RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
This study is called RESET-Myositis and it's looking into a new treatment, CABA-201, for specific types of muscle conditions. These conditions, like dermatomyositis and juvenile idiopathic inflammatory myopathy, are when your immune system mistakenly attacks your own body, causing muscle weakness and inflammation. Researchers want to find out if CABA-201 is safe and if it helps improve these conditions in people whose current treatments aren't working well enough. It's an active study for both adults and children, where doctors will closely monitor participants to understand how the new treatment affects them. The goal is to find better ways to help people living with these challenging muscle conditions.
At a glance
What is this study about?
This research study, called RESET-Myositis, is investigating a new treatment named CABA-201 for a group of conditions that cause your muscles to become weak and inflamed. These conditions are sometimes called 'myositis' and happen when your body's immune system, which normally fights off germs, mistakenly attacks healthy tissues in your muscles. This can lead to symptoms like muscle pain, weakness, and sometimes skin rashes. Common types of these conditions include dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, and similar conditions in children.
Even though we don't fully understand why these conditions happen, we know that certain immune cells, called B cells, play a role in making the body attack itself. When current treatments aren't enough to control the condition, new approaches are needed. That's where CABA-201 comes in. It's a type of 'cell therapy' designed to target these B cells. Before receiving CABA-201, participants will also have a preparation treatment using two other medicines called fludarabine and cyclophosphamide.
The main goals of this study are to find out how safe CABA-201 is and how well it works in helping adults and children with these active muscle conditions. Researchers will be observing participants very carefully to see if the treatment reduces muscle weakness and inflammation. This research is important because it could lead to new and more effective ways to manage these challenging conditions and improve the lives of those affected.
Key takeaways
- This study is testing a new treatment called CABA-201 for certain muscle-weakening conditions.
- It's for both adults and children (ages 6 to 75) whose conditions are still active despite usual care.
- The main goals are to check if the new treatment is safe and if it helps improve symptoms.
- Participants will receive CABA-201 after a preparatory treatment and have regular health checks.
- Your safety and wellbeing are the top priority throughout the study.
- You can stop participating at any time without affecting your medical care.
Who may be eligible?
To join this study, we're looking for adults aged 18 to 75, or children aged 6 to 17, who have been diagnosed with certain muscle conditions like dermatomyositis, anti-synthetase syndrome, or immune-mediated necrotizing myopathy. It's important that your condition is still active, meaning you're still experiencing symptoms like muscle weakness, or signs of inflammation despite your current treatments. Doctors use tests like blood samples (looking at muscle enzymes), special scans (MRI), or muscle biopsies to confirm that your condition is active.
There are also some reasons why you might not be able to join. For instance, if you have certain other serious health problems affecting your heart, lungs, kidneys, or liver, or if you have a current infection that needs treatment. You also can't take part if you've had certain complex medical procedures in the past, such as an organ transplant or specific cell therapies.
The research team will carefully check your full medical history and current health to make sure that taking part in the study is safe for you. They will explain all the specific requirements in detail if you are interested.
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 between 6 and 75 years old?
- Have you been diagnosed with dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, or a similar childhood muscle condition?
- Is your condition still active, meaning you still have muscle weakness or other signs of inflammation, despite current treatments?
- Do you have good overall health without other serious medical conditions that might prevent you from joining?
- Have you never had a CAR T cell therapy or an organ/stem cell transplant?
What does participation involve?
If you decide to take part in this study, you would first go through a screening process to make sure it's suitable and safe for you. This involves detailed medical checks and tests. If you meet the criteria, you would then receive the study treatment, CABA-201, which is given after a preparation treatment using two other medicines. This treatment involves a single dose of CABA-201.
Throughout the study, you would have regular visits to the hospital or clinic. These visits will include various assessments such as blood tests, physical examinations, and possibly imaging scans like MRI, to check how you are responding to the treatment and to monitor your health. The medical team will be closely watching for any side effects and tracking the progress of your muscle condition.
The full duration of your participation in the study, including all treatments and follow-up assessments, will be explained in detail by the research team. This will likely involve a number of visits over a set period to ensure all information is collected safely and accurately.
Potential risks and benefits
Locations (35)
- University of California Irvine - Accepting Adult PatientsVerified postcodeOrange, United States· Recruiting
- University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile PatientsVerified postcodeSan Francisco, United States· Recruiting
- Children's Hospital Colorado - Accepting Juvenile PatientsVerified postcodeAurora, United States· Recruiting
- Mayo Clinic Florida - Accepting Adult PatientsVerified postcodeJacksonville, United States· Recruiting
- Johns Hopkins All Children's Hospital - Accepting Juvenile PatientsVerified postcodeSt. Petersburg, United States· Recruiting
- Children's Healthcare of Atlanta - Accepting Juvenile PatientsVerified postcodeAtlanta, United States· Recruiting
- Emory University - Accepting Adult PatientsVerified postcodeAtlanta, United States· Recruiting
- Ann & Robert H. Lurie Children's Hospital of Chicago - Accepting Young Adult and Juvenile PatientsVerified postcodeChicago, United States· Recruiting
- Northwestern Memorial Hospital - Accepting Adult PatientsVerified postcodeChicago, United States· Recruiting
- The University of Chicago Medical Center - Accepting Adult and Juvenile PatientsVerified postcodeChicago, United States· Recruiting
- University of Kansas Medical Center - Accepting Adult PatientsVerified postcodeKansas City, United States· Recruiting
- National Institutes of Health - Accepting Adult and Juvenile PatientsVerified postcodeBethesda, United States· Not yet recruiting
Common questions
What kind of muscle conditions are included in this study?
This study is for specific muscle conditions where your immune system attacks your muscles, such as dermatomyositis, anti-synthetase syndrome, and immune-mediated necrotizing myopathy, including similar conditions in children.
What is CABA-201?
CABA-201 is a new type of cell therapy being studied. It's designed to target specific immune cells in your body that are thought to be involved in these muscle conditions.
Will I receive other treatments during the study?
Yes, before receiving CABA-201, you will have a preparation treatment using two other medicines: fludarabine and cyclophosphamide.
Who can participate in this study?
Adults aged 18-75 and children aged 6-17 with an active form of these muscle conditions, who haven't responded well to standard treatments, may be eligible.
What is the main goal of the study?
The study aims to find out if CABA-201 is safe and effective in improving these muscle conditions in people whose current treatments aren't working as well as hoped.
How to find out more
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Always speak to your GP or specialist before deciding to take part in a study.
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