienda Ospedaliero-Universitaria Sant'Andrea

    RECRUITING

    Rome, 00189, Italy

  • Azienda Ospedaliero-Universitaria di Bologna IRCCS Policlinico di S.Orsola

    RECRUITING

    Bologna, 40138, Italy

  • Boston University (BU) School of Medicine

    RECRUITING

    Boston, Massachusetts, 02118, United States

  • Brigham and Women's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • CAPED - Centro Avancado de Pesquisa, Estudos e Diagnostico

    RECRUITING

    Ribeirão Preto, 14026-900, Brazil

  • CHU Bordeaux - Hopital Pellegrin

    RECRUITING

    Bordeaux, 33076, France

  • CHU Rennes

    RECRUITING

    Rennes, 35000, France

  • CHU de Fort de France

    RECRUITING

    Fort-de-France, 97261, Martinique

  • CHU de Toulouse - Hopital Rangueil

    RECRUITING

    Toulouse, 31400, France

  • Cardiopulmonar da Bahia S.A. (Hospital Cardio Pulmonar)

    RECRUITING

    Salvador, 40170-130, Brazil

  • Changhua Christian Hospital - Taiwan

    RECRUITING

    Changhua, 500, Taiwan

  • Charite Universitaetsmedizin Berlin

    RECRUITING

    Berlin, 10117, Germany

  • Cleveland Clinic

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Cleveland Clinic Florida

    RECRUITING

    Weston, Florida, 33331, United States

  • Columbia University Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Emory University School of Medicine

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Erasmus MC

    RECRUITING

    Rotterdam, 3015 GD, Netherlands

  • Fiona Stanley Hospital

    RECRUITING

    Murdoch, 6150, Australia

  • Fondazione IRCCS Policlinico San Matteo

    RECRUITING

    Pavia, 27100, Italy

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    RECRUITING

    Rome, 00168, Italy

  • Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica - Ospedale San Cataldo

    RECRUITING

    Pisa, 56124, Italy

  • Fundacao Centro Medico de Campinas

    RECRUITING

    Campinas, 13083-190, Brazil

  • General Hospital of Athens - Alexandra

    RECRUITING

    Athens, 11528, Greece

  • Henry Ford Health System

    RECRUITING

    Detroit, Michigan, 48202, United States

  • Hippokration General Hospital of Athens

    RECRUITING

    Athens, 11527, Greece

  • Hospital Britanico de Buenos Aires

    RECRUITING

    Buenos Aires, C1280AEB, Argentina

  • Hospital Clinico Universitario de Salamanca

    RECRUITING

    Salamanca, 37007, Spain

  • Hospital Italiano de Buenos Aires

    RECRUITING

    Buenos Aires, C1199ABB, Argentina

  • Hospital Universitari de Bellvitge

    RECRUITING

    Barcelona, 08907, Spain

  • Hospital Universitario Juan Ramon Jimenez

    RECRUITING

    Huelva, Spain

  • Hospital Universitario Puerta de Hierro

    RECRUITING

    Majadahonda, Spain

  • Hospital Universitario Son Llatzer

    RECRUITING

    Palma de Mallorca, 07198, Spain

  • Hospital Universitario de A Coruna

    RECRUITING

    A Coruña, 15006, Spain

  • ICBA - Instituto Cardiovascular de Buenos Aires S.A.

    RECRUITING

    CABA, C1428ART, Argentina

  • INOVA Fairfax Hospital

    RECRUITING

    Falls Church, Virginia, 22042, United States

  • Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP

    RECRUITING

    Cerqueira César, 05403-000, Brazil

  • John H. Stroger, Jr. Hospital of Cook County

    RECRUITING

    Chicago, Illinois, 60612, United States

  • Johns Hopkins University

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • Kumamoto University Hospital

    RECRUITING

    Kumamoto, 860-8556, Japan

  • Laurelton Heart Specialists

    RECRUITING

    Rosedale, New York, 11422, United States

  • Maastricht University Medical Center

    RECRUITING

    Maastricht, 6229 HX, Netherlands

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic - Jacksonville

    RECRUITING

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic - Rochester

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • MedStar Washington Hospital Center - MedStar Heart and Vascular Institute

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Medical University of South Carolina (MUSC)

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Mount Sinai Hospital

    RECRUITING

    New York, New York, 10029, United States

  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)

    RECRUITING

    Tlalpan, 14080, Mexico

  • National Neuromuscular Research Institute

    RECRUITING

    Austin, Texas, 78759, United States

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 10016, Taiwan

  • National University Hospital - National University Health System

    RECRUITING

    Singapore, 119228, Singapore

  • New York University (NYU) School of Medicine - Langone Medical Center

    RECRUITING

    New York, New York, 10016, United States

  • Norrlands universitetssjukhus (University Hospital of Umea)

    RECRUITING

    Umeå, 90185, Sweden

  • Oregon Health & Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Penn Presbyterian Medical Center

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Princess Alexandra Hospital

    RECRUITING

    Woolloongabba, 4102, Australia

  • Prisma Health Cancer Institute

    RECRUITING

    Greenville, South Carolina, 89605, United States

  • Rutgers-Robert Wood Johnson Medical School

    RECRUITING

    New Brunswick, New Jersey, 08901, United States

  • Samsung Medical Center

    RECRUITING

    Seoul, 06351, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Seoul St. Mary's Hospital

    RECRUITING

    Seoul, 06591, South Korea

  • Severance Hospital, Yonsei University Health System

    RECRUITING

    Seoul, 03722, South Korea

  • Shinshu University Hospital

    RECRUITING

    Nagano, 390-8621, Japan

  • Singapore General Hospital

    RECRUITING

    Singapore, 169608, Singapore

  • St Vincent's Hospital Sydney

    RECRUITING

    Darlinghurst, 2010, Australia

  • St. Bartholomew's Hospital

    RECRUITING

    London, EC1A 7BE, United Kingdom

  • St. Luke's Hospital of Kansas City

    RECRUITING

    Kansas City, Missouri, 64111, United States

  • St. Michael's Hospital

    RECRUITING

    Toronto, Ontario, M5B1W8, Canada

  • Stanford University

    RECRUITING

    Stanford, California, 94305, United States

  • Taipei Veterans General Hospital

    RECRUITING

    Taipei, 11217, Taiwan

  • Tallaght University Hospital - The Adelaide and Meath Hospital

    RECRUITING

    Dublin, D24 NR04, Ireland

  • The Mater Misericordiae University Hospital

    RECRUITING

    Dublin, D07 R2WY, Ireland

  • UZ Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • Unidade Local de Saude de Santa Maria EPE - Hospital de Santa Maria

    RECRUITING

    Lisbon, 1649-035, Portugal

  • Unidade Local de Saude de Santo Antonio EPE - Hospital de Santo Antonio

    RECRUITING

    Porto, 4099-001, Portugal

  • Universitaetsklinikum Heidelberg

    RECRUITING

    Heidelberg, 69120, Germany

  • Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz

    RECRUITING

    Mainz, 55131, Germany

  • Universitair Medisch Centrum Utrecht

    RECRUITING

    Utrecht, 3584 CX, Netherlands

  • University College London Hospitals NHS Foundation Trust

    RECRUITING

    London, WC1N 3BG, United Kingdom

  • University Malaya Medical Centre (UMMC)

    RECRUITING

    Kuala Lumpur, 59100, Malaysia

  • University Medical Center Groningen

    RECRUITING

    Groningen, 9713 GZ, Netherlands

  • University of British Columbia

    RECRUITING

    Vancouver, British Columbia, V6Z1Y6, Canada

  • University of Calgary

    RECRUITING

    Calgary, Alberta, T2N 4Z6, Canada

  • University of California, Los Angeles (UCLA) - David Geffen School of Medicine

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of California, San Diego (UCSD) - Medical Center

    RECRUITING

    La Jolla, California, 92037, United States

  • University of California, San Francisco (UCSF)

    RECRUITING

    San Francisco, California, 94143, United States

  • University of Chicago - Medical Center

    RECRUITING

    Chicago, Illinois, 60637, United States

  • University of Colorado Anschutz

    RECRUITING

    Aurora, Colorado, 80045, United States

  • University of Maryland Medical Center

    RECRUITING

    Baltimore, Maryland, 21201, United States

  • University of Miami

    RECRUITING

    Miami, Florida, 33136, United States

  • University of Pittsburgh Medical Center, Presbyterian Hospital

    RECRUITING

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Texas Southwestern

    RECRUITING

    Dallas, Texas, 75390, United States

  • University of Utah

    RECRUITING

    Salt Lake City, Utah, 84132, United States

  • University of Washington Medical Center

    RECRUITING

    Seattle, Washington, 98195, United States

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23219, United States

  • Washington University in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

  • Westmead Hospital

    RECRUITING

    Westmead, NSW 2145, Australia

  • Yale University School of Medicine - Section of Cardiology

    RECRUITING

    New Haven, Connecticut, 06519, United States

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