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

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What is this study about?

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.

Who Can Join the Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsmedizin Goettingen Goettingen Germany
Hospital Universitario De Navarra Pamplona Spain

Other Sites

Site Name City Country Status
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP Frankfurt Germany
Hunfvtqq Vooa dfqnnyne Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not yet recruiting
01.04.2026
Italy Italy
Not yet recruiting
01.04.2026
Spain Spain
Not yet recruiting
01.04.2026

Trial locations

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.

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.

Trial ID:
2025-521145-24-01
Protocol code:
CAB-201-002
NCT ID:
NCT06154252
Trial Phase:
Human Pharmacology (Phase I) – Other

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