RecruitingPHASE3INTERVENTIONAL

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

This study is investigating a new medication called acoramidis. It's for people who have a faulty gene that tends to cause a condition called transthyretin amyloidosis (ATTR). In ATTR, a protein known as TTR starts to fall apart and forms 'sticky plaques' in various body parts, including the heart and nerves, causing damage. Acoramidis is designed to keep this protein stable. The study aims to find out if taking acoramidis early, before any symptoms appear, can prevent or delay the heart and nerve problems associated with ATTR. This is a crucial step to see if early treatment can make a big difference in people's lives.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Eidos Therapeutics, a BridgeBio company

Enrolment target

587

Start

12 May 2025

Estimated completion

01 Dec 2032

Amyloidosis Amyloid Cardiomyopathy Transthyretin Amyloidosis Cardiomyopathies Heart Diseases Polyneuropathies

What is this study about?

Imagine your body has tiny building blocks called proteins. One of these proteins, called TTR, sometimes breaks down and forms sticky clumps, a bit like glue. These clumps can then gather in different parts of your body, like your heart and nerves, causing them trouble. This condition is called transthyretin amyloidosis (ATTR).

Some people inherit a faulty gene that makes their TTR protein more likely to break down. This study is for these individuals who have this faulty gene but haven't started to feel unwell or show any signs of the disease yet. The study wants to see if a new medication, called acoramidis, can help. Acoramidis works by holding the TTR protein together, hoping to stop it from breaking down and forming these harmful clumps.

The main goal is to find out if taking acoramidis early can prevent or delay these problems from starting, especially those affecting the heart (which can lead to heart failure) or the nerves (which can cause nerve damage). If successful, this could mean that people with this faulty gene could start treatment before they get sick, potentially avoiding or delaying serious health issues.

Key takeaways

This study is for people with a genetic risk for ATTR, but no symptoms.
It tests if a drug called acoramidis can prevent heart or nerve problems.
Participants will receive either acoramidis or a placebo.
Regular health checks, including heart scans, will be part of the study.
Taking part helps research and might benefit your health.

Who may be eligible?

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.

Quick self-check

Am I aged between 18 and 75 years?
Do I have a confirmed faulty TTR gene from a genetic test?
Do I currently have no symptoms or signs of ATTR heart or nerve problems?
Am I roughly within 10 years of the typical age of onset for ATTR in my family or genetic type?
Am I not currently taking other similar ATTR-preventing medications?
Do I not have any serious heart, kidney, or liver conditions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

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.

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might benefit from the study drug helping to prevent or delay the ATTR condition, or you might help researchers learn more about this disease, which could help others in the future. However, there's always a chance the drug might not work for you, or you might experience side effects, though doctors will monitor you closely for these. You will be told all known risks before you decide to join. It's very important to remember that you can decide to leave the study at any time, for any reason, without it affecting your medical care.

Locations (101)

University of California, San Diego (UCSD) - Medical Center
La Jolla, United States· Recruiting
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, United States· Recruiting
University of California, San Francisco (UCSF)
San Francisco, United States· Recruiting
Stanford University
Stanford, United States· Recruiting
University of Colorado Anschutz
Aurora, United States· Recruiting
Yale University School of Medicine - Section of Cardiology
New Haven, United States· Recruiting
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., United States· Recruiting
Mayo Clinic - Jacksonville
Jacksonville, United States· Recruiting
Cleveland Clinic Florida
Weston, United States· Recruiting
Emory University School of Medicine
Atlanta, United States· Recruiting
John H. Stroger, Jr. Hospital of Cook County
Chicago, United States· Recruiting
University of Chicago - Medical Center
Chicago, United States· Recruiting

+89 more sites — see the official record for the full list.

Common questions

What is Transthyretin Amyloidosis (ATTR)?

It's a condition where a protein in your body breaks down and forms sticky clumps that can damage organs like the heart and nerves.

What does 'asymptomatic carrier' mean?

It means you have a faulty gene that could cause ATTR, but you don't have any symptoms or signs of the disease yet.

What is acoramidis?

It's an experimental drug designed to stop the protein from breaking down and causing those harmful clumps.

Will I know if I'm getting the real drug or a dummy pill?

No, this is a 'blinded' study, meaning neither you nor your doctor will know what you are receiving. This ensures the results are as accurate as possible.

How long will I need to be in the study?

The study involves ongoing treatment and check-ups over a period of time, which your doctor can explain in more detail.

How to find out more

Medical Information

medinfo@eidostx.com 1-844-550-2246 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.