RecruitingPhase I/IIInterventional

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.

At a glance

Status

Recruiting

Phase

Phase I/II

Sponsor

Advanced Clinical

Enrolment target

Start

20 Dec 2023

Estimated completion

31 Dec 2026

Active Idiopathic Inflammatory Myopathy including dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, juvenile idiopathic inflammatory myopathy, juvenile dermatomyositis, juvenile polymyositis, juvenile myositis

What is this study about?

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.

Key takeaways

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.

Who may be eligible?

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.

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.

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.

Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

The main hope of this study is that CABA-201 will reduce muscle inflammation, improve strength, and help control your symptoms, though benefits cannot be promised. There are also important risks. The preparatory chemotherapy can cause hair loss, nausea, bladder irritation, and low blood counts, which increases your risk of serious infections. The cell infusion can trigger an immune reaction called cytokine release syndrome, causing severe flu-like illness with high fever and fast heartbeat, as well as temporary nervous system issues such as confusion or headaches. Taking part is completely voluntary, and you are free to leave the trial at any time without any impact on your routine medical care.

Locations (5)

Northern Care Alliance NHS Foundation Trust
City only
Salford, England
Kings College Hospital NHS Foundation Trust
City only
London, England
National Hospital for Neurology and Neurosurgery
Approximate
London, England
Manchester Royal Infirmary
Approximate
Manchester, England
NIHR Great Ormond Street Hospital Clinical Research Facility
City only
London, England

Common questions

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.

How to find out more

Christina Shepherd

cshepherd@advancedclinical.com +1 203 894-6508 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.