Science / ATTRv / ACT-EARLY

ACT-EARLY

A Clinical Trial Studying the Prevention of Hereditary ATTR (or ATTRv)

About the ACT-EARLY Study

This study is designed to evaluate whether acoramidis can prevent or delay the onset of disease in individuals at risk for hereditary transthyretin amyloidosis (ATTRv) who have not yet developed symptoms.

This study is being conducted in individuals who carry a TTR genetic variant and who have not yet developed symptoms of, or been diagnosed with ATTRv. Participants will be randomly assigned (by chance) to receive either the investigational oral drug acoramidis or a placebo (an inactive substance), with a 50% likelihood for each group.

Who is eligible?

You may be eligible for this study if you are between 18 and 75 years old, have inherited a TTR genetic variant that puts you at risk of developing ATTRv amyloid disease, and are within 10 years, or older, of the typical age when symptoms of ATTRv amyloid disease are expected to develop.


The open label extension is available to qualified participants who develop ATTR-CM during the treatment period.

Participants who develop ATTR-PN only are not eligible for the open label extension and should seek therapy outside of this trial.

What the ACT-EARLY Study Is Exploring

01

Test whether acoramidis can delay the onset of or prevent the development of ATTRv disease in individuals who carry a TTR genetic variant and who have not yet gotten sick from or been diagnosed with ATTRv disease.

02

Understand how early treatment, before ATTRv disease has been diagnosed, can impact and possibly prevent the onset of ATTRv.

03

Identify early signs of ATTRv disease through routine clinic visits that will include physical exams, imaging tests of your heart, nerve testing, and blood tests designed to screen for ATTRv.

ACT-EARLY Study Design

ACT-EARLY

Phase 3

Objective

Compare acoramidis to placebo to study if it can delay or prevent the development of ATTR disease; patients and doctors won’t know which one is given

Participants

Disease-free carriers of genetic mutations in the TTR gene that put them at elevated risk for ATTR disease; participants must be ages 18 to 75 and within 10 years of the expected age of disease-onset for people with their genetic mutation

Key Measures

Time to development of ATTR disease (ATTR-CM or ATTR-PN*); Eligible participants who develop ATTR-CM or both ATTR-CM and –PN are eligible to receive acoramidis in the open-label extension†

Enrolling

ACT-EARLY OLE

Open-label Extension

Objective

Study the safety and effectiveness of acoramidis over the long-term; all participants who qualify will receive acoramidis

Participants

Eligible participants who completed the Phase 3 ACT-EARLY study with the development of ATTR-CM or ATTR-CM and –PN.

Key Measures

Safety and efficacy at preventing cardiovascular morbidity and mortality

Enrolling

*ATTR-CM = ATTR cardiomyopathy; ATTR-PN = ATTR polyneuropathy

†subjects who develop ATTR-PN alone are not eligible for OLE and should seek standard of care from their physician

Key Study Details

  • study duration

    Up to 7 years

  • Screening

    Health and genetic history evaluation

  • Study Treatment

    Acoramidis or placebo taken orally twice daily

  • Follow-up

    Routine monitoring about every 6 months including physical exams, imaging of your heart, nerve testing, and blood tests

  • Start Date

    May 2025

  • primary completion (estimated)

    October 2031
Click to zoom

ACT-EARLY Locator

Use the interactive map to explore participating ACT-EARLY study locations across the United States and internationally.

Map loading...