RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Active Juvenile Idiopathic Inflammatory Myopathy
Inflammatory myopathies are rare autoimmune diseases where the immune system mistakenly attacks healthy muscles, causing persistent weakness, fatigue, and pain. When standard medications fail to control the condition, new options are needed. The RESET-Myositis study is testing an experimental treatment called CABA-201. This is a type of CAR T-cell therapy, which uses your own living immune cells. Doctors collect white blood cells from your blood, modify them in a specialised laboratory to recognise and remove the harmful cells driving the disease, and return them through a vein. The trial is open to children aged 6 and older and adults up to 75 years old who still have active disease despite standard care. Participants receive a short course of chemotherapy to prepare their bodies, followed by the cell infusion and a 10-day hospital stay for close monitoring. Follow-up continues long-term to check safety and effectiveness.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
Inflammatory myopathies, often referred to simply as myositis, are long-term conditions where the immune system attacks muscle tissue. In healthy people, the immune system defends against infection. In people with myositis, specialised white blood cells called B cells help trigger inflammation that damages muscles, leading to muscle weakness, aching, joint pain, and sometimes skin rashes. While standard treatments such as steroids and immune-suppressing drugs help many people, others continue to struggle with disabling symptoms.
This trial is investigating whether a personalised treatment called CABA-201 can help reset the immune system. CABA-201 is a CAR T-cell therapy. To create it, doctors collect some of your own T cells (another type of infection-fighting white blood cell) and add a targeting tool to them. When placed back into your bloodstream, these altered cells seek out and clear away the faulty B cells causing your inflammation, giving your immune system a chance to recover.
Finding new ways to treat myositis is crucial for patients whose disease does not respond to regular medicines. If successful, CABA-201 could provide a long-lasting way to reduce muscle damage, restore strength, and improve quality of life for both adults and children living with this challenging illness.
النقاط الرئيسية
- Tests a personalised cell therapy (CABA-201) designed to reset the immune system.
- Open to adults and children aged 6 and older with active, hard-to-treat myositis.
- Uses your own modified white blood cells to target harmful immune cells.
- Involves a short course of chemotherapy followed by an approximate 10-day hospital stay.
- Follows participants for up to 15 years to track long-term safety and health.
من قد يكون مؤهلاً؟
This study is open to children aged 6 to 17 and adults aged 18 to 75 who have been diagnosed with an active inflammatory muscle disease. This includes conditions such as dermatomyositis, anti-synthetase syndrome, and immune-mediated necrotising myopathy.
To be eligible, you must have active signs of the illness—such as ongoing muscle weakness, high levels of muscle enzymes in your blood, rashes, or inflammation seen on scans or muscle tests—even though you have already tried standard treatments.
You will not be able to take part if you have a serious active infection, severe allergies to the chemotherapy drugs used in the trial, or health issues that make collecting your blood cells unsafe. The study team will carry out detailed checks to confirm whether joining is safe and suitable for you.
هل يمكن أن تناسبك هذه الدراسة؟
أجب عن هذه الأسئلة السريعة لترى ما إذا كنت مؤهلاً. هذا دليل فقط - الفريق البحثي هو الذي يتخذ القرار النهائي.
- I am between 6 and 75 years of age.
- I have a confirmed diagnosis of an inflammatory muscle disease (myositis).
- I still experience muscle weakness or active inflammation.
- My condition has not improved enough with standard treatments.
- I am able to stay in hospital for around 10 days and attend regular follow-up visits.
ماذا يتضمن المشاركة؟
Taking part begins with screening visits, where the team performs blood tests, heart checks, and scans to ensure you can safely join. Next, you will undergo a routine procedure called leukapheresis, where blood is drawn from a vein, passed through a machine that separates out the white blood cells needed to make CABA-201, and returned to your body.
While your cells are being prepared in a specialised laboratory, you will wait a few weeks. Five days before your infusion, you will receive two chemotherapy medicines (fludarabine and cyclophosphamide) through a drip. This pre-treatment temporarily clears room in your bone marrow so the new cells can multiply. You then receive CABA-201 through a drip on Day 1. Following this, you will stay in hospital for about 10 days so the medical team can watch you closely for any reactions. Afterwards, you will attend regular outpatient check-ups for 1 to 3 years, followed by long-term yearly safety monitoring for up to 15 years.
المخاطر والفوائد المحتملة
المواقع (5)
- Northern Care Alliance NHS Foundation Trustالمدينة فقطSalford, England
- Kings College Hospital NHS Foundation Trustالمدينة فقطلندن، إنجلترا
- National Hospital for Neurology and Neurosurgeryتقريبيلندن، إنجلترا
- مستشفى مانشستر الملكيتقريبيمانشستر، إنجلترا
- NIHR Great Ormond Street Hospital Clinical Research Facilityالمدينة فقطلندن، إنجلترا
أسئلة شائعة
Is CABA-201 a type of chemotherapy?
No. CABA-201 is a personalised cell therapy made from your own white blood cells. However, you will receive a short course of chemotherapy just before the infusion to prepare your body for the new cells.
Why do I need to stay in hospital for 10 days?
The hospital stay allows the medical team to monitor you round-the-clock for common early side effects, such as fevers or immune reactions, and treat them straight away if they happen.
Can children participate in this study?
Yes, children aged 6 to 17 who have active juvenile myositis that has not improved on standard treatments may be eligible to join.
Why does the study track participants for 15 years?
Because CAR T-cell therapies involve modifying genes inside cells, international health regulators require doctors to check in with participants for 15 years to ensure long-term safety.
What happens if I decide to withdraw from the trial?
You are free to stop participating at any time. Your decision will not change your relationship with your doctors or affect the standard NHS care you receive.
كيفية معرفة المزيد
Christina Shepherd
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نقاش
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