A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
This study is exploring a new medicine called CAEL-101 for people with a specific type of AL amyloidosis that affects the heart. AL amyloidosis happens when abnormal proteins build up in organs, like the heart, causing problems. CAEL-101 is designed to clear these harmful protein deposits. Researchers want to find out if CAEL-101, when given with standard treatments, helps patients live longer, reduces heart-related hospital visits, and is safe and well-tolerated. This is compared to receiving a dummy drug (placebo) alongside the standard treatment. The study involves about 267 patients and will follow them for an extended period.
At a glance
What is this study about?
AL amyloidosis is a serious condition where your body makes abnormal proteins. These proteins don't fold correctly and can't be broken down. Instead, they clump together to form sticky deposits called amyloid fibrils. These deposits can build up in various organs, including the heart, kidneys, liver, and nerves, stopping them from working properly. When it affects the heart, it can be very serious.
This study focuses on a new drug called CAEL-101. CAEL-101 is a type of medicine designed to help remove these amyloid deposits from your organs. The main goal is to see if adding CAEL-101 to your regular treatment can help people with severe heart AL amyloidosis live longer and reduce their chances of being hospitalised due to heart problems. Researchers also want to check if the new drug is safe and doesn't cause too many side effects.
Patients taking part will be divided into two groups by chance. One group will receive CAEL-101 along with their standard treatment (which often involves a combination of medicines called CyBorD). The other group will receive a placebo, which looks like CAEL-101 but contains no active medicine, also alongside their standard treatment. This double-blind approach helps ensure the results are fair and unbiased, as neither you nor your doctor will know which treatment you are receiving until the end of the study.
Key takeaways
- Tests a new medicine, CAEL-101, for heart-affected AL amyloidosis.
- Compares CAEL-101 plus standard care to a dummy drug plus standard care.
- Aims to see if CAEL-101 improves survival and reduces hospital stays.
- Designed to be 'double-blind' – neither you nor your doctor knows which treatment group you are in.
- Participation involves regular check-ups and monitoring.
- You can stop participating at any time.
Who may be eligible?
This study is for adults aged 18 or over with a specific type of AL amyloidosis that has damaged their heart. Your doctors would have already confirmed that you have stage IIIa AL amyloidosis using special tests, and your heart must show signs of being affected by the condition.
To join, doctors need to show that you have specific levels of abnormal proteins in your blood, which confirms your AL amyloidosis. You also need to be planning to start a standard treatment called CyBorD for your condition, as this study combines the new drug with this existing treatment.
There are also some other medical details that your doctors will check, including specific heart measurements and a confirmed diagnosis of AL amyloidosis using tissue samples. If you are a woman who could become pregnant, you would need to have a negative pregnancy test and agree to use effective contraception during and for several months after the study.
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 AL amyloidosis that affects your heart?
- Are you planning to start a specific standard treatment called CyBorD?
- Do you have specific abnormal protein levels in your blood as confirmed by your doctor?
- If you are a woman of childbearing age, are you willing to use effective contraception?
What does participation involve?
If you decide to take part in this study, you will be assigned to one of two groups by chance. You will either receive the new medicine, CAEL-101, or a dummy drug (placebo). Both groups will also receive the standard treatment for AL amyloidosis, which is usually a combination of medicines called CyBorD. Neither you nor your study doctor will know which treatment you are receiving.
Your participation will involve regular visits to the clinic for medical check-ups, blood tests, and other assessments to monitor your health and how the treatment is working. You will continue to receive treatment and be monitored for an extended period, until either the study ends, you pass away, or you have a heart transplant or a specific heart assist device fitted. The total duration of your involvement will depend on these factors and the overall length of the study.
Potential risks and benefits
Locations (110)
- Research SiteVerified postcodeScottsdale, United States
- Research SiteVerified postcodeDuarte, United States
- Research SiteVerified postcodePalo Alto, United States
- Research SiteVerified postcodeSan Francisco, United States
- Research SiteVerified postcodeJacksonville, United States
- Research SiteVerified postcodeWeston, United States
- Research SiteVerified postcodeIndianapolis, United States
- Research SiteVerified postcodeNew Orleans, United States
- Research SiteVerified postcodeBaltimore, United States
- Research SiteVerified postcodeBoston, United States
- Research SiteVerified postcodeBoston, United States
- Research SiteVerified postcodeBoston, United States
Common questions
What is AL amyloidosis?
It's a serious condition where abnormal proteins build up in organs, like the heart, causing them to not work properly.
What is CAEL-101?
It's a new experimental medicine designed to help remove the harmful protein deposits from your organs.
What is a 'placebo'?
A placebo is a dummy drug that looks like the real medicine but contains no active ingredients. It's used to compare against the new drug.
Will I know if I'm getting the new drug or the placebo?
No, this is a 'double-blind' study, meaning neither you nor your doctors will know which treatment you are receiving until the study concludes.
Can I leave the study if I change my mind?
Yes, you can withdraw from the study at any time without having to give a reason, and it won't affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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