A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
This study is for people in the UK with a rare condition called AL amyloidosis, specifically when it has returned or hasn't responded to earlier treatments. Researchers are testing a new type of treatment called AZD0120, which is a cell therapy. The main goals of the study are to understand how safe this new treatment is and how well it works. This is a "Phase 1b/2" study, meaning it's still in the early stages of testing in humans, looking at basic safety, dosage, and initial signs of effectiveness. By taking part, volunteers could help scientists learn more about new ways to treat this challenging condition.
At a glance
What is this study about?
This study is looking at a new treatment called AZD0120 for people who have AL amyloidosis. AL amyloidosis is a rare condition where abnormal proteins build up in various organs, like the heart, kidneys, or liver, and can stop them from working properly. We're particularly interested in people whose AL amyloidosis has come back after treatment (relapsed) or hasn't improved with previous treatments (refractory).
The investigational treatment, AZD0120, is a type of "cell therapy." This means it uses modified cells from a patient's own body to fight the disease. The study is in an early stage (Phase 1b/2), which means we're carefully checking to see how safe this new treatment is and if it shows any signs of working. Understanding how safe and effective this treatment is will help us learn more about new ways to manage AL amyloidosis.
Taking part in this study could help researchers develop new and better treatments for AL amyloidosis in the future. The information gathered will be crucial in deciding if this new cell therapy has the potential to help patients with this condition.
Key takeaways
- Tests a new cell therapy (AZD0120) for AL amyloidosis.
- For people whose AL amyloidosis has returned or not responded to other treatments.
- Aims to check the treatment's safety and how well it works.
- Participation will involve regular clinic visits and monitoring.
- You can stop participating at any time.
- Could help develop future treatments for AL amyloidosis.
Who may be eligible?
To be considered for this study, you would need to have a confirmed diagnosis of AL amyloidosis that affects at least one organ in your body. Your AL amyloidosis must also have either returned after previous treatment or not responded to it, and there needs to be a measurable amount of the disease in your blood.
There are also some health requirements, such as generally being well enough to take part and not having certain other medical conditions. For example, you wouldn't be able to join if you have another type of amyloidosis, severe heart or lung problems, or certain types of blood cancer. You also can't have had similar cell therapies or certain other treatments in the recent past.
If you are a woman who could become pregnant, you would need a negative pregnancy test before starting treatment and agree to use effective birth control throughout the study. Men also need to agree to use effective birth control during 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.
- Do I have a confirmed diagnosis of AL amyloidosis?
- Has my AL amyloidosis returned or not responded to previous treatments?
- Is my AL amyloidosis currently affecting at least one organ?
- Am I generally well enough to participate in a study?
- Have I had certain other cell therapies or intense treatments recently? (If yes, you might not be eligible)
- Am I willing and able to attend all required study visits?
What does participation involve?
If you decide to participate in this study, you will receive the investigational treatment, AZD0120. This will involve regular visits to the study clinic for assessments. These assessments will likely include blood tests, physical examinations, and other tests to monitor your health and how the treatment is affecting you and your AL amyloidosis. You will need to carefully follow the study schedule and requirements.
The specific duration of your participation will depend on the study plan and your individual response, but there will be an initial treatment period followed by ongoing monitoring and follow-up visits. You will have a study team to guide you through the process, answer your questions, and monitor your well-being closely.
Potential risks and benefits
Locations (18)
- Research SiteVerified postcodePhoenix, United States· Recruiting
- Research SiteVerified postcodeSan Francisco, United States· Not yet recruiting
- Research SiteVerified postcodeTampa, United States· Recruiting
- Research SiteVerified postcodeBoston, United States· Recruiting
- Research SiteVerified postcodeDetroit, United States· Recruiting
- Research SiteVerified postcodeRochester, United States· Recruiting
- Research SiteVerified postcodeSt Louis, United States· Recruiting
- Research SiteVerified postcodeNew York, United States· Recruiting
- Research SiteVerified postcodeNew York, United States· Recruiting
- Research SiteVerified postcodeNew York, United States· Recruiting
- Research SiteVerified postcodeCleveland, United States· Recruiting
- Research SiteVerified postcodeNashville, United States· Recruiting
Common questions
What is AL amyloidosis?
AL amyloidosis is a rare condition where abnormal proteins build up in organs, which can stop them from working properly.
What is 'cell therapy'?
Cell therapy uses modified cells, often from your own body, to help fight the disease.
What does 'relapsed or refractory' mean?
It means your AL amyloidosis has either come back after treatment or hasn't responded to previous treatments.
Will I get the new treatment if I join?
Yes, if you meet the study criteria, you would receive the investigational treatment, AZD0120.
Can I stop participating in the study at any time?
Yes, you are free to withdraw from the study at any point without affecting your ongoing medical care.
How to find out more
Alexion Pharmaceuticals, Inc. (Sponsor)
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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