Open-Label Study Evaluating CABA-201 Cell Therapy for Active Idiopathic Inflammatory Myopathies and Juvenile Idiopathic Inflammatory Myopathy

What this Trial Is About

Researchers are evaluating the safety and effectiveness of an investigational cell therapy called CABA-201 in people with active idiopathic inflammatory myopathy IIM or juvenile idiopathic inflammatory myopathy JIIM. These are rare autoimmune diseases that cause muscle inflammation and weakness. The study focuses on specific subtypes including dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, and juvenile idiopathic inflammatory myopathy, which may involve B cells attacking the bodys tissues. Participants receive a single intravenous infusion of CABA-201 after preconditioning treatment with the drugs fludarabine and cyclophosphamide. The study includes different cohorts based on the specific subtype of myopathy. The treatment is given once, and the study evaluates the effects in this open-label, non-randomized setting. During the study, participants are closely monitored for adverse events up to 28 days after infusion, and the improvement in disease symptoms is assessed over weeks to months depending on the subtype. Researchers also track laboratory results, biomarkers related to muscle inflammation and autoantibodies, and pharmacokinetics and pharmacodynamics of the therapy. Follow-up assessments extend up to 156 weeks for some measures, allowing long-term evaluation of safety and response. The study involves participants aged 6 to 75 years with active disease despite prior or current standard treatments.

RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy