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

This study is looking for men and women between 18 and 75 years old. To join, you must have had a genetic test that shows you carry a specific faulty gene (called a TTR gene variant) that is known to cause transthyretin amyloidosis (ATTR). Importantly, you should not currently have any symptoms or signs of ATTR, either in your heart or nerves. Your age also needs to be roughly within 10 years (either younger or older) of the typical age when this condition usually starts in families with your specific faulty gene.

There are also some reasons why you might not be able to join. For example, if you are already taking other medicines that work in a similar way to acoramidis, or if you have certain other serious health conditions like significant kidney, liver, or heart disease. You also can't take part if you've recently had major surgery or are planning to have one soon, or if you have an allergy to acoramidis.

If you decide to take part in this study, you'll be given either the study drug, acoramidis, or a dummy pill (placebo) to take. You won't know which one you're receiving, and neither will your study doctor – this is done to make sure the results are fair. You'll have regular visits to the clinic where doctors will perform various health checks, including scans like heart MRI scans, blood tests, and nerve tests to see if the drug is working and to monitor your health. The study will involve ongoing treatment and check-ups over a period of time to see if acoramidis can prevent the disease from developing.