AmyloidosisPhase 3NCT06563895
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
A Phase 3 study testing Acoramidis in people with Amyloidosis and Amyloid Cardiomyopathy, run by Eidos Therapeutics, a BridgeBio company at 104 sites in 24 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- What is being tested?
- Acoramidis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 587 participants
- Where
- 104 sitesin 24 countries
- Study dates
- 12 May 2025to Dec 2032
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female ≥ 18 to ≤ 75 years of age inclusive.
- Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or all other pathogenic TTR variants).
- Participant's age is within 10 years younger than or older than PADO.
Who cannot join
- Evidence of ATTR-CM or ATTR-PN.
- Current or past (within last 1 to 12 months, depending on specific agent) 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 5 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.
Trial locations and contacts
104 sites in 24 countries. Contact the study team or a site near you to ask about taking part.
104 sites in 24 countries
United States40 sites, 40 recruiting
- Recruiting
Emory University School of Medicine
Atlanta, Georgia, 30322
- Recruiting
University of Colorado Anschutz
Aurora, Colorado, 80045
- Recruiting
National Neuromuscular Research Institute
Austin, Texas, 78759
- Recruiting
University of Maryland Medical Center
Baltimore, Maryland, 21201
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21287
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
- Recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
- Recruiting
Boston University (BU) School of Medicine
Boston, Massachusetts, 02118
- Recruiting
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425
- Recruiting
John H. Stroger, Jr. Hospital of Cook County
Chicago, Illinois, 60612
- Recruiting
University of Chicago - Medical Center
Chicago, Illinois, 60637
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
- Recruiting
University of Texas Southwestern
Dallas, Texas, 75390
- Recruiting
Henry Ford Health System
Detroit, Michigan, 48202
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
- Recruiting
INOVA Fairfax Hospital
Falls Church, Virginia, 22042
- Recruiting
Prisma Health Cancer Institute
Greenville, South Carolina, 89605
- Recruiting
Mayo Clinic - Jacksonville
Jacksonville, Florida, 32224
- Recruiting
St. Luke's Hospital of Kansas City
Kansas City, Missouri, 64111
- Recruiting
University of California, San Diego (UCSD) - Medical Center
La Jolla, California, 92037
- Recruiting
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California, 90095
- Recruiting
University of Miami
Miami, Florida, 33136
- Recruiting
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
- Recruiting
Yale University School of Medicine - Section of Cardiology
New Haven, Connecticut, 06519
- Recruiting
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, 10016
- Recruiting
Mount Sinai Hospital
New York, New York, 10029
- Recruiting
Columbia University Medical Center
New York, New York, 10032
- Recruiting
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104
- Recruiting
University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, Pennsylvania, 15213
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239
- Recruiting
Virginia Commonwealth University
Richmond, Virginia, 23219
- Recruiting
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
- Recruiting
Laurelton Heart Specialists
Rosedale, New York, 11422
- Recruiting
University of Utah
Salt Lake City, Utah, 84132
- Recruiting
University of California, San Francisco (UCSF)
San Francisco, California, 94143
- Recruiting
University of Washington Medical Center
Seattle, Washington, 98195
- Recruiting
Washington University in St. Louis
St Louis, Missouri, 63110
- Recruiting
Stanford University
Stanford, California, 94305
- Recruiting
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, 20010
- Recruiting
Cleveland Clinic Florida
Weston, Florida, 33331
Italy7 sites, 7 recruiting
- Recruiting
Azienda Ospedaliero-Universitaria di Bologna IRCCS Policlinico di S.Orsola
Bologna, 40138
- Recruiting
Azienda Ospedaliero-Universitaria Careggi
Florence
- Recruiting
Fondazione IRCCS Policlinico San Matteo
Pavia, 27100
- Recruiting
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanità Pubblica - Ospedale San Cataldo
Pisa, 56124
- Recruiting
Azienda Ospedaliero Universitaria Policlinico Umberto I
Roma, 00161
- Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, 00168
- Recruiting
Azienda Ospedaliero-Universitaria Sant'Andrea
Rome, 00189
Spain6 sites, 6 recruiting
- Recruiting
Hospital Universitario de A Coruna
A Coruña, 15006
- Recruiting
Hospital Universitari de Bellvitge
Barcelona, 08907
- Recruiting
Hospital Universitario Juan Ramon Jimenez
Huelva
- Recruiting
Hospital Universitario Puerta de Hierro
Majadahonda
- Recruiting
Hospital Universitario Son Llatzer
Palma de Mallorca, 07198
- Recruiting
Hospital Clinico Universitario de Salamanca
Salamanca, 37007
France5 sites, 5 recruiting
- Recruiting
CHU Bordeaux - Hopital Pellegrin
Bordeaux, 33076
- Recruiting
AP-HP Hopital Henri Mondor
Créteil, 94000
- Recruiting
AP-HP Hopital Bicetre
Le Kremlin-Bicêtre, 94270
- Recruiting
CHU Rennes
Rennes, 35000
- Recruiting
CHU de Toulouse - Hopital Rangueil
Toulouse, 31400
Australia4 sites, 4 recruiting
- Recruiting
St Vincent's Hospital Sydney
Darlinghurst, 2010
- Recruiting
Fiona Stanley Hospital
Murdoch, 6150
- Recruiting
Westmead Hospital
Westmead, NSW 2145
- Recruiting
Princess Alexandra Hospital
Woolloongabba, 4102
Brazil4 sites, 4 recruiting
- Recruiting
Fundacao Centro Medico de Campinas
Campinas, 13083-190
- Recruiting
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
Cerqueira César, 05403-000
- Recruiting
CAPED - Centro Avancado de Pesquisa, Estudos e Diagnostico
Ribeirão Preto, 14026-900
- Recruiting
Cardiopulmonar da Bahia S.A. (Hospital Cardio Pulmonar)
Salvador, 40170-130
Netherlands4 sites, 4 recruiting
- Recruiting
University Medical Center Groningen
Groningen, 9713 GZ
- Recruiting
Maastricht University Medical Center
Maastricht, 6229 HX
- Recruiting
Erasmus MC
Rotterdam, 3015 GD
- Recruiting
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
South Korea4 sites, 4 recruiting
- Recruiting
Seoul National University Hospital
Seoul, 03080
- Recruiting
Severance Hospital, Yonsei University Health System
Seoul, 03722
- Recruiting
Samsung Medical Center
Seoul, 06351
- Recruiting
Seoul St. Mary's Hospital
Seoul, 06591
Argentina3 sites, 3 recruiting
- Recruiting
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB
- Recruiting
Hospital Britanico de Buenos Aires
Buenos Aires, C1280AEB
- Recruiting
ICBA - Instituto Cardiovascular de Buenos Aires S.A.
CABA, C1428ART
Canada3 sites, 3 recruiting
- Recruiting
University of Calgary
Calgary, Alberta, T2N 4Z6
- Recruiting
St. Michael's Hospital
Toronto, Ontario, M5B1W8
- Recruiting
University of British Columbia
Vancouver, British Columbia, V6Z1Y6
Germany3 sites, 3 recruiting
- Recruiting
Charite Universitaetsmedizin Berlin
Berlin, 10117
- Recruiting
Universitaetsklinikum Heidelberg
Heidelberg, 69120
- Recruiting
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
Mainz, 55131
Taiwan3 sites, 3 recruiting
- Recruiting
Changhua Christian Hospital - Taiwan
Changhua, 500
- Recruiting
National Taiwan University Hospital
Taipei, 10016
- Recruiting
Taipei Veterans General Hospital
Taipei, 11217
Greece2 sites, 2 recruiting
- Recruiting
Hippokration General Hospital of Athens
Athens, 11527
- Recruiting
General Hospital of Athens - Alexandra
Athens, 11528
Ireland2 sites, 2 recruiting
- Recruiting
The Mater Misericordiae University Hospital
Dublin, D07 R2WY
- Recruiting
Tallaght University Hospital - The Adelaide and Meath Hospital
Dublin, D24 NR04
Japan2 sites, 2 recruiting
- Recruiting
Kumamoto University Hospital
Kumamoto, 860-8556
- Recruiting
Shinshu University Hospital
Nagano, 390-8621
Portugal2 sites, 2 recruiting
- Recruiting
Unidade Local de Saude de Santa Maria EPE - Hospital de Santa Maria
Lisbon, 1649-035
- Recruiting
Unidade Local de Saude de Santo Antonio EPE - Hospital de Santo Antonio
Porto, 4099-001
Singapore2 sites, 2 recruiting
- Recruiting
National University Hospital - National University Health System
Singapore, 119228
- Recruiting
Singapore General Hospital
Singapore, 169608
United Kingdom2 sites, 2 recruiting
- Recruiting
St. Bartholomew's Hospital
London, EC1A 7BE
- Recruiting
University College London Hospitals NHS Foundation Trust
London, WC1N 3BG
Malaysia1 site, 1 recruiting
- Recruiting
University Malaya Medical Centre (UMMC)
Kuala Lumpur, 59100
Martinique1 site, 1 recruiting
- Recruiting
CHU de Fort de France
Fort-de-France, 97261
Mexico1 site, 1 recruiting
- Recruiting
National Institute of Medical Sciences and Nutrition - Salvador Zubiran(INCMNSZ)
Tlalpan, 14080
Sweden1 site, 1 recruiting
- Recruiting
Norrlands universitetssjukhus (University Hospital of Umea)
Umeå, 90185
About this study
Official title: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
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.
Read more
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.
Read the detailed description
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.
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
- Being tested
- Acoramidis, Placebo oral tablet
- Keywords
- Amyloidosis, ATTR-CM, ATTR-PN, Transthyretin, Amyloid, TTR
- Medical subject headings
- Amyloidosis, Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Transthyretin-Related, Cardiomyopathies, Heart Diseases, Polyneuropathies, Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AcoramidisDrug
Also called AG10, ALXN2060
TTR stabilizer administered orally twice daily (BID)
Placebo oral tabletDrug
Non-active control administered orally twice daily (BID)
| Study group | Role | What they receive |
|---|---|---|
| acoramidis | Experimental | Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID) Receives: Drug: Acoramidis |
| Placebo | Placebo comparator | Subjects will receive placebo to match twice daily Receives: Drug: Placebo oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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)
ATTR-CM defined by biopsy or imaging-based diagnosis
Measured: Since randomization up to approximately 7 years or until the study is declared over
Time to development of ATTR-PN (centrally adjudicated)
ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
Measured: Since randomization up to approximately 7 years or until the study is declared over
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2024
- Study start12 May 2025
- Primary completionOct 2031 (estimated)
- Study completionDec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 587 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06563895
- Sponsor's protocol ID
- AG10-501
- Other IDs
- U1111-1307-8107, jRCT2071250116
- Status last verified
- Aug 2026
- First submitted
- 5 Aug 2024
- Last updated
- 4 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No