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Study identifier: NCT06563895 Synced from ClinicalTrials.gov · July 28, 2026
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Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

Condition: Amyloidosis · Amyloid Cardiomyopathy · Transthyretin Amyloidosis  ·  Sponsor: Eidos Therapeutics, a BridgeBio company

PhasePhase 3
Planned participants587
Who can joinAll sexes, 18 Years to 75 Years
Healthy volunteersNo

About this study

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 dis…

This description comes directly from the study's public registry record.

Talk to the study team

Medical Information  ·  1-844-550-2246  ·  medinfo@eidostx.com

Always discuss trial participation with your own doctor first.

Locations (103)

University of California, San Diego (UCSD) - Medical CenterLa Jolla, California, United StatesRecruiting
University of California, Los Angeles (UCLA) - David Geffen School of MedicineLos Angeles, California, United StatesRecruiting
University of California, San Francisco (UCSF)San Francisco, California, United StatesRecruiting
Stanford UniversityStanford, California, United StatesRecruiting
University of Colorado AnschutzAurora, Colorado, United StatesRecruiting
Yale University School of Medicine - Section of CardiologyNew Haven, Connecticut, United StatesRecruiting
MedStar Washington Hospital Center - MedStar Heart and Vascular InstituteWashington D.C., District of Columbia, United StatesRecruiting
Mayo Clinic - JacksonvilleJacksonville, Florida, United StatesRecruiting
Cleveland Clinic FloridaWeston, Florida, United StatesRecruiting
Emory University School of MedicineAtlanta, Georgia, United StatesRecruiting
John H. Stroger, Jr. Hospital of Cook CountyChicago, Illinois, United StatesRecruiting
University of Chicago - Medical CenterChicago, Illinois, United StatesRecruiting

+ 91 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06563895