Clinical Trial
Study Details
Study Overview
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.
The AG10-501 ACT-EARLY study is a randomized, multicenter, double-blind, placebo-controlled study of acoramidis for prevention of ATTR (with specific reference to either its cardiomyopathic or polyneuropathic manifestations). Participants will be stratified at randomization.
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 within 10 years younger than or older 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.
Contacts and Locations
University of California, San Diego (UCSD) - Medical Center
La Jolla, California 92037
United States
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California 90095
United States
University of California, San Francisco (UCSF)
San Francisco, California 94143
United States
Stanford University
Stanford, California 94305
United States
University of Colorado Anschutz
Aurora, Colorado 80045
United States
Yale University School of Medicine - Section of Cardiology
New Haven, Connecticut 06519
United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia 20010
United States
Mayo Clinic - Jacksonville
Jacksonville, Florida 32224
United States
University of Miami
Miami, Florida 33136
United States
Cleveland Clinic Florida
Weston, Florida 33331
United States
Emory University School of Medicine
Atlanta, Georgia 30322
United States
John H. Stroger, Jr. Hospital of Cook County
Chicago, Illinois 60612
United States
University of Chicago - Medical Center
Chicago, Illinois 60637
United States
University of Maryland Medical Center
Baltimore, Maryland 21201
United States
Johns Hopkins University
Baltimore, Maryland 21287
United States
Massachusetts General Hospital
Boston, Massachusetts 02114
United States
Brigham and Women's Hospital
Boston, Massachusetts 02115
United States
Boston University (BU) School of Medicine
Boston, Massachusetts 02118
United States
Henry Ford Health System
Detroit, Michigan 48202
United States
Mayo Clinic - Rochester
Rochester, Minnesota 55905
United States
St. Luke's Hospital of Kansas City
Kansas City, Missouri 64111
United States
Washington University in St. Louis
St Louis, Missouri 63110
United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey 08901
United States
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York 10016
United States
Mount Sinai Hospital
New York, New York 10029
United States
Columbia University Medical Center
New York, New York 10032
United States
Laurelton Heart Specialists
Rosedale, New York 11422
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
Cleveland Clinic
Cleveland, Ohio 44195
United States
Oregon Health & Science University
Portland, Oregon 97239
United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania 19104
United States
University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, Pennsylvania 15213
United States
Medical University of South Carolina (MUSC)
Charleston, South Carolina 29425
United States
Prisma Health Cancer Institute
Greenville, South Carolina 89605
United States
National Neuromuscular Research Institute
Austin, Texas 78759
United States
University of Texas Southwestern
Dallas, Texas 75390
United States
University of Utah
Salt Lake City, Utah 84132
United States
INOVA Fairfax Hospital
Falls Church, Virginia 22042
United States
Virginia Commonwealth University
Richmond, Virginia 23219
United States
University of Washington Medical Center
Seattle, Washington 98195
United States
Hospital Italiano de Buenos Aires
C1199ABB Buenos Aires
Argentina
Hospital Britanico de Buenos Aires
C1280AEB Buenos Aires
Argentina
ICBA - Instituto Cardiovascular de Buenos Aires S.A.
C1428ART CABA
Argentina
St Vincent's Hospital Sydney
Darlinghurst 2010
Australia
Fiona Stanley Hospital
Murdoch 6150
Australia
Westmead Hospital
Westmead NSW 2145
Australia
Princess Alexandra Hospital
Woolloongabba 4102
Australia
UZ Leuven
3000 Leuven
Belgium
Fundacao Centro Medico de Campinas
Campinas
13083-190
Brazil
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
Cerqueira César
05403-000
Brazil
CAPED - Centro Avancado de Pesquisa, Estudos e Diagnostico
Ribeirão Preto
14026-900
Brazil
Cardiopulmonar da Bahia S.A. (Hospital Cardio Pulmonar)
Salvador
40170-130
Brazil
University of Calgary
Calgary Alberta T2N 4Z6
Canada
University of British Columbia
Vancouver British Columbia V6Z1Y6
Canada
St. Michael's Hospital
Toronto Ontario M5B1W8
Canada
Aarhus University Hospital
8200 Aarhus
Denmark
CHU Bordeaux - Hopital Pellegrin
33076 Bordeaux
France
AP-HP Hopital Henri Mondor
94000 Créteil
France
AP-HP Hopital Bicetre
94270 Le Kremlin-Bicêtre
France
CHU Rennes
35000 Rennes
France
CHU de Toulouse - Hopital Rangueil
31400 Toulouse
France
Charite Universitaetsmedizin Berlin
10117 Berlin
Germany
Universitaetsklinikum Heidelberg
69120 Heidelberg
Germany
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
55131 Mainz
Germany
Hippokration General Hospital of Athens
11527 Athens
Greece
General Hospital of Athens - Alexandra
11528 Athens
Greece
The Mater Misericordiae University Hospital
Dublin
D07 R2WY
Ireland
Tallaght University Hospital - The Adelaide and Meath Hospital
Dublin
D24 NR04
Ireland
Azienda Ospedaliero-Universitaria di Bologna IRCCS Policlinico di S.Orsola
40138 Bologna
Italy
Azienda Ospedaliero-Universitaria Careggi
Florence
Italy
Fondazione IRCCS Policlinico San Matteo
27100 Pavia
Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica - Ospedale San Cataldo
56124 Pisa
Italy
Azienda Ospedaliero Universitaria Policlinico Umberto I
00161 Roma
Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
00168 Rome
Italy
Azienda Ospedaliero-Universitaria Sant'Andrea
00189 Rome
Italy
Kumamoto University Hospital
Kumamoto
860-8556
Japan
Shinshu University Hospital
Nagano
390-8621
Japan
University Malaya Medical Centre (UMMC)
59100 Kuala Lumpur
Malaysia
CHU de Fort de France
97261 Fort-de-France
Martinique
National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)
14080 Tlalpan
Mexico
University Medical Center Groningen
9713 GZ Groningen
Netherlands
Maastricht University Medical Center
6229 HX Maastricht
Netherlands
Erasmus MC
3015 GD Rotterdam
Netherlands
Universitair Medisch Centrum Utrecht
3584 CX Utrecht
Netherlands
Unidade Local de Saude de Santa Maria EPE - Hospital de Santa Maria
1649-035 Lisbon
Portugal
Unidade Local de Saude de Santo Antonio EPE - Hospital de Santo Antonio
4099-001 Porto
Portugal
National University Hospital - National University Health System
Singapore 119228
Singapore
Singapore General Hospital
Singapore 169608
Singapore
Seoul National University Hospital
Seoul
03080
South Korea
Severance Hospital, Yonsei University Health System
Seoul
03722
South Korea
Samsung Medical Center
Seoul
06351
South Korea
Seoul St. Mary's Hospital
Seoul
06591
South Korea
Hospital Universitario de A Coruna
15006 A Coruña
Spain
Hospital Universitari de Bellvitge
08907 Barcelona
Spain
Hospital Universitario Juan Ramon Jimenez
Huelva
Spain
Hospital Universitario Puerta de Hierro
Majadahonda
Spain
Hospital Universitario Son Llatzer
07198 Palma de Mallorca
Spain
Hospital Clinico Universitario de Salamanca
37007 Salamanca
Spain
Norrlands universitetssjukhus (University Hospital of Umea)
90185 Umeå
Sweden
Changhua Christian Hospital - Taiwan
Changhua, 500
Taiwan
National Taiwan University Hospital
Taipei, 10016
Taiwan
Taipei Veterans General Hospital
Taipei, 11217
Taiwan
St. Bartholomew's Hospital
London
EC1A 7BE
United Kingdom
University College London Hospitals NHS Foundation Trust
London
WC1N 3BG
United Kingdom