Study of BCMA-CD19 CAR-T Cell Therapy for Adults with Difficult-to-Treat Autoimmune Diseases

What this Trial Is About

Researchers are evaluating the safety and effectiveness of BCMA-CD19 chimeric antigen receptor CAR-modified T cells in adults with multiple refractory autoimmune diseases that have not responded to other treatments. This open-label, single-center, single-arm Phase 2 study focuses on various autoimmune conditions including systemic lupus erythematosus, Sjogren syndrome, inflammatory myopathy, systemic sclerosis, Behcets disease, ANCA-associated vasculitis, antiphospholipid syndrome, acquired thrombotic thrombocytopenic purpura, and IgG4-related disease. The study aims to assess the complete remission rate and laboratory markers as primary outcomes, along with pharmacokinetics, long-term effectiveness, and safety as secondary outcomes. Participants receive a single intravenous dose of BCMA-CD19 targeted CAR-T cells at 3106 cells per kilogram of body weight, administered 1 to 2 days after preconditioning. The total volume infused ranges from approximately 10 to 70 mL. This single-dose treatment is followed by monitoring for clinical and laboratory improvements to evaluate treatment effects. The study spans up to 52 weeks depending on the specific autoimmune condition being treated. Throughout the study, participants undergo regular evaluations of clinical signs, laboratory tests, and pharmacokinetic assessments to track safety and efficacy. Outcome measures are collected from enrollment through either 24 or 52 weeks depending on the disease. Participants must adhere to stable doses of glucocorticoids and disease-modifying drugs during the trial. The study includes long-term safety monitoring and requires informed consent and contraception use for participants who may have children. Total participation lasts up to about one year from enrollment.

BCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases