Not yet recruitingPHASE2INTERVENTIONAL

A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM.

This research study is looking at a new combination of treatments for a heart condition called Transthyretin Amyloid Cardiomyopathy, or ATTR-CM. Specifically, it's comparing two medicines used together: eplontersen and ALXN2220, against eplontersen and a placebo (a dummy treatment). The main goal is to find out if this new combination is safe and effective in improving the condition for adults living with ATTR-CM. Participants will be randomly assigned to one of these groups and will receive their assigned treatment every four weeks. They'll also take vitamin A daily. This is a Phase 2 study, meaning it's exploring the treatment's effectiveness and safety in a larger group of people.

At a glance

Status

Not yet recruiting

Phase

PHASE2

Sponsor

AstraZeneca

Enrolment target

326

Start

30 Jun 2026

Estimated completion

23 Feb 2029

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

What is this study about?

This study is looking into new ways to treat a serious heart condition called Transthyretin Amyloid Cardiomyopathy, often shortened to ATTR-CM. In ATTR-CM, a protein called transthyretin doesn't fold correctly and builds up in the heart, making it stiff and unable to pump blood properly. This can lead to symptoms like breathlessness and swelling.

The researchers want to see if combining two different medicines, eplontersen and ALXN2220, works better than using eplontersen with a placebo (a 'dummy' treatment that has no active medicine in it). Eplontersen is a medicine designed to reduce the amount of the faulty transthyretin protein, and ALXN2220 is another medicine that works in a different way to potentially help the heart. By comparing these combinations, the study aims to find out if this new approach can improve the health of people with ATTR-CM and is safe to use.

This is a 'double-blind' study, which means neither you nor your study doctor will know whether you are receiving ALXN2220 or the placebo. This helps ensure that everyone involved remains unbiased about the results. The study is also 'randomised,' meaning participants are put into groups by chance. This helps make sure the groups are similar and that the results are reliable. It's a Phase 2 study, which means the treatments have already been tested for basic safety in smaller groups, and now researchers are looking more closely at how well they work and their side effects in a larger number of patients.

Key takeaways

This study is testing two medicines for ATTR-CM: eplontersen and ALXN2220.
It aims to see if the combination of these two drugs is safe and helpful.
Participants will receive either ALXN2220 or a placebo, alongside eplontersen.
You will also need to take a daily vitamin A supplement.
This is a 'double-blind, randomised' study, meaning neither you nor your doctor will know which treatment you are receiving.

Who may be eligible?

To join this study, you need to be an adult between 18 and 85 years old and have been diagnosed with ATTR-CM. Your ATTR-CM diagnosis needs to be confirmed in specific ways, such as a heart biopsy or special heart scans, as outlined by the doctors.

You also need to be able to comfortably participate in certain tests, like exercise tests, and your heart condition should be stable enough, as judged by the study doctor. There are specific measurements, like certain protein levels in your blood and heart wall thickness, that will be checked to see if you fit the study criteria. You shouldn’t have other types of amyloidosis, a condition that also causes protein build-up, and you must be willing to take daily vitamin A supplements.

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 85 years old?
Do you have a confirmed diagnosis of ATTR-CM?
Are you able to undergo exercise tests?
Are you willing to take a daily vitamin A supplement?
Do you *not* have other specific types of amyloidosis?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned by chance (randomised) to one of two groups. One group will receive eplontersen along with ALXN2220, and the other group will receive eplontersen with a placebo. Both treatments will be given once every four weeks. You will also need to take a daily vitamin A supplement (3000 IU).

Throughout the study, you'll have regular visits to the clinic for various assessments, such as blood tests, heart scans, and exercise tests, to monitor your health and how you respond to the treatment. The exact number of visits and their duration will be explained by the study team. They will track your progress and any side effects. The total length of your participation in this study will also be explained in detail.

Potential risks and benefits

Participating in a study means you might receive a new treatment that could potentially help your condition more than current options. However, there's no guarantee it will work for you, and sometimes new treatments have side effects that aren't fully known yet. You might also experience side effects from taking vitamin A. It's important to remember that you can stop being part of the study at any time, for any reason, without it affecting your usual medical care. The study team will explain all known risks and benefits in detail before you decide to join.

Locations (78)

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

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La Jolla, United States
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San Francisco, United States
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Stanford, United States
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Aurora, United States
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Washington D.C., United States
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Jacksonville, United States
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Miami, United States
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Weston, United States
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Chicago, United States
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Boston, United States
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Kansas City, United States
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St Louis, United States

Common questions

What is ATTR-CM?

ATTR-CM is a heart condition where a faulty protein builds up in the heart, making it stiff and weaker over time.

What is a 'placebo'?

A placebo is a substance or treatment that looks exactly like the real medicine but contains no active ingredients. It helps researchers compare the true effects of the drug.

Why is vitamin A needed in this study?

The study mentions daily vitamin A supplements. The study team will explain why this is a part of the treatment plan for trial participants.

How long will I be in the study?

The exact length of your participation will be discussed with you by the study team, as it varies depending on the trial design.

Will I know which treatment I'm getting?

No, this is a 'double-blind' study, meaning neither you nor your doctor will know if you're receiving ALXN2220 or the placebo to keep the results fair.

How to find out more

AstraZeneca Clinical Study Information Center

information.center@astrazeneca.com 1-877-240-9479 Official trial record

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