Not yet recruitingPHASE3INTERVENTIONAL

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy

This research study is about a new medicine called ALXN2220, designed for adults who have a specific heart condition called Transthyretin Amyloid Cardiomyopathy (ATTR-CM). This condition causes abnormal protein deposits to build up in the heart, making it harder for the heart to pump blood effectively. The main aim of this study is to see how safe and effective ALXN2220 is over a long period when added to standard care. This particular study is an 'open-label' study, meaning everyone involved, including the patients and doctors, knows they are receiving the study medicine. It is open to people who have already finished a previous study (Study 301) for the same medicine. The findings will help doctors understand more about this potential new treatment.

At a glance

Status

Not yet recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

1,180

Start

23 Nov 2027

Estimated completion

11 Jul 2030

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

What is this study about?

This clinical trial is looking into a new medicine called ALXN2220 for adults who have a heart condition known as Transthyretin Amyloid Cardiomyopathy, often shortened to ATTR-CM. This condition happens when a faulty protein called transthyretin builds up in the heart, making it stiff and difficult for it to work properly. The study aims to see how well ALXN2220 works in the long run and if it's safe for patients.

This specific study is a follow-on from an earlier study (Study 301) for the same medicine. This means that only people who have already participated in and completed Study 301 are eligible to join this new one. In this study, everyone involved – the patients and the doctors – will know that the patients are receiving the ALXN2220 medicine, alongside their usual heart care. This is often called an 'open-label' study.

The overall goal is to gather more information about ALXN2220, especially its long-term effects. The hope is that this research will help in understanding how to better treat ATTR-CM and improve the lives of people living with this condition.

Key takeaways

This study is testing a medicine called ALXN2220 for a heart condition called ATTR-CM.
It's a follow-on study for people who completed a previous study (Study 301) for the same medicine.
The main goals are to check the medicine's long-term safety and how well it works.
Participants will receive the ALXN2220 medicine alongside their usual care.
Your medical team will explain all the details, including potential benefits and risks.

Who may be eligible?

To be considered for this study, you must have already taken part in and finished an earlier study called 'Study 301' for the same medicine, ALXN2220. This means you would have completed all parts of that previous study, including the final check-up.

There are also some reasons why you might not be able to join. For example, if you stopped taking the study medicine in the previous study, or if you completely withdrew your consent from Study 301, you wouldn't be able to participate. Also, if you have known allergies to proteins or any ingredients in ALXN2220, or if you've had a heart transplant or certain heart device implants, you would not be able to join. Doctors will also check for any new or worsening health issues that might make it unsafe for you to take part.

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.

Have you already completed 'Study 301' for ALXN2220?
Did you continue taking the study medicine throughout 'Study 301'?
Do you have any known allergies to proteins or any ingredients in ALXN2220?
Have you had a heart transplant or certain heart device implants?
Are you willing and able to follow all study instructions and attend appointments?

Answer every question to see your result.

What does participation involve?

If you join this study, you will continue to receive the study medicine, ALXN2220, along with your usual heart care. Because this is a follow-on study, the exact details of what your participation involves will be explained by the study team, building on your experience from the previous study (Study 301). This will typically include regular visits to the clinic for health checks, blood tests, and other assessments to see how the medicine is working and to monitor your health. The study team will track your progress over a long period to understand the long-term effects of ALXN2220. They will give you full details about the number of visits, tests, and how long you will be involved.

Potential risks and benefits

Like all clinical studies, there are potential benefits and risks. The potential benefit is that ALXN2220 might help manage your ATTR-CM condition over the long term, potentially improving your health. However, the medicine may also have side effects, which the study team will discuss with you in detail. There might be some discomfort from tests like blood draws, and there's always a chance the medicine might not work for you. It's very important to remember that participating is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (195)

Research Site
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Irvine, United States
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La Jolla, United States
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Palo Alto, United States
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San Francisco, 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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Weston, United States
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Atlanta, United States
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Atlanta, United States
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Indianapolis, United States
Research Site
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Boston, United States

Common questions

What is ATTR-CM?

ATTR-CM stands for Transthyretin Amyloid Cardiomyopathy. It's a heart condition where faulty proteins build up in the heart, making it stiff and harder for it to pump blood properly.

What is ALXN2220?

ALXN2220 is the name of the new medicine being studied. Researchers want to see if it's a safe and effective treatment for ATTR-CM.

Who can join this study?

This study is specifically for adults who have already participated in and completed an earlier study (Study 301) for the same medicine, ALXN2220.

Will I know if I'm getting the actual medicine?

Yes, in this particular study, called an 'open-label' study, everyone involved knows that participants are receiving the ALXN2220 medicine.

How long will the study last?

This study is designed to look at the long-term effects of the medicine. The study team will provide you with the exact duration and schedule of visits.

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.