Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895 | Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: AG10-501U1111-1307-8107jRCT2071250116
• Study Type: interventional
• Target: 582
• Locations: 24 countries
• Sites: 98

Brief Summary

Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age. 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.

Conditions

Transthyretin Amyloidosis; Cardiomyopathies; Heart Diseases; Polyneuropathies; Amyloidosis; Amyloid Cardiomyopathy

Keywords

Amyloidosis; ATTR-CM; ATTR-PN; Transthyretin; Amyloid; TTR

Outcome Measures

Primary Outcomes (1)

• Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated)

  * ATTR-CM defined by biopsy or imaging-based diagnosis * ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis

  Since randomization up to approximately 7 years or until the study is declared over

Secondary Outcomes (2)

• Time to development of ATTR-CM (centrally adjudicated)

  Since randomization up to approximately 7 years or until the study is declared over

• Time to development of ATTR-PN (centrally adjudicated)

  Since randomization up to approximately 7 years or until the study is declared over

Study Arms (2)

acoramidis

EXPERIMENTAL

Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)

Drug: Acoramidis

Placebo

PLACEBO COMPARATOR

Subjects will receive placebo to match twice daily

Drug: Placebo oral tablet

Interventions

Acoramidis DRUG

TTR stabilizer administered orally twice daily (BID)

Also known as: AG10, ALXN2060

acoramidis

Placebo oral tablet DRUG

Non-active control administered orally twice daily (BID)

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female ≥ 18 to ≤ 75 years of age inclusive.
• 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.

You may not qualify if:

• Evidence of ATTR-CM or ATTR-PN.
• 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.

Sponsors & Collaborators

• Eidos Therapeutics, a BridgeBio company: lead

Study Sites (98)

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

La Jolla, California, 92037, United States

RECRUITING

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

Los Angeles, California, 90095, United States

RECRUITING

University of California, San Francisco (UCSF)

San Francisco, California, 94143, United States

RECRUITING

Stanford University

Stanford, California, 94305, United States

RECRUITING

University of Colorado Anschutz

Aurora, Colorado, 80045, United States

RECRUITING

Yale University School of Medicine - Section of Cardiology

New Haven, Connecticut, 06519, United States

RECRUITING

MedStar Washington Hospital Center - MedStar Heart and Vascular Institute

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

RECRUITING

Mayo Clinic - Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Cleveland Clinic Florida

Weston, Florida, 33331, United States

RECRUITING

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

RECRUITING

John H. Stroger, Jr. Hospital of Cook County

Chicago, Illinois, 60612, United States

RECRUITING

University of Chicago - Medical Center

Chicago, Illinois, 60637, United States

RECRUITING

University of Maryland Medical Center

Baltimore, Maryland, 21201, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Boston University (BU) School of Medicine

Boston, Massachusetts, 02118, United States

RECRUITING

Henry Ford Health System

Detroit, Michigan, 48202, United States

RECRUITING

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

St. Luke's Hospital of Kansas City

Kansas City, Missouri, 64111, United States

RECRUITING

Washington University in St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

RECRUITING

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

New York, New York, 10016, United States

RECRUITING

Mount Sinai Hospital

New York, New York, 10029, United States

RECRUITING

Columbia University Medical Center

New York, New York, 10032, United States

RECRUITING

Laurelton Heart Specialists

Rosedale, New York, 11422, United States

RECRUITING

Duke University Medical Center

Durham, North Carolina, 27710, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

RECRUITING

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pittsburgh Medical Center, Presbyterian Hospital

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States

RECRUITING

Prisma Health Cancer Institute

Greenville, South Carolina, 89605, United States

RECRUITING

National Neuromuscular Research Institute

Austin, Texas, 78759, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75390, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84132, United States

RECRUITING

INOVA Fairfax Hospital

Falls Church, Virginia, 22042, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

RECRUITING

Hospital Italiano de Buenos Aires

Buenos Aires, C1199ABB, Argentina

RECRUITING

Hospital Britanico de Buenos Aires

Buenos Aires, C1280AEB, Argentina

RECRUITING

St Vincent's Hospital Sydney

Darlinghurst, 2010, Australia

RECRUITING

Westmead Hospital

Westmead, NSW 2145, Australia

RECRUITING

UZ Leuven

Leuven, 3000, Belgium

RECRUITING

Fundacao Centro Medico de Campinas

Campinas, 13083-190, Brazil

RECRUITING

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

Cerqueira César, 05403-000, Brazil

RECRUITING

CAPED - Centro Avancado de Pesquisa, Estudos e Diagnostico

Ribeirão Preto, 14026-900, Brazil

RECRUITING

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

Salvador, 40170-130, Brazil

RECRUITING

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

RECRUITING

University of British Columbia

Vancouver, British Columbia, V6Z1Y6, Canada

RECRUITING

St. Michael's Hospital

Toronto, Ontario, M5B1W8, Canada

RECRUITING

Hvitfeldt Poulsen

Aarhus, 8200, Denmark

RECRUITING

CHU Bordeaux - Hopital Pellegrin

Bordeaux, 33076, France

RECRUITING

AP-HP Hopital Henri Mondor

Créteil, 94000, France

RECRUITING

AP-HP Hopital Bicetre

Le Kremlin-Bicêtre, 94270, France

RECRUITING

CHU Rennes

Rennes, 35000, France

RECRUITING

CHU de Toulouse - Hopital Rangueil

Toulouse, 31400, France

RECRUITING

Charite Universitaetsmedizin Berlin

Berlin, 10117, Germany

RECRUITING

Universitaetsklinikum Heidelberg

Heidelberg, 69120, Germany

RECRUITING

Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz

Mainz, 55131, Germany

RECRUITING

Hippokration General Hospital of Athens

Athens, 11527, Greece

RECRUITING

General Hospital of Athens - Alexandra

Athens, 11528, Greece

RECRUITING

The Mater Misericordiae University Hospital

Dublin, D07 R2WY, Ireland

RECRUITING

Tallaght University Hospital - The Adelaide and Meath Hospital

Dublin, D24 NR04, Ireland

RECRUITING

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

Bologna, 40138, Italy

RECRUITING

Azienda Ospedaliero-Universitaria Careggi

Florence, Italy

RECRUITING

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

RECRUITING

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

Pisa, 56124, Italy

RECRUITING

Azienda Ospedaliero Universitaria Policlinico Umberto I

Roma, 00161, Italy

RECRUITING

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

Rome, 00168, Italy

RECRUITING

Azienda Ospedaliero-Universitaria Sant'Andrea

Rome, 00189, Italy

RECRUITING

Kumamoto University Hospital

Kumamoto, 860-8556, Japan

RECRUITING

Shinshu University Hospital

Nagano, 390-8621, Japan

RECRUITING

University Malaya Medical Centre (UMMC)

Kuala Lumpur, 59100, Malaysia

RECRUITING

CHU de Fort de France

Fort-de-France, 97261, Martinique

RECRUITING

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

Tlalpan, 14080, Mexico

RECRUITING

University Medical Center Groningen

Groningen, 9713 GZ, Netherlands

RECRUITING

Maastricht University Medical Center

Maastricht, 6229 HX, Netherlands

RECRUITING

Erasmus MC

Rotterdam, 3015 GD, Netherlands

RECRUITING

Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands

RECRUITING

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

Lisbon, 1649-035, Portugal

RECRUITING

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

Porto, 4099-001, Portugal

RECRUITING

National University Hospital - National University Health System

Singapore, 119228, Singapore

RECRUITING

Singapore General Hospital

Singapore, 169608, Singapore

RECRUITING

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

Seoul St. Mary's Hospital

Seoul, 06591, South Korea

RECRUITING

Hospital Universitari de Bellvitge

Barcelona, 08907, Spain

RECRUITING

Hospital Universitario Juan Ramon Jimenez

Huelva, Spain

RECRUITING

Hospital Universitario Puerta de Hierro

Majadahonda, Spain

RECRUITING

Hospital Universitario Son Llatzer

Palma de Mallorca, 07198, Spain

RECRUITING

Hospital Clinico Universitario de Salamanca

Salamanca, 37007, Spain

RECRUITING

Norrlands universitetssjukhus (University Hospital of Umea)

Umeå, 90185, Sweden

RECRUITING

Changhua Christian Hospital - Taiwan

Changhua, 500, Taiwan

RECRUITING

National Taiwan University Hospital

Taipei, 10016, Taiwan

RECRUITING

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

RECRUITING

St. Bartholomew's Hospital

London, EC1A 7BE, United Kingdom

RECRUITING

University College London Hospitals NHS Foundation Trust

London, WC1N 3BG, United Kingdom

RECRUITING

MeSH Terms

Conditions

Amyloidosis; Amyloid Neuropathies, Familial; Amyloidosis, Hereditary, Transthyretin-Related; Cardiomyopathies; Heart Diseases; Polyneuropathies; Alzheimer Disease

Interventions

attruby

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Cardiovascular Diseases; Dementia; Brain Diseases; Central Nervous System Diseases; Tauopathies; Neurocognitive Disorders; Mental Disorders

Central Study Contacts

Medical Information

CONTACT

1-415-887-1471; medinfo@eidostx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2024
• First Posted: August 21, 2024
• Study Start: May 12, 2025
• Primary Completion (Estimated): October 1, 2031
• Study Completion (Estimated): December 1, 2032
• Last Updated: March 30, 2026

Record last verified: 2026-03

Locations

IT (7); KR (4); AR (2); GB (2); ES (5); BE (1); JP (2); TW (3); GR (2); PT (2); BR (4); DE (3); AU (2); MA (1); SE (1); US (38); DK (1); ME (1); CA (3); FR (5); IE (2); MY (1); NL (4); SG (2)