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 with 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.
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
Key Study Details
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study duration
Up to 7 years
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Screening
Health and genetic history evaluation
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Study Treatment
Acoramidis or placebo taken orally twice daily
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Follow-up
Routine monitoring about every 6 months including physical exams, imaging of your heart, nerve testing, and blood tests
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Start Date
May 2025
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primary completion (estimated)
October 2031
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