Not yet recruitingRare disease

A study to evaluate the safety and effectiveness of CABA-201, fludarabine, and cyclophosphamide in patients with active inflammatory myopathy

Fast replyRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on individuals with active Idiopathic Inflammatory Myopathy, a group of rare diseases that cause muscle inflammation and weakness. This category includes conditions such as dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, and Juvenile Idiopathic Inflammatory Myopathy, which is a similar condition that occurs in children. Some participants may have previously been treated with medications such as cyclophosphamide or fludarabine.

The purpose of the study is to evaluate the safety and effectiveness of a treatment called CABA-201. This treatment is a type of CAR T-cell therapy, which involves using specially modified immune cells to target specific proteins on the surface of certain cells in the body. This medication is administered through an intravenous infusion, which is a method of delivering medicine directly into a vein.

During the study, participants will undergo regular monitoring to track how they respond to the treatment. This includes checking for any adverse events, which are unexpected or unwanted medical problems. Healthcare providers will also monitor levels of B cells, which are a type of white blood cell, and muscle enzymes in the blood to assess muscle health. Additionally, the levels of autoantibodies, which are proteins produced by the immune system that mistakenly attack the body's own tissues, will be measured.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Study entry and administration of background medications
Upon joining the study, intravenous infusions, which are liquids delivered directly into a vein, are administered. these involve background medications such as cyclophosphamide and fludarabine.
Step 2
Administration of test therapy
The test treatment, known as CABA-201, is administered via intravenous infusion.
Step 3
Monitoring and evaluation
During the process, adverse events, which are any unexpected or harmful medical occurrences, are recorded to evaluate safety.
Blood tests are conducted to check the levels of B cells and CABA-201-positive T cells in the blood.
Blood samples are analyzed to monitor muscle enzymes, which are proteins that can indicate muscle damage, as well as autoantibodies, which are proteins produced by the immune system that mistakenly attack the body's own tissues.
vital signs, such as blood pressure and heart rate, are monitored.
An improvement score is used to track progress based on specific core measures.

Who can join the trial?

7 criteria

You must be between 6 and 75 years old.
You must have a confirmed diagnosis of Idiopathic Inflammatory Myopathy, which is a group of rare diseases that cause muscle inflammation and weakness.
Your diagnosis must specifically be one of the following: dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, or Juvenile Idiopathic Inflammatory Myopathy.
Your diagnosis must be confirmed by the presence of myositis-specific antibodies, which are special proteins in your blood that help doctors identify this specific type of muscle disease.
You must show signs of active disease, meaning the condition is currently ongoing, even if you are already taking standard medical treatments.
Active disease must be proven by one or more of the following: high levels of creatine kinase (an enzyme in the blood that increases when muscles are damaged), a skin rash associated with dermatomyositis, or evidence of muscle issues found through a muscle biopsy (taking a small piece of muscle to examine it), an MRI (a detailed medical picture of the inside of the body), or an electromyography (a test that measures the electrical activity in your muscles).
You must be experiencing muscle weakness.

Who cannot join the trial?

9 criteria

You cannot participate if you have a contraindication to leukapheresis, which means there is a specific medical reason or condition that makes it unsafe for doctors to perform a procedure that filters your blood to remove white blood cells.
You cannot participate if you have ever had a severe allergic reaction, such as anaphylaxis (a life-threatening allergic response), to the medicines fludarabine or cyclophosphamide or the substances they turn into in your body.
You cannot participate if you have an active infection that requires medical treatment at the time you are being checked for the study.
You cannot participate if you currently have serious or worsening symptoms of diseases affecting your kidneys (renal), liver (hepatic), blood (hematological), stomach or intestines (gastrointestinal), lungs (pulmonary), mental health (psychiatric), heart (cardiac), nerves (neurological), or brain (cerebral).
You cannot participate if you have serious infections like sepsis (a life-threatening reaction to an infection) or opportunistic infections (infections that happen more easily in people with weakened immune systems).
You cannot participate if you have significant problems with how your lungs or heart are working.
You cannot participate if you have previously received CAR T cell therapy, which is a type of treatment where a patient's own immune cells are changed in a lab to fight disease.
You cannot participate if you have previously had a solid organ transplant (such as a heart, liver, kidney, or lung) or a hematopoietic cell transplant (a transplant of blood-forming stem cells).
You cannot participate if you have any other medical conditions that the lead doctor believes could put you at too much risk, interfere with checking how safe the study drug is, or make the study procedures difficult.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
Responsive
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP
Frankfurt, Germany
Verified
Hospital Universitario De Navarra
Pamplona, Spain

Trial locations

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Investigated drugs

CABA-201 is an experimental therapy being tested in this study. It consists of specially prepared immune cells (T cells) taken from the patient's own body and modified to target and attack specific cells that have a protein called CD19. This therapy is administered through an intravenous infusion.

What is already known about the treatment

Cyclophosphamide monohydrate
This medication is a well-established chemotherapy agent administered as an intravenous infusion to treat various serious conditions, including certain autoimmune diseases. It works by entering cells and damaging the DNA, which prevents rapidly dividing cells from multiplying. As an alkylating agent, it is used to suppress an overactive immune system.
Fludarabine phosphate
This drug is a widely used medication in cancer treatment and immune disorders, delivered through intravenous infusion. It belongs to a class of drugs called purine analogs that work by mimicking the building blocks of DNA to stop cancer cells from growing. By interfering with the way cells copy their genetic material, it effectively reduces the number of harmful cells in the body.
CABA-201
This is an experimental therapy currently being studied in clinical trials for its potential to treat rare immune system diseases. It is administered via intravenous infusion and consists of specially engineered immune cells designed to target and remove specific cells marked by the CD19 protein. As a chimeric antigen receptor (CAR) T-cell therapy, it works by teaching the patient's own immune system to recognize and attack specific targets in the body.

Investigated diseases

Dermatomyositis
This is an inflammatory disease that causes muscle weakness and a distinctive skin rash. The condition often begins with weakness in the muscles closest to the trunk, such as the hips and shoulders. It can also involve skin changes, including red or purple rashes on the face or knuckles. The inflammation can affect various organ systems over time.
Anti-synthetase syndrome
This is a complex condition characterized by a group of symptoms including muscle inflammation and lung issues. It often presents with swollen hands, fever, and lung scarring. The disease involves the immune system attacking specific proteins within the body. It can progress through varying stages of muscle and respiratory involvement.
Immune-mediated necrotizing myopathy
This is a condition where the immune system causes muscle cell death. It leads to progressive and severe muscle weakness, often starting in the proximal muscles. The damage is characterized by significant inflammation and breakdown of muscle tissue. The condition typically worsens as the muscle fibers are destroyed.
Juvenile idiopathic inflammatory myopathy
This is a rare form of inflammatory muscle disease that occurs in children and adolescents. It involves chronic muscle weakness and inflammation caused by an overactive immune system. The disease can impact a child's physical development and daily activities. It often progresses by gradually reducing muscle strength.