Active not recruitingOBSERVATIONAL

A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

This study is looking into dilated cardiomyopathy (DCM), a condition where the heart muscle is weakened and enlarged, making it harder for the heart to pump blood effectively. Specifically, it focuses on DCM caused by a change in the BAG3 gene. The main goal is to understand how this particular type of DCM progresses naturally over time in adults. Participants will continue with their usual medical care while researchers observe their health. This will help doctors and scientists gain a better understanding of BAG3 DCM, which can then guide the development of new treatments and improve care in the future. The study will last for three years.

At a glance

Status

Active not recruiting

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

Start

14 Oct 2022

Estimated completion

12 Aug 2027

Cardiomyopathy, Dilated Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)

What is this study about?

This study is all about understanding a specific type of heart condition called Dilated Cardiomyopathy (DCM). DCM means your heart muscle has become weakened and enlarged, making it less effective at pumping blood around your body. This particular study is interested in DCM that is caused by a change, or mutation, in a gene called BAG3.

The main aim is to observe how this condition naturally develops and changes over time in adults. Think of it like watching a plant grow – the researchers want to see its natural progression without interfering. By carefully observing people with BAG3 DCM, the research team hopes to learn more about its unique characteristics and how it affects people's health.

This knowledge is really important because a better understanding of the condition can help doctors and scientists in the future. It could lead to developing new treatments, improving existing ones, or finding better ways to support people living with BAG3 DCM. Participants will continue to receive their normal medical care throughout the study.

Key takeaways

Study focuses on BAG3-related dilated cardiomyopathy (DCM).
Aims to understand the natural progression of the condition.
No new treatments – participants continue usual care.
Involves regular visits and medical tests over three years.
Contribution helps future understanding and potential treatments for BAG3 DCM.

Who may be eligible?

To be considered for this study, you would need to have been diagnosed with DCM that is known to be caused by a change in your BAG3 gene. Your heart condition should be stable, meaning you haven't recently had a severe worsening of heart failure, a heart attack, or major heart surgery within the last few months.

Your heart's pumping ability, often measured as 'ejection fraction,' would need to be reduced (50% or less). You should also be generally well enough to take part and not have other serious conditions that might significantly shorten your life or interfere with the study. You must be at least 18 years old.

There are also some reasons why you might not be able to join, such as if you've had a heart transplant, have very poor kidney function, or are already taking part in another new drug trial within a certain time frame. The researchers also aim to include a variety of people, so there's a limit on how many close family members can participate from the same family.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of Dilated Cardiomyopathy (DCM) caused by a BAG3 gene change?
Is your heart condition generally stable, without recent severe heart failure, heart attack, or major heart surgery?
Has your doctor told you that your heart's pumping ability (ejection fraction) is 50% or less?

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

What does participation involve?

If you join this study, you will be observed for three years. You'll continue to receive all your usual medical care and medications from your own doctors. The study itself doesn't involve new treatments, but rather carefully watching how your health progresses.

Over the three years, you'll visit the study site at least eight times. This means roughly every three months for the first year, and then once a year for the second and third years. During these visits, the study team will carry out various checks including a physical examination, heart scans (like MRI and echocardiogram), ECGs (which check your heart's electrical activity), tests to see how your heart and lungs work during exercise, and blood tests. You'll also wear a device to monitor your activity and answer questionnaires about your health and quality of life.

In addition to the visits, the study team will call you on the phone once during the study to check in. The total duration of your participation in the study will be three years.

Potential risks and benefits

Taking part in this study won't offer a direct medical benefit as you won't receive new treatments, but your regular health will be carefully monitored, which might be reassuring. The main benefit is knowing you're contributing to a better understanding of BAG3 DCM, which could help future patients. There are some risks associated with the tests, such as minor discomfort from blood tests or lying still for scans, or light activity during exercise tests. You have the right to withdraw from the study at any time without giving a reason, and this will not affect your usual medical care.

Locations (15)

Research Site
Palo Alto, United States
Research Site
Aurora, United States
Research Site
Boston, United States
Research Site
Rochester, United States
Research Site
The Bronx, United States
Research Site
Philadelphia, United States
Research Site
Amsterdam, Netherlands
Research Site
Maastricht, Netherlands
Research Site
Warsaw, Poland
Research Site
Majadahonda, Spain
Research Site
El Palmar, Spain
Research Site
A Coruña, Spain

+3 more sites — see the official record for the full list.

Common questions

What is BAG3 dilated cardiomyopathy?

It's a heart condition where the heart muscle becomes weakened and enlarged, making it harder to pump blood. This specific type is caused by a change in a gene called BAG3.

Will I receive new medicine in this study?

No, this study does not involve new treatments. You will continue with your usual medical care as prescribed by your own doctors.

How long will I need to be in the study?

The study will last for three years in total.

How many times will I have to visit the study site?

You will visit the study site at least eight times over the three years, mostly in the first year, and then annually.

What information will the study collect about me?

The study will collect information through physical exams, heart scans, ECGs, exercise tests, blood tests, activity monitoring, and questionnaires about your health and quality of life.