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Preventing transthyretin amyloidosis in young carriers of a genetic variant

A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)

Phase 3 Interventional Eidos Therapeutics, a BridgeBio company · NCT06563895

This study is testing if a medication called acoramidis can help prevent transthyretin amyloidosis in young adults who carry a specific genetic variant that puts them at risk for the disease.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment587 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorEidos Therapeutics, a BridgeBio company Industry-sponsored
Locations101 sites (La Jolla, California and 100 other locations)
Trial IDNCT06563895 on ClinicalTrials.gov

What this trial studies

This study investigates the use of acoramidis to prevent transthyretin amyloidosis (ATTR) in asymptomatic carriers of pathogenic TTR gene variants. It is a randomized, multicenter, double-blind, placebo-controlled trial that aims to assess the efficacy of the treatment in preventing the onset of cardiomyopathic or polyneuropathic manifestations of the disease. Participants aged 18 to 75, who are carriers of a known pathogenic TTR variant, will be enrolled based on their predicted age of disease onset. The study will compare the effects of acoramidis against a placebo over a specified period.

Who should consider this trial

Good fit: Ideal candidates are asymptomatic individuals aged 18 to 75 who carry a known pathogenic TTR gene variant.

Not a fit: Patients who already exhibit symptoms of ATTR or have a TTR variant that is phenotypically protective will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly delay or prevent the onset of serious heart and nerve complications associated with transthyretin amyloidosis.

How similar studies have performed: While there have been studies on treating symptomatic ATTR, this preventive approach in asymptomatic carriers is relatively novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

La Jolla, California and 100 other locations

+51 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions AmyloidosisAmyloid CardiomyopathyTransthyretin AmyloidosisCardiomyopathiesHeart DiseasesPolyneuropathiesATTR-CMATTR-PN
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.