RecruitingPHASE1, PHASE2INTERVENTIONAL

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Doctors are studying a new medicine called ALXN2350 for adults who have a heart condition known as BAG3-associated dilated cardiomyopathy. This condition means the heart muscle is stretched and thin, making it harder to pump blood. This study is in its early stages (Phase 1/2), which means researchers are checking how safe the medicine is, if people can take it without too many problems, and if it shows any signs of helping the heart. Participants will receive one dose of ALXN2350 into a vein. The main goal is to find the right dose and understand the effects of this new treatment. It's for adults aged 18 to 70 who meet certain health criteria.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

Start

24 Oct 2025

Estimated completion

27 Jan 2032

BAG3 Mutation Associated Dilated Cardiomyopathy

What is this study about?

This research study is about a new treatment, ALXN2350, for a specific heart problem called BAG3-associated dilated cardiomyopathy. This condition affects your heart muscle, making it weaker and enlarged, which means it struggles to pump blood effectively around your body. It's caused by a change (mutation) in a gene called BAG3. When your heart can't pump blood well, it can lead to symptoms like tiredness, shortness of breath, and swelling.

The study is in an early stage, called Phase 1/2. This means it's one of the first times this new treatment is being tested in people. The main aims are to see if the treatment is safe, if people can tolerate it without serious side effects, and to look for early signs that it might be working to help the heart. Researchers will also be trying to figure out the best amount of the medicine to give.

Participants in the study will receive one dose of ALXN2350 directly into a vein. This study is 'open-label,' which means both you and the study doctors will know you are receiving the active treatment. This research is important because it could lead to a new way to treat this specific type of heart condition, potentially offering a new option for people who currently rely on other heart medications.

Key takeaways

This study is for adults (18-70) with a heart condition called BAG3-associated dilated cardiomyopathy.
It's an early-stage study to check the safety and potential benefits of a new medicine, ALXN2350.
Participants will receive one dose of ALXN2350 into a vein.
Regular health checks and heart scans will be part of the study.
You must be on stable heart failure medication to be considered.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

For you to be considered for this study, you need to be an adult between 18 and 70 years old. You must have been diagnosed with BAG3-associated dilated cardiomyopathy, which means tests have shown a specific change in your BAG3 gene and your heart muscle is weakened and enlarged.

It's important that you are already taking a stable combination of your usual heart failure medications, as agreed with your doctor. Doctors also need to be able to get clear pictures of your heart using an ultrasound (echocardiogram) so they can monitor its health throughout the study.

There are also reasons why you might not be able to join. For example, if your body already has certain antibodies (natural defences) against a common virus called AAV9, you won't be able to take part. Also, if you have another known serious genetic cause for your dilated cardiomyopathy, or if your heart failure is suddenly much worse (decompensated heart failure), you would not be eligible. Other health conditions may also prevent participation.

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.

Are you between 18 and 70 years old?
Do you have a diagnosis of BAG3-associated dilated cardiomyopathy?
Are you currently taking a stable set of heart failure medications?
Are you able to have clear heart scans (echocardiograms)?
Have you been told you do not have antibodies to the AAV9 virus?
Is your heart failure currently stable and not getting suddenly worse?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive one dose of the study medicine, ALXN2350, into a vein. Before and after this, you'll have regular visits to the study clinic. During these visits, the study team will carry out various check-ups to see how you're doing. This will include physical exams, blood tests, heart scans (echocardiograms), and other tests to check your heart health and general well-being.

You'll need to answer questions about your health and any symptoms you experience. The study team will carefully monitor you for any side effects. The total duration of your involvement in the study will last for a certain period, involving several follow-up appointments after you receive the medicine. You will be told the exact schedule of visits and the total expected time commitment before you decide to join.

Potential risks and benefits

Taking part in a study like this might offer some potential benefits, such as access to a new treatment that isn't yet widely available, and close monitoring of your heart health by medical experts. However, there are also potential risks, including side effects from the study medicine, or that the treatment may not help your condition. The study team will explain all known or expected risks in detail. Remember, participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (10)

Research Site
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Birmingham, United States· Recruiting
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Boston, United States· Recruiting
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Cincinnati, United States· Recruiting
Research Site
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Portland, United States· Recruiting
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Philadelphia, United States· Recruiting
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Houston, United States· Recruiting
Research Site
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Massa, Italy· Recruiting
Research Site
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Barcelona, Spain· Recruiting
Research Site
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Majadahonda, Spain· Recruiting
Research Site
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London, United Kingdom· Recruiting

Common questions

What is BAG3-associated dilated cardiomyopathy?

It's a heart condition where the heart muscle becomes stretched and thin, making it harder for the heart to pump blood. It's caused by a specific change in a gene called BAG3.

What is ALXN2350?

ALXN2350 is a new medicine being tested in this study to see if it can help improve the heart function in people with BAG3-associated dilated cardiomyopathy.

Will I know if I'm getting the actual medicine or a dummy treatment?

In this specific study, it's an 'open-label' study, which means both you and the study doctors will know that you are receiving the ALXN2350 medicine.

How will I receive the medicine?

You will receive the ALXN2350 medicine as a single dose given directly into a vein (an 'IV infusion').

Can I stop participating in the study if I change my mind?

Yes, you can choose to leave the study at any time, for any reason, and it will not affect your usual medical care.

How to find out more

Alexion Pharmaceuticals, Inc. (Sponsor)

clinicaltrials@alexion.com 1-855-752-2356 Official trial record

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