A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis
This study is investigating a new medication called birtamimab for a serious heart condition known as AL amyloidosis. This condition involves abnormal proteins building up in the heart and other organs. The study aims to find out if birtamimab, when given alongside the usual treatment, can improve how long people live. Participants will be split into groups; one group will receive birtamimab and regular care, while the other will receive a dummy treatment (placebo) and regular care. Researchers will also look at how far people can walk in six minutes and how their quality of life changes. The study is a carefully designed 'Phase 3' trial, which means it's one of the final steps before a new medicine might become widely available.
At a glance
What is this study about?
This study is for people living with a serious heart condition called Light Chain (AL) Amyloidosis. In this condition, abnormal proteins can build up in organs, especially the heart, making it harder for them to work properly. Researchers are testing a new medicine called birtamimab alongside the usual care you might already be receiving. The main goal is to see if adding birtamimab helps people live longer.
To make sure the study results are fair and accurate, participants will be randomly assigned to one of two groups. One group will receive birtamimab plus their standard care, and the other group will receive a 'placebo' (a dummy treatment that looks like birtamimab but contains no active medicine) plus their standard care. Neither you nor your doctor will know which treatment you are receiving, which helps prevent bias in the results. This way, we can be more certain if any positive changes are truly due to birtamimab.
Besides looking at how long people live, the study will also check other important things. For example, researchers will measure how far you can walk in six minutes, which helps understand your physical ability. They will also look at your quality of life using a special questionnaire to see if the treatment helps you feel better overall. These additional checks give a more complete picture of how the treatment affects people's lives.
Key takeaways
- This study is testing a new drug, birtamimab, for AL amyloidosis.
- It aims to see if birtamimab helps people live longer.
- Participants will receive either birtamimab or a placebo, plus standard care.
- Your physical ability and quality of life will also be checked.
- The study involves regular hospital visits for treatments and tests.
Who may be eligible?
To be part of this study, you need to be at least 18 years old. Both men and women are welcome to take part.
Specifically, the study is designed for individuals who have been diagnosed with a certain serious stage of heart damage from AL amyloidosis, referred to as 'Mayo Stage IV'. This means your condition would have been assessed by doctors to fit specific criteria.
There might be other health conditions or medications that would prevent someone from joining the study, as researchers need to ensure it's safe for everyone involved. Your doctor would help determine if this study is a good fit for you.
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.
- Am I 18 years old or older?
- Have I been diagnosed with Light Chain (AL) Amyloidosis?
- Has my doctor told me I have 'Mayo Stage IV' amyloidosis?
- Am I able to attend regular hospital visits?
What does participation involve?
If you decide to participate, you would receive either the study drug (birtamimab) or a placebo, in addition to your current standard care for AL amyloidosis. This medication would be given through an infusion, which is like a drip into your arm.
You would have regular visits to the hospital or clinic for these infusions and for various tests. These tests might include blood tests, heart checks, and assessments of your walking ability to see how you are responding to treatment. You would also fill out questionnaires about how you are feeling and your quality of life.
The study would continue for a period, during which you would regularly receive your assigned treatment and have these check-ups. The total time you would be involved in the study would be explained to you by the research team.
Potential risks and benefits
Locations (14)
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Common questions
What is AL amyloidosis?
It's a serious condition where abnormal proteins called amyloid build up in organs like the heart, making them not work as well.
What is birtamimab?
It's a new medication being tested in this study to see if it can help people with AL amyloidosis.
What does 'placebo' mean?
A placebo is a dummy treatment that looks exactly like the study drug but contains no active medicine. It helps researchers fairly compare the new drug's effects.
Will I know if I'm getting the real drug or the placebo?
No, neither you nor your doctors will know while the study is ongoing. This is done to make the results as accurate as possible.
How long will the study last for me?
The exact duration for your participation will be explained by the study team, but it involves regular visits over a period of time.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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