Active not recruitingPHASE3INTERVENTIONAL

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

This study is an extension to an earlier trial, focusing on the long-term safety of a treatment called acoramidis (AG10) for people with a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). This condition causes abnormal protein deposits in the heart, leading to heart failure. Participants in this study will have already completed a previous 30-month study of acoramidis. The aim is to see how safe the drug is over a longer period when taken alongside standard heart failure treatments. Researchers will also observe if the drug continues to help with the condition. It's an 'open-label' study, meaning everyone knows they are receiving acoramidis.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Eidos Therapeutics, a BridgeBio company

Enrolment target

389

Start

27 Oct 2021

Estimated completion

15 May 2028

Amyloid Cardiomyopathy, Transthyretin-Related

What is this study about?

This study is all about a heart condition called transthyretin amyloid cardiomyopathy, often shortened to ATTR-CM. This condition happens when abnormal proteins build up in the heart, making it stiff and less able to pump blood properly, which can lead to heart failure symptoms like breathlessness and tiredness. Scientists are testing a medication called acoramidis (AG10) to see if it can help.

This isn't the first time acoramidis has been studied. This particular study is for people who have already taken part in a longer, earlier study (called ATTRibute-CM) that lasted 30 months. Think of it as a follow-up – now that the main part of the earlier study is complete, researchers want to continue to monitor the safety of acoramidis over an even longer period. This study is 'open-label,' which means all participants will know they are receiving acoramidis, unlike some studies where people might receive a dummy treatment.

The main goal is to check the long-term safety of acoramidis when patients are also taking their usual heart failure medications. Researchers will carefully watch for any side effects and how well people tolerate the treatment. They will also gather more information on how effective the drug is and explore other aspects of the condition and its treatment. This kind of research helps doctors understand new medicines better and ensures they are as safe and helpful as possible for people living with ATTR-CM.

Key takeaways

This study is a follow-up for people who already finished a 30-month study of acoramidis (AG10) for ATTR-CM.
The main goal is to understand the long-term safety of acoramidis.
Everyone in this study will know they are receiving acoramidis.
You cannot take other ATTR-CM medications or supplements while in this study.
Your health will be closely monitored throughout your participation.

Who may be eligible?

You might be able to join this study if you've already completed the full 30 months of the previous study (called AG10-301) and finished all the required health checks for that study. You also need to be able to understand and sign the consent form to show you agree to take part.

There are some conditions that would stop you from joining. For example, if you've had a heart attack, certain heart procedures, a stroke, or a mini-stroke (TIA) recently (within the last 3 months). If you have a very unstable heart condition that the study doctor thinks makes it too risky for you, you wouldn't be able to join. Also, if you've had a heart or liver transplant in the last year, or if you're on a waiting list for one, or if you've had a heart pump installed, you wouldn't be able to participate. You also can't join if you have a different type of amyloidosis (called AL amyloidosis).

It's important that you are not taking certain other medications or supplements for ATTR-CM while in this study, including another approved drug called tafamidis. If you are a woman who could become pregnant, you'll need to use very reliable birth control during the study and for a month after stopping the medication. Men who could father a child will also need to use specific birth control methods.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Did I complete the full 30 months of the previous study (AG10-301)?
Do I have ATTR-CM with heart failure symptoms?
Am I willing to use specific birth control methods if necessary?
Have I had a heart attack, stroke, or heart transplant recently (in the last 3 months to 1 year)?
Am I open to not taking other ATTR-CM-specific medications or supplements during the study?

Answer every question to see your result.

What does participation involve?

If you join this study, your first visit for this study might be the same day you finish your final checks for the previous study (AG10-301). You would have taken your last dose of the previous study medication (which might have been acoramidis or a dummy pill) the night before. On your first day in this new study, after some initial health checks, you would start taking open-label acoramidis, meaning you and your doctor will know you are receiving the active drug.

During the study, you'll have regular visits to the clinic where doctors and nurses will monitor your health, perform various tests, and check for any side effects. You'll continue to take acoramidis and your usual heart failure medications. It's very important that you don't take any other medications, supplements, or participate in other clinical trials for ATTR-CM during this study. If you choose to take another ATTR-CM treatment, you would need to stop acoramidis and might be asked to leave the study. If you decide to stop participating at any point, you'll have a final check-up to ensure your safety.

Potential risks and benefits

Potential benefits of taking part in this study include continuing to receive acoramidis, which may help manage your ATTR-CM symptoms, and contributing valuable information that could help others with the same condition in the future. However, there's no guarantee you'll personally benefit. As with any medication, there are potential risks and side effects from acoramidis, though they are currently being studied. Your health will be closely monitored to identify any issues. You are free to withdraw from the study at any time for any reason, without it affecting your usual medical care.

Locations (72)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Cedars-Sinai Medical Center
Verified postcode
Los Angeles, United States
University of Colorado Hospital - Anschutz Medical Campus
Verified postcode
Aurora, United States
Northwestern University
Verified postcode
Chicago, United States
University of Chicago Medical Center
Verified postcode
Chicago, United States
NorthShore University Health System
Verified postcode
Evanston, United States
MedStar Medical Group Cardiology at Franklin Square
Verified postcode
Baltimore, United States
Boston University Medical Center General Clinical Research Unit
Verified postcode
Boston, United States
Mayo Clinic - Rochester
Verified postcode
Rochester, United States
Saint Luke's Hospital of Kansas City
Verified postcode
Kansas City, United States
Washington University
Verified postcode
St Louis, United States
New York University Langone Health
Verified postcode
New York, United States
Columbia University Irving Medical Center
Verified postcode
New York, United States

Common questions

What is 'open-label'?

Open-label means that both you and the study doctor will know that you are receiving the study medication, acoramidis, rather than a dummy pill.

Can I take other ATTR-CM medications?

No, you cannot take any other approved or research medications for ATTR-CM, including tafamidis, at any point during this study.

What is ATTR-CM?

ATTR-CM is a heart condition where abnormal proteins build up in the heart, making it stiff and affecting how well it pumps blood.

What if I decide to stop the study?

You can leave the study at any time. You would have a final check-up to ensure your health and safety.

How long will this study last for each person?

The information provided does not specify a total duration for each person in this particular extension study, only that it is a long-term safety monitoring study.