Actively Recruiting
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Acoramidis for Preventing Transthyretin Amyloidosis in Adults with a Pathogenic TTR Variant
Led by Eidos Therapeutics, a BridgeBio company · Updated on 2026-05-12
587
Participants Needed
101
Research Sites
61 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Transthyretin amyloidosis (ATTR) is a condition where the transthyretin (TTR) protein breaks apart and forms harmful amyloid plaques that build up in various organs, causing damage. There are two types: wild-type ATTR, which develops with age, and variant ATTR, which is inherited through a defective TTR gene. Amyloid buildup in the heart leads to transthyretin amyloid cardiomyopathy (ATTR-CM), and in nerves, it causes transthyretin amyloid polyneuropathy (ATTR-PN). Researchers are studying acoramidis, a drug designed to stabilize TTR protein to prevent or delay these diseases in adults who carry the inherited defective gene but have no symptoms yet. This clinical trial is a phase 3, randomized, double-blind, placebo-controlled study involving asymptomatic adults aged 18 to 75 who carry a pathogenic TTR gene variant. Participants will be randomly assigned to receive either oral acoramidis 712 mg twice daily or a matching placebo. The study will stratify participants based on their predicted age of disease onset and will follow them for up to approximately 7 years or until the study ends. During the study, participants will be regularly monitored for the development of ATTR-CM or ATTR-PN through centrally adjudicated assessments. The main measurement is the time until these conditions develop. The study also tracks safety and secondary outcomes such as the specific onset of ATTR-CM or ATTR-PN. Participants will attend study visits for evaluations including medical exams and tests over the course of the trial, which could last about 7 years or until the study concludes.
CONDITIONS
Brief Title
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 18 to 75 years inclusive
- Established genotype with a known pathogenic or likely pathogenic TTR gene variant
- Age within 10 years younger or older than predicted age of disease onset based on family history or published data
You will not qualify if you...
- Evidence of transthyretin amyloid cardiomyopathy or polyneuropathy
- Current or recent treatment with other TTR modifying therapies
- Unable or contraindicated to undergo cardiac magnetic resonance testing
- Major organ dysfunction including kidney, liver, heart disease, or neuropathy
- Other significant diseases such as cancer within 5 years, untreated thyroid disorders, type 1 diabetes, active hepatitis B or C, or HIV
- Major surgery within past 3 months or planned within next 12 months
- Known allergy to acoramidis
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 7 years or until the study is declared over
Participants receive acoramidis or placebo orally twice daily to prevent transthyretin amyloidosis.
Regular visits as scheduled during the treatment period
Trial Site Locations
Total: 101 locations
1
University of California, San Diego (UCSD) - Medical Center
La Jolla, California, United States, 92037
Actively Recruiting
2
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California, United States, 90095
Actively Recruiting
3
University of California, San Francisco (UCSF)
San Francisco, California, United States, 94143
Actively Recruiting
4
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
5
University of Colorado Anschutz
Aurora, Colorado, United States, 80045
Actively Recruiting
6
Yale University School of Medicine - Section of Cardiology
New Haven, Connecticut, United States, 06519
Actively Recruiting
7
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
8
Mayo Clinic - Jacksonville
Jacksonville, Florida, United States, 32224
Actively Recruiting
9
Cleveland Clinic Florida
Weston, Florida, United States, 33331
Actively Recruiting
10
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
11
John H. Stroger, Jr. Hospital of Cook County
Chicago, Illinois, United States, 60612
Actively Recruiting
12
University of Chicago - Medical Center
Chicago, Illinois, United States, 60637
Actively Recruiting
13
University of Maryland Medical Center
Baltimore, Maryland, United States, 21201
Actively Recruiting
14
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Actively Recruiting
15
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
16
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
17
Boston University (BU) School of Medicine
Boston, Massachusetts, United States, 02118
Actively Recruiting
18
Henry Ford Health System
Detroit, Michigan, United States, 48202
Actively Recruiting
19
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
Actively Recruiting
20
St. Luke's Hospital of Kansas City
Kansas City, Missouri, United States, 64111
Actively Recruiting
21
Washington University in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
22
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States, 08901
Actively Recruiting
23
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, United States, 10016
Actively Recruiting
24
Mount Sinai Hospital
New York, New York, United States, 10029
Actively Recruiting
25
Columbia University Medical Center
New York, New York, United States, 10032
Actively Recruiting
26
Laurelton Heart Specialists
Rosedale, New York, United States, 11422
Actively Recruiting
27
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
28
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
29
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
30
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
31
University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
32
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States, 29425
Actively Recruiting
33
Prisma Health Cancer Institute
Greenville, South Carolina, United States, 89605
Actively Recruiting
34
National Neuromuscular Research Institute
Austin, Texas, United States, 78759
Actively Recruiting
35
University of Texas Southwestern
Dallas, Texas, United States, 75390
Actively Recruiting
36
University of Utah
Salt Lake City, Utah, United States, 84132
Actively Recruiting
37
INOVA Fairfax Hospital
Falls Church, Virginia, United States, 22042
Actively Recruiting
38
Virginia Commonwealth University
Richmond, Virginia, United States, 23219
Actively Recruiting
39
University of Washington Medical Center
Seattle, Washington, United States, 98195
Actively Recruiting
40
Hospital Italiano de Buenos Aires
Buenos Aires, Argentina, C1199ABB
Actively Recruiting
41
Hospital Britanico de Buenos Aires
Buenos Aires, Argentina, C1280AEB
Actively Recruiting
42
St Vincent's Hospital Sydney
Darlinghurst, Australia, 2010
Actively Recruiting
43
Fiona Stanley Hospital
Murdoch, Australia, 6150
Actively Recruiting
44
Westmead Hospital
Westmead, Australia, NSW 2145
Actively Recruiting
45
Princess Alexandra Hospital
Woolloongabba, Australia, 4102
Actively Recruiting
46
UZ Leuven
Leuven, Belgium, 3000
Actively Recruiting
47
Fundacao Centro Medico de Campinas
Campinas, Brazil, 13083-190
Actively Recruiting
48
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
Cerqueira César, Brazil, 05403-000
Actively Recruiting
49
CAPED - Centro Avancado de Pesquisa, Estudos e Diagnostico
Ribeirão Preto, Brazil, 14026-900
Actively Recruiting
50
Cardiopulmonar da Bahia S.A. (Hospital Cardio Pulmonar)
Salvador, Brazil, 40170-130
Actively Recruiting
51
University of Calgary
Calgary, Alberta, Canada, T2N 4Z6
Actively Recruiting
52
University of British Columbia
Vancouver, British Columbia, Canada, V6Z1Y6
Actively Recruiting
53
St. Michael's Hospital
Toronto, Ontario, Canada, M5B1W8
Actively Recruiting
54
Hvitfeldt Poulsen
Aarhus, Denmark, 8200
Actively Recruiting
55
CHU Bordeaux - Hopital Pellegrin
Bordeaux, France, 33076
Actively Recruiting
56
AP-HP Hopital Henri Mondor
Créteil, France, 94000
Actively Recruiting
57
AP-HP Hopital Bicetre
Le Kremlin-Bicêtre, France, 94270
Actively Recruiting
58
CHU Rennes
Rennes, France, 35000
Actively Recruiting
59
CHU de Toulouse - Hopital Rangueil
Toulouse, France, 31400
Actively Recruiting
60
Charite Universitaetsmedizin Berlin
Berlin, Germany, 10117
Actively Recruiting
61
Universitaetsklinikum Heidelberg
Heidelberg, Germany, 69120
Actively Recruiting
62
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
Mainz, Germany, 55131
Actively Recruiting
63
Hippokration General Hospital of Athens
Athens, Greece, 11527
Actively Recruiting
64
General Hospital of Athens - Alexandra
Athens, Greece, 11528
Actively Recruiting
65
The Mater Misericordiae University Hospital
Dublin, Ireland, D07 R2WY
Actively Recruiting
66
Tallaght University Hospital - The Adelaide and Meath Hospital
Dublin, Ireland, D24 NR04
Actively Recruiting
67
Azienda Ospedaliero-Universitaria di Bologna IRCCS Policlinico di S.Orsola
Bologna, Italy, 40138
Actively Recruiting
68
Azienda Ospedaliero-Universitaria Careggi
Florence, Italy
Actively Recruiting
69
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy, 27100
Actively Recruiting
70
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica - Ospedale San Cataldo
Pisa, Italy, 56124
Actively Recruiting
71
Azienda Ospedaliero Universitaria Policlinico Umberto I
Roma, Italy, 00161
Actively Recruiting
72
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy, 00168
Actively Recruiting
73
Azienda Ospedaliero-Universitaria Sant'Andrea
Rome, Italy, 00189
Actively Recruiting
74
Kumamoto University Hospital
Kumamoto, Japan, 860-8556
Actively Recruiting
75
Shinshu University Hospital
Nagano, Japan, 390-8621
Actively Recruiting
76
University Malaya Medical Centre (UMMC)
Kuala Lumpur, Malaysia, 59100
Actively Recruiting
77
CHU de Fort de France
Fort-de-France, Martinique, 97261
Actively Recruiting
78
National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)
Tlalpan, Mexico, 14080
Actively Recruiting
79
University Medical Center Groningen
Groningen, Netherlands, 9713 GZ
Actively Recruiting
80
Maastricht University Medical Center
Maastricht, Netherlands, 6229 HX
Actively Recruiting
81
Erasmus MC
Rotterdam, Netherlands, 3015 GD
Actively Recruiting
82
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands, 3584 CX
Actively Recruiting
83
Unidade Local de Saude de Santa Maria EPE - Hospital de Santa Maria
Lisbon, Portugal, 1649-035
Actively Recruiting
84
Unidade Local de Saude de Santo Antonio EPE - Hospital de Santo Antonio
Porto, Portugal, 4099-001
Actively Recruiting
85
National University Hospital - National University Health System
Singapore, Singapore, 119228
Actively Recruiting
86
Singapore General Hospital
Singapore, Singapore, 169608
Actively Recruiting
87
Seoul National University Hospital
Seoul, South Korea, 03080
Actively Recruiting
88
Severance Hospital, Yonsei University Health System
Seoul, South Korea, 03722
Actively Recruiting
89
Samsung Medical Center
Seoul, South Korea, 06351
Actively Recruiting
90
Seoul St. Mary's Hospital
Seoul, South Korea, 06591
Actively Recruiting
91
Hospital Universitari de Bellvitge
Barcelona, Spain, 08907
Actively Recruiting
92
Hospital Universitario Juan Ramon Jimenez
Huelva, Spain
Actively Recruiting
93
Hospital Universitario Puerta de Hierro
Majadahonda, Spain
Actively Recruiting
94
Hospital Universitario Son Llatzer
Palma de Mallorca, Spain, 07198
Actively Recruiting
95
Hospital Clinico Universitario de Salamanca
Salamanca, Spain, 37007
Actively Recruiting
96
Norrlands universitetssjukhus (University Hospital of Umea)
Umeå, Sweden, 90185
Actively Recruiting
97
Changhua Christian Hospital - Taiwan
Changhua, Taiwan, 500
Actively Recruiting
98
National Taiwan University Hospital
Taipei, Taiwan, 10016
Actively Recruiting
99
Taipei Veterans General Hospital
Taipei, Taiwan, 11217
Actively Recruiting
100
St. Bartholomew's Hospital
London, United Kingdom, EC1A 7BE
Actively Recruiting
101
University College London Hospitals NHS Foundation Trust
London, United Kingdom, WC1N 3BG
Actively Recruiting
Research Team
M
Medical Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
PREVENTION
Number of Arms
2
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