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Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
NCT06563895 · Eidos Therapeutics, a BridgeBio company
In plain English
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Official title
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
About this study
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 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.
Eligibility criteria
Key Inclusion Criteria:
* 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.
Key Exclusion Criteria:
* 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.
Study design
Enrollment target: 582 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2025-05-12
Estimated completion: 2032-12
Last updated: 2026-03-30
Interventions
Drug: AcoramidisDrug: Placebo oral tablet
Primary outcomes
- • Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated) (Since randomization up to approximately 7 years or until the study is declared over)
Sponsor
Eidos Therapeutics, a BridgeBio company · industry
Contacts & investigators
ContactMedical Information · contact · medinfo@eidostx.com · 1-415-887-1471
All locations (98)
University of California, San Diego (UCSD) - Medical CenterRecruiting
La Jolla, California, United States
University of California, Los Angeles (UCLA) - David Geffen School of MedicineRecruiting
Los Angeles, California, United States
University of California, San Francisco (UCSF)Recruiting
San Francisco, California, United States
Stanford UniversityRecruiting
Stanford, California, United States
University of Colorado AnschutzRecruiting
Aurora, Colorado, United States
Yale University School of Medicine - Section of CardiologyRecruiting
New Haven, Connecticut, United States
MedStar Washington Hospital Center - MedStar Heart and Vascular InstituteRecruiting
Washington D.C., District of Columbia, United States
Mayo Clinic - JacksonvilleRecruiting
Jacksonville, Florida, United States
Cleveland Clinic FloridaRecruiting
Weston, Florida, United States
Emory University School of MedicineRecruiting
Atlanta, Georgia, United States
John H. Stroger, Jr. Hospital of Cook CountyRecruiting
Chicago, Illinois, United States
University of Chicago - Medical CenterRecruiting
Chicago, Illinois, United States
University of Maryland Medical CenterRecruiting
Baltimore, Maryland, United States
Johns Hopkins UniversityRecruiting
Baltimore, Maryland, United States
Massachusetts General HospitalRecruiting
Boston, Massachusetts, United States
Brigham and Women's HospitalRecruiting
Boston, Massachusetts, United States
Boston University (BU) School of MedicineRecruiting
Boston, Massachusetts, United States
Henry Ford Health SystemRecruiting
Detroit, Michigan, United States
Mayo Clinic - RochesterRecruiting
Rochester, Minnesota, United States
St. Luke's Hospital of Kansas CityRecruiting
Kansas City, Missouri, United States
Washington University in St. LouisRecruiting
St Louis, Missouri, United States
Rutgers-Robert Wood Johnson Medical SchoolRecruiting
New Brunswick, New Jersey, United States
New York University (NYU) School of Medicine - Langone Medical CenterRecruiting
New York, New York, United States
Mount Sinai HospitalRecruiting
New York, New York, United States
Columbia University Medical CenterRecruiting
New York, New York, United States
Laurelton Heart SpecialistsRecruiting
Rosedale, New York, United States
Duke University Medical CenterRecruiting
Durham, North Carolina, United States
Cleveland ClinicRecruiting
Cleveland, Ohio, United States
Oregon Health & Science UniversityRecruiting
Portland, Oregon, United States
Penn Presbyterian Medical CenterRecruiting
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center, Presbyterian HospitalRecruiting
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina (MUSC)Recruiting
Charleston, South Carolina, United States
Prisma Health Cancer InstituteRecruiting
Greenville, South Carolina, United States
National Neuromuscular Research InstituteRecruiting
Austin, Texas, United States
University of Texas SouthwesternRecruiting
Dallas, Texas, United States
University of UtahRecruiting
Salt Lake City, Utah, United States
INOVA Fairfax HospitalRecruiting
Falls Church, Virginia, United States
Virginia Commonwealth UniversityRecruiting
Richmond, Virginia, United States
Hospital Italiano de Buenos AiresRecruiting
Buenos Aires, Argentina
Hospital Britanico de Buenos AiresRecruiting
Buenos Aires, Argentina
St Vincent's Hospital SydneyRecruiting
Darlinghurst, Australia
Westmead HospitalRecruiting
Westmead, Australia
UZ LeuvenRecruiting
Leuven, Belgium
Fundacao Centro Medico de CampinasRecruiting
Campinas, Brazil
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSPRecruiting
Cerqueira César, Brazil
CAPED - Centro Avancado de Pesquisa, Estudos e DiagnosticoRecruiting
Ribeirão Preto, Brazil
Cardiopulmonar da Bahia S.A. (Hospital Cardio Pulmonar)Recruiting
Salvador, Brazil
University of CalgaryRecruiting
Calgary, Alberta, Canada
University of British ColumbiaRecruiting
Vancouver, British Columbia, Canada
St. Michael's HospitalRecruiting
Toronto, Ontario, Canada
Hvitfeldt PoulsenRecruiting
Aarhus, Denmark
CHU Bordeaux - Hopital PellegrinRecruiting
Bordeaux, France
AP-HP Hopital Henri MondorRecruiting
Créteil, France
AP-HP Hopital BicetreRecruiting
Le Kremlin-Bicêtre, France
CHU RennesRecruiting
Rennes, France
CHU de Toulouse - Hopital RangueilRecruiting
Toulouse, France
Charite Universitaetsmedizin BerlinRecruiting
Berlin, Germany
Universitaetsklinikum HeidelbergRecruiting
Heidelberg, Germany
Universitaetsmedizin der Johannes Gutenberg - Universitaet MainzRecruiting
Mainz, Germany
Hippokration General Hospital of AthensRecruiting
Athens, Greece
General Hospital of Athens - AlexandraRecruiting
Athens, Greece
The Mater Misericordiae University HospitalRecruiting
Dublin, Ireland
Tallaght University Hospital - The Adelaide and Meath HospitalRecruiting
Dublin, Ireland
Azienda Ospedaliero-Universitaria di Bologna IRCCS Policlinico di S.OrsolaRecruiting
Bologna, Italy
Azienda Ospedaliero-Universitaria CareggiRecruiting
Florence, Italy
Fondazione IRCCS Policlinico San MatteoRecruiting
Pavia, Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica - Ospedale San CataldoRecruiting
Pisa, Italy
Azienda Ospedaliero Universitaria Policlinico Umberto IRecruiting
Roma, Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro CuoreRecruiting
Rome, Italy
Azienda Ospedaliero-Universitaria Sant'AndreaRecruiting
Rome, Italy
Kumamoto University HospitalRecruiting
Kumamoto, Japan
Shinshu University HospitalRecruiting
Nagano, Japan
University Malaya Medical Centre (UMMC)Recruiting
Kuala Lumpur, Malaysia
CHU de Fort de FranceRecruiting
Fort-de-France, Martinique
National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)Recruiting
Tlalpan, Mexico
University Medical Center GroningenRecruiting
Groningen, Netherlands
Maastricht University Medical CenterRecruiting
Maastricht, Netherlands
Erasmus MCRecruiting
Rotterdam, Netherlands
Universitair Medisch Centrum UtrechtRecruiting
Utrecht, Netherlands
Unidade Local de Saude de Santa Maria EPE - Hospital de Santa MariaRecruiting
Lisbon, Portugal
Unidade Local de Saude de Santo Antonio EPE - Hospital de Santo AntonioRecruiting
Porto, Portugal
National University Hospital - National University Health SystemRecruiting
Singapore, Singapore
Singapore General HospitalRecruiting
Singapore, Singapore
Seoul National University HospitalRecruiting
Seoul, South Korea
Severance Hospital, Yonsei University Health SystemRecruiting
Seoul, South Korea
Samsung Medical CenterRecruiting
Seoul, South Korea
Seoul St. Mary's HospitalRecruiting
Seoul, South Korea
Hospital Universitari de BellvitgeRecruiting
Barcelona, Spain
Hospital Universitario Juan Ramon JimenezRecruiting
Huelva, Spain
Hospital Universitario Puerta de HierroRecruiting
Majadahonda, Spain
Hospital Universitario Son LlatzerRecruiting
Palma de Mallorca, Spain
Hospital Clinico Universitario de SalamancaRecruiting
Salamanca, Spain
Norrlands universitetssjukhus (University Hospital of Umea)Recruiting
Umeå, Sweden
Changhua Christian Hospital - TaiwanRecruiting
Changhua, Taiwan
National Taiwan University HospitalRecruiting
Taipei, Taiwan
Taipei Veterans General HospitalRecruiting
Taipei, Taiwan
St. Bartholomew's HospitalRecruiting
London, United Kingdom
University College London Hospitals NHS Foundation TrustRecruiting
London, United Kingdom