A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Subjects with Active Idiopathic Inflammatory Myopathy or Active Juvenile Idiopathic Inflammatory Myopathy
This study is looking into a new treatment called CABA-201 for people with certain muscle conditions known as active idiopathic inflammatory myopathy, which includes conditions like dermatomyositis, or active juvenile idiopathic inflammatory myopathy in children. These conditions cause muscle weakness and inflammation. The main goal is to find out if this new cell therapy is safe and what side effects it might have. Researchers will also check if it helps improve the disease by looking at things like muscle enzyme levels, special immune cells, and overall improvement scores. This is an early-stage study, meaning it's one of the first times this treatment is being tested in people.
At a glance
What is this study about?
This study is investigating a new treatment called CABA-201 for a group of conditions known as inflammatory myopathies. These are diseases where the body's immune system mistakenly attacks its own muscles, leading to weakness, pain, and sometimes skin rashes or other problems. This study is open to both adults with active idiopathic inflammatory myopathy (including specific types like dermatomyositis or anti-synthetase syndrome) and children with active juvenile idiopathic inflammatory myopathy.
CABA-201 is a type of 'cell therapy'. This means it uses your own immune cells, called T-cells, which are specially modified in the lab. These modified T-cells are designed to target and remove other specific immune cells (called B cells) that are thought to play a role in these muscle diseases. Before receiving CABA-201, participants will be given some standard medications (like cyclophosphamide and fludarabine) to prepare their body for the new cells. The study is designed in two phases, starting with a focus on safety and then looking at how well it works.
The main aim of this research is to see if CABA-201 is safe to use and what side effects it might cause. Researchers will also look at whether it helps improve the disease. They will do this by measuring various things in your blood, such as levels of muscle enzymes, specific immune cells, and signs of inflammation. They will also use a special scoring system to measure how much symptoms improve over time. This kind of research is very important for finding new ways to treat these challenging conditions.
Key takeaways
- This study is testing a new cell therapy (CABA-201) for muscle diseases.
- It's open to adults and children with active inflammatory myopathies.
- The main goals are to check if CABA-201 is safe and if it helps symptoms.
- Participants will receive medication, have cells collected, and then receive the modified cells.
- Regular clinic visits and blood tests will be part of the study.
- This is an early-stage study, so the treatment is still under investigation.
Who may be eligible?
To join this study, you generally need to be at least 18 years old or be a child with juvenile idiopathic inflammatory myopathy. Both men and women can take part.
Specifically, you must have an active form of idiopathic inflammatory myopathy. This includes conditions like dermatomyositis, anti-synthetase syndrome, or immune-mediated necrotizing myopathy if you are an adult. If you are a child, you must have active juvenile idiopathic inflammatory myopathy.
There will also be other specific health requirements that the study doctors will check to make sure the treatment is safe for you and that you are the right fit for 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 or older, or a child with juvenile idiopathic inflammatory myopathy?
- Do you have an active form of idiopathic inflammatory myopathy (e.g., dermatomyositis, anti-synthetase syndrome, or immune-mediated necrotizing myopathy for adults)?
- Are you female or male?
- Are you able to attend regular clinic visits and have blood tests?
What does participation involve?
If you decide to take part in this study, you will first undergo screening tests to see if you meet all the requirements. If you do, you will receive some medications (cyclophosphamide and fludarabine) through an injection or drip to prepare your body. Then, your own immune cells will be collected. These cells will be sent to a lab to be specially changed into CABA-201, which will then be given back to you through a drip.
Throughout the study, you will have regular appointments at the clinic. These visits will involve blood tests to check your overall health, the levels of the study drug, and how your immune system is responding. Doctors will also check your vital signs (like blood pressure and heart rate) and keep a close eye on any side effects or changes in your muscle disease symptoms. The exact duration of your participation and the number of visits will be explained by the study team, but it will involve a period of treatment followed by careful monitoring.
Potential risks and benefits
Locations (3)
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedGermany
Common questions
What is an 'inflammatory myopathy'?
It's a group of conditions where your body's immune system mistakenly attacks your muscles, causing weakness and inflammation.
What is CABA-201?
It's a new type of treatment called 'cell therapy'. It uses your own T-cells, which are modified in the lab to help fight the disease.
Will I have to stay in hospital?
The study involves receiving medications and the cell therapy, which usually means some time in the hospital, followed by regular clinic visits for monitoring.
What does 'Phase 1/2' mean?
It means this is an early stage of testing the treatment. Phase 1 focuses on safety, and Phase 2 looks at how well it works and more about safety.
Can children join this study?
Yes, children with active juvenile idiopathic inflammatory myopathy are eligible to take part in this study.
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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