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Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Sponsor: Eidos Therapeutics, a BridgeBio company
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.
Official title: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
582
Start Date
2025-05-12
Completion Date
2032-12
Last Updated
2026-03-30
Healthy Volunteers
No
Conditions
Interventions
Acoramidis
TTR stabilizer administered orally twice daily (BID)
Placebo oral tablet
Non-active control administered orally twice daily (BID)
Locations (98)
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
Cleveland Clinic Florida
Weston, 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
Hospital Britanico 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
CAPED - Centro Avancado de Pesquisa, Estudos e Diagnostico
Ribeirão Preto, 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
St. Michael's Hospital
Toronto, Ontario, 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
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
Mainz, Germany
Hippokration General Hospital of Athens
Athens, Greece
General Hospital of Athens - Alexandra
Athens, Greece
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
Azienda Ospedaliero Universitaria Policlinico Umberto I
Roma, Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy
Azienda Ospedaliero-Universitaria Sant'Andrea
Rome, Italy
Kumamoto University Hospital
Kumamoto, Japan
Shinshu University Hospital
Nagano, Japan
University Malaya Medical Centre (UMMC)
Kuala Lumpur, Malaysia
CHU de Fort de France
Fort-de-France, Martinique
National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)
Tlalpan, Mexico
University Medical Center Groningen
Groningen, Netherlands
Maastricht University Medical Center
Maastricht, 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
National University Hospital - National University Health System
Singapore, Singapore
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