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

RecruitingPhase 3Ages 18 Years - 75 YearsAllEidos Therapeutics, a BridgeBio company
NCT06563895 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
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.
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What it tests

  • Acoramidis
  • Placebo oral tablet
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Where

104 sites
University of California, San Diego (UCSD) - Medical Center
La Jolla, United States
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, United States
University of California, San Francisco (UCSF)
San Francisco, United States
Stanford University
Stanford, United States
University of Colorado Anschutz
Aurora, United States
Yale University School of Medicine - Section of Cardiology
New Haven, United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., United States
Mayo Clinic - Jacksonville
Jacksonville, United States

…and 96 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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