CABA-201 in JIIM - Clinical Trial

What is the Purpose of this Study?

Participants will take part in the main study, including screening, treatment, and follow up, for about 3 years. After receiving the study treatment, they will continue in a long term safety monitoring program for an additional 15 years so researchers can track their health and any delayed side effects.

What is the Condition Being Studied?

Idiopathic Inflammatory Myopathy (IIM) and Juvenile Idiopathic Myopathy (JIIM)

Who Can Participate in the Study?

Participants may be able to join if they are 6 to 17 years old and have a definite or probable inflammatory muscle disease, including juvenile inflammatory muscle disease. They must have blood test results showing certain disease-related antibodies and signs of active disease, such as abnormal blood tests, MRI findings, muscle biopsy results, or a skin rash related to the disease.

Age Group
Adults, Children

What is Involved?

This study is testing a personalized CAR T cell therapy called CABA-201 in people with inflammatory muscle diseases, including juvenile forms of these diseases. Researchers want to learn if the treatment is safe and well tolerated. The treatment uses a participant's own immune cells, which are modified in a laboratory and then returned to the body to help remove immune cells that may be causing the disease.

Study Details

Full Title
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Subjects with Active Idiopathic Inflammatory Myopathy or Active Juvenile Idiopathic Inflammatory Myopathy
Principal Investigator
Kaveh Ardalan, MD, MS
Pediatric Rheumatologist
Protocol Number
IRB: PRO00116563
NCT: NCT06154252
Phase
Phase I/II
ClinicalTrials.gov
Enrollment Status
Open for Enrollment
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