Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
NCT ID: NCT06563895
Last Updated: 2025-12-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
582 participants
INTERVENTIONAL
2025-05-12
2032-12-31
Brief Summary
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Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.
Detailed Description
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The study population will be asymptomatic carriers of a known pathogenic TTR gene variant. A participant must be 18 to 75 inclusive years of age, and the age of the participant must be no more than 10 years younger than the predicted age of disease onset (PADO) based either on family history (pedigree analysis) or, if family history is insufficient, based on a TTR Variant Actuarial table from published literature. For example, if PADO for a given individual is found to be 50 years, the age of the participant must be between 40 and 75 years inclusive.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
PREVENTION
QUADRUPLE
Study Groups
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acoramidis
Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)
Acoramidis
TTR stabilizer administered orally twice daily (BID)
Placebo
Subjects will receive placebo to match twice daily
Placebo oral tablet
Non-active control administered orally twice daily (BID)
Interventions
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Acoramidis
TTR stabilizer administered orally twice daily (BID)
Placebo oral tablet
Non-active control administered orally twice daily (BID)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participants must have an established genotype (hetero- or homozygosity) of a TTR gene variant that is known to be pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or any other pathogenic TTR variant(s)) confirmed by central laboratory prior to randomization.
* Participant's age is no more than 10 years (≤ 10) younger than the PADO.
Exclusion Criteria
* Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H).
* Current or past 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 3 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.
18 Years
75 Years
ALL
No
Sponsors
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Eidos Therapeutics, a BridgeBio company
INDUSTRY
Responsible Party
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Locations
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University of California, San Diego (UCSD) - Medical Center
La Jolla, California, United States
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California, United States
University of California, San Francisco (UCSF)
San Francisco, California, United States
Stanford University
Stanford, California, United States
University of Colorado Anschutz
Aurora, Colorado, United States
Yale University School of Medicine - Section of Cardiology
New Haven, Connecticut, United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, United States
Mayo Clinic - Jacksonville
Jacksonville, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
John H. Stroger, Jr. Hospital of Cook County
Chicago, Illinois, United States
University of Chicago - Medical Center
Chicago, Illinois, United States
University of Maryland Medical Center
Baltimore, Maryland, United States
Johns Hopkins University
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Boston University (BU) School of Medicine
Boston, Massachusetts, United States
Henry Ford Health System
Detroit, Michigan, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
St. Luke's Hospital of Kansas City
Kansas City, Missouri, United States
Washington University in St. Louis
St Louis, Missouri, United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, United States
Mount Sinai Hospital
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
Laurelton Heart Specialists
Rosedale, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Cleveland Clinic
Cleveland, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States
Prisma Health Cancer Institute
Greenville, South Carolina, United States
National Neuromuscular Research Institute
Austin, Texas, United States
University of Texas Southwestern
Dallas, Texas, United States
University of Utah
Salt Lake City, Utah, United States
INOVA Fairfax Hospital
Falls Church, Virginia, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Hospital Italiano de Buenos Aires
Buenos Aires, , Argentina
St Vincent's Hospital Sydney
Darlinghurst, , Australia
Westmead Hospital
Westmead, , Australia
UZ Leuven
Leuven, , Belgium
Fundacao Centro Medico de Campinas
Campinas, , Brazil
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
Cerqueira César, , Brazil
Cardiopulmonar da Bahia S.A. (Hospital Cardio Pulmonar)
Salvador, , Brazil
University of Calgary
Calgary, Alberta, Canada
University of British Columbia
Vancouver, British Columbia, Canada
Hvitfeldt Poulsen
Aarhus, , Denmark
CHU Bordeaux - Hopital Pellegrin
Bordeaux, , France
AP-HP Hopital Henri Mondor
Créteil, , France
AP-HP Hopital Bicetre
Le Kremlin-Bicêtre, , France
CHU Rennes
Rennes, , France
CHU de Toulouse - Hopital Rangueil
Toulouse, , France
Charite Universitaetsmedizin Berlin
Berlin, , Germany
Universitaetsklinikum Heidelberg
Heidelberg, , Germany
The Mater Misericordiae University Hospital
Dublin, , Ireland
Tallaght University Hospital - The Adelaide and Meath Hospital
Dublin, , Ireland
Azienda Ospedaliero-Universitaria di Bologna IRCCS Policlinico di S.Orsola
Bologna, , Italy
Azienda Ospedaliero-Universitaria Careggi
Florence, , Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica - Ospedale San Cataldo
Pisa, , Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, , Italy
Azienda Ospedaliero-Universitaria Sant'Andrea
Rome, , Italy
University Malaya Medical Centre (UMMC)
Kuala Lumpur, , Malaysia
University Medical Center Groningen
Groningen, , Netherlands
Erasmus MC
Rotterdam, , Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, , Netherlands
Unidade Local de Saude de Santa Maria EPE - Hospital de Santa Maria
Lisbon, , Portugal
Unidade Local de Saude de Santo Antonio EPE - Hospital de Santo Antonio
Porto, , Portugal
Singapore General Hospital
Singapore, , Singapore
Seoul National University Hospital
Seoul, , South Korea
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Seoul St. Mary's Hospital
Seoul, , South Korea
Hospital Universitari de Bellvitge
Barcelona, , Spain
Hospital Universitario Juan Ramon Jimenez
Huelva, , Spain
Hospital Universitario Puerta de Hierro
Majadahonda, , Spain
Hospital Universitario Son Llatzer
Palma de Mallorca, , Spain
Hospital Clinico Universitario de Salamanca
Salamanca, , Spain
Norrlands universitetssjukhus (University Hospital of Umea)
Umeå, , Sweden
Changhua Christian Hospital - Taiwan
Changhua, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Taipei Veterans General Hospital
Taipei, , Taiwan
St. Bartholomew's Hospital
London, , United Kingdom
University College London Hospitals NHS Foundation Trust
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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U1111-1307-8107
Identifier Type: OTHER
Identifier Source: secondary_id
jRCT2071250116
Identifier Type: OTHER
Identifier Source: secondary_id
AG10-501
Identifier Type: -
Identifier Source: org_study_id