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ACT-EARLY Study for Asymptomatic Carriers of a Pathogenic TTR Variant

This study is for people who carry a specific gene change (pathogenic TTR variant) that can lead to transthyretin amyloidosis (ATTR). ATTR is a disease where a protein called transthyretin (TTR) breaks down and forms sticky clumps (amyloid) in organs like the heart. This study is testing if a drug called acoramidis can prevent ATTR from developing in people who have the gene change but don't yet have symptoms. You might be able to join if you are between 18 and 75 years old and have a known pathogenic TTR gene variant. The main goal is to see if acoramidis can delay or stop the first signs of ATTR, such as heart or nerve problems, over approximately seven years. The study is currently unclear on its status.

Study design
This is a randomized, multicenter, double-blind study, meaning participants will be assigned by chance to receive either acoramidis or a placebo (an inactive pill), and neither you nor your doctor will know which you are receiving. The study plans to enroll 587 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed from the start of the study for up to approximately 7 years, or until the study ends, to see if ATTR develops.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06563895

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

Recruiting
PHASE3Ages 18–75InterventionalPrevention
Eidos Therapeutics, a BridgeBio company
~587 participants
Updated 2026-08-04 on ClinicalTrials.gov
What's tested:AcoramidisPlacebo oral tablet

At a glance

Recruiting sites
104 of 104 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated)
Measured over Since randomization up to approximately 7 years or until the study is declared over

Conditions

Amyloidosis
Amyloid Cardiomyopathy
Transthyretin Amyloidosis
Cardiomyopathies
Heart Diseases
Polyneuropathies

Explore related trials

ATTR AmyloidosisTTR Gene

Where it's being run

104 sites across 48 states
Italy7
Spain6
France5
California4
New York4
Australia4
Brazil4
Netherlands4

Who to contact

Medical Information
[email protected]
1-844-550-2246
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

Exclusion

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.

What this trial measures

  • 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

    * ATTR-CM defined by biopsy or imaging-based diagnosis * ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis