Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

This study is investigating a new treatment called CAEL-101 for a condition called AL amyloidosis, specifically in people whose hearts are significantly affected (Mayo Stage IIIb). AL amyloidosis happens when abnormal proteins build up in organs, like the heart, causing them not to work properly. CAEL-101 is designed to remove these harmful protein deposits. Researchers want to find out if adding CAEL-101 to standard treatment (CyBorD) can help people live longer and reduce hospital visits due to heart problems. They will also be checking if CAEL-101 is safe and causes few side effects. This is a Phase 3 study, meaning it's one of the final stages of testing before a medicine might become widely available.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

125

Start

25 Aug 2020

Estimated completion

22 Oct 2027

AL Amyloidosis

What is this study about?

AL amyloidosis is a rare but serious condition where special proteins, called amyloid, build up in different organs of the body, preventing them from working as they should. Think of it like a sticky substance clogging up important machinery. In this study, we're focusing on people whose hearts are particularly affected by these deposits, which can lead to serious health problems and hospital stays. The amyloid deposits start from abnormal cells in the bone marrow.

The study is looking at a new medicine called CAEL-101. This medicine is a type of antibody, which means it's designed to target and help remove these harmful amyloid deposits from organs. Researchers want to see if adding CAEL-101 to the usual treatment for AL amyloidosis (which is often a combination of medicines called CyBorD) can make a real difference for patients. They hope it might help people live longer, reduce the number of times they need to go into hospital for heart-related issues, and generally be a safe treatment.

This is a 'Phase 3' study, which means it's a large, important step in testing a new medicine. It's designed to show if CAEL-101 is effective and safe before it can be considered for wider use. Participants will either receive CAEL-101 plus standard treatment or a placebo (a dummy medicine with no active ingredients) plus standard treatment. This way, researchers can accurately compare the effects of CAEL-101.

Key takeaways

This study evaluates CAEL-101 for severe AL amyloidosis affecting the heart.
CAEL-101 aims to remove harmful protein deposits from organs.
It's a Phase 3 study, comparing CAEL-101 + standard treatment vs. placebo + standard treatment.
Participation involves regular hospital visits, infusions, and long-term monitoring.
Potential benefits include access to a new treatment, but risks and side effects are possible.
You can withdraw from the study at any time without penalty.

Who may be eligible?

To join this study, you would need to be an adult (18 years or older) with a specific advanced stage of AL amyloidosis that significantly affects your heart. Doctors classify this as Mayo Stage IIIb. This means your heart has been really impacted by the amyloid deposits, and certain blood tests show a high level of a marker called NT-proBNP.

You also need to have measurable signs of the abnormal cells that cause AL amyloidosis in your blood, and your doctors must have confirmed the presence of amyloid deposits in your tissues, usually through a biopsy. Importantly, you should be planning to start or currently be receiving a specific combination of standard treatments called CyBorD for your condition, as the study medicine will be given alongside this.

There are also some other medical details involved, so the best way to find out if this study might be right for you is to talk to your doctor. They can check all the criteria against your current health and medical history.

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 18 years old or older?
Have you been diagnosed with advanced AL amyloidosis that affects your heart (Mayo Stage IIIb)?
Are you planning to receive or currently receiving CyBorD type treatment?
Have your doctors confirmed AL amyloidosis through specific tissue and blood tests?
Are you able to attend regular hospital visits and receive intravenous infusions?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be randomly assigned to one of two groups. One group will receive the new study medicine, CAEL-101, along with the standard treatment (CyBorD). The other group will receive a placebo (a dummy medicine that looks like CAEL-101 but has no active ingredients) along with the standard treatment. Neither you nor your study doctor will know which treatment group you are in, to make sure the results are as fair as possible.

You will have regular hospital visits for assessments, which will include blood tests, heart checks, and other medical exams. You will receive the study medicine (CAEL-101 or placebo) as an intravenous infusion (given into a vein, usually in your arm). These infusions will be given over a period of time, and the frequency will be decided by the study doctors. The study will follow patients for quite a long time, from when you start until the study officially ends or until specific events happen, such as a heart transplant or other serious outcomes. The main part of the study is expected to last at least 18 months after the last patient starts treatment, but follow-up could continue for longer.

Potential risks and benefits

Participating in this study might offer potential benefits, such as potentially receiving a new treatment (CAEL-101) that could improve your condition or contribute to a better understanding of AL amyloidosis. However, there's no guarantee that you will personally benefit. Like all medicines, CAEL-101, or even standard treatments, can have side effects, and some may be serious. You will be closely monitored for any reactions or health changes. It's important to remember that participation is completely 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 (90)

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

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Duarte, United States
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Palo Alto, United States
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San Francisco, United States
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Weston, United States
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Indianapolis, United States
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New Orleans, United States
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Boston, United States
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Boston, United States
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Boston, United States
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Detroit, United States
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Rochester, United States
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St Louis, United States

Common questions

What is AL amyloidosis?

It's a condition where abnormal proteins build up in your body's organs, like the heart, making them struggle to work properly.

What is CAEL-101?

It's a new medicine being tested. It's an antibody designed to help remove the harmful protein deposits from your organs.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the real treatment but contains no active ingredients. It's used to compare against the active drug.

How long will I be in the study?

The main part of the study will last at least 18 months, but you might be followed for longer, until the study officially concludes.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, and it won't affect the medical care you receive.