Recruiting

Phase 3

AcoramidisAcoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

Sponsor:

Eidos Therapeutics, a BridgeBio company

Code:

NCT06563895

Conditions

Amyloidosis

Amyloid Cardiomyopathy

Transthyretin Amyloidosis

Cardiomyopathies

Heart Diseases

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Acoramidis

Placebo oral tablet

Study Details

Brief 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. 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.

Conditions

Amyloidosis

Amyloid Cardiomyopathy

Transthyretin Amyloidosis

Cardiomyopathies

Heart Diseases

Study ID

NCT06563895

Start date

May 12, 2025

Status verified date

Aug, 2026

Completion date

Dec, 2032

Anticipated

Primary completion date

Oct, 2031

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Key Inclusion Criteria:

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.

Key Exclusion Criteria:

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.

Study Design

Enrollment

587 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Prevention

Interventions and Outcome Measures

Arms

experimental: acoramidis

Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)

placebo comparator: Placebo

Subjects will receive placebo to match twice daily

Interventions

Acoramidis

TTR stabilizer administered orally twice daily (BID)

Placebo oral tablet

Non-active control administered orally twice daily (BID)

Primary outcome measure

Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated) [ Time Frame: Since randomization up to approximately 7 years or until the study is declared over ]

Central Contacts and Locations

Central contacts

Medical Information

1-844-550-2246 medinfo@eidostx.com

Locations

University of California, San Diego (UCSD) - Medical Center

Recruiting

La Jolla, California, United States, 92037

University of California, Los Angeles (UCLA) - David Geffen School of Medicine

Recruiting

Los Angeles, California, United States, 90095

University of California, San Francisco (UCSF)

Recruiting

San Francisco, California, United States, 94143

Stanford University

Recruiting

Stanford, California, United States, 94305

University of Colorado Anschutz

Recruiting

Aurora, Colorado, United States, 80045

Yale University School of Medicine - Section of Cardiology

Recruiting

New Haven, Connecticut, United States, 06519

MedStar Washington Hospital Center - MedStar Heart and Vascular Institute

Recruiting

Washington D.C., District of Columbia, United States, 20010

Mayo Clinic - Jacksonville

Recruiting

Jacksonville, Florida, United States, 32224

University of Miami

Recruiting

Miami, Florida, United States, 33136

Cleveland Clinic Florida

Recruiting

Weston, Florida, United States, 33331

Emory University School of Medicine

Recruiting

Atlanta, Georgia, United States, 30322

John H. Stroger, Jr. Hospital of Cook County

Recruiting

Chicago, Illinois, United States, 60612

University of Chicago - Medical Center

Recruiting

Chicago, Illinois, United States, 60637

University of Maryland Medical Center

Recruiting

Baltimore, Maryland, United States, 21201

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21287

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Brigham and Women's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Boston University (BU) School of Medicine

Recruiting

Boston, Massachusetts, United States, 02118

Henry Ford Health System

Recruiting

Detroit, Michigan, United States, 48202

Mayo Clinic - Rochester

Recruiting

Rochester, Minnesota, United States, 55905

St. Luke's Hospital of Kansas City

Recruiting

Kansas City, Missouri, United States, 64111

Washington University in St. Louis

Recruiting

St Louis, Missouri, United States, 63110

Rutgers-Robert Wood Johnson Medical School

Recruiting

New Brunswick, New Jersey, United States, 08901

New York University (NYU) School of Medicine - Langone Medical Center

Recruiting

New York, New York, United States, 10016

Mount Sinai Hospital

Recruiting

New York, New York, United States, 10029

Columbia University Medical Center

Recruiting

New York, New York, United States, 10032

Laurelton Heart Specialists

Recruiting

Rosedale, New York, United States, 11422

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Oregon Health & Science University

Recruiting

Portland, Oregon, United States, 97239

Penn Presbyterian Medical Center

Recruiting

Philadelphia, Pennsylvania, United States, 19104

University of Pittsburgh Medical Center, Presbyterian Hospital

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

Medical University of South Carolina (MUSC)

Recruiting

Charleston, South Carolina, United States, 29425

Prisma Health Cancer Institute

Recruiting

Greenville, South Carolina, United States, 89605

National Neuromuscular Research Institute

Recruiting

Austin, Texas, United States, 78759

University of Texas Southwestern

Recruiting

Dallas, Texas, United States, 75390

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84132

INOVA Fairfax Hospital

Recruiting

Falls Church, Virginia, United States, 22042

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23219

University of Washington Medical Center

Recruiting

Seattle, Washington, United States, 98195

University of Calgary

Recruiting

Calgary, Alberta, Canada, T2N 4Z6

University of British Columbia

Recruiting

Vancouver, British Columbia, Canada, V6Z1Y6

St. Michael's Hospital

Recruiting

Toronto, Ontario, Canada, M5B1W8

More Information

Sponsor

Eidos Therapeutics, a BridgeBio company

Last update posted

Aug 4, 2026

Last verified

Aug, 2026

Keywords

Amyloidosis
ATTR-CM
ATTR-PN
Transthyretin
Amyloid
TTR

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Eidos Therapeutics, a BridgeBio company on 2026-08-04.