This study is an open-label, single-arm, dose-escalation trial primarily designed to evaluate the safety and efficacy of a universal CAR-T cell therapy targeting CD19 in the treatment of patients with RJDM. Additionally, the study aims to characterize its pharmacokinetic and pharmacodynamic properties, explore its role in immune system reconstitution, and assess long-term survival benefits.
(1) Age ≥ 5 years old;
(2) Diagnosis of juvenile dermatomyositis (JDM) according to Bohan and Peter criteria;
Intolerance or inadequate response to glucocorticoids (prednisone 1-2mg/kg/d or equivalent dose of other hormones) and at least two immunosuppressants, with hormone therapy lasting for at least 6 months;
Rapid progression of the disease and/or involvement of organs such as lungs, heart, and gastrointestinal tract;
(1) Previously received CAR-T cell therapy (except for those whose safety risks have been judged as eliminated by the investigator);
(2) Accompanied by primary immunodeficiency disease or severe secondary immunodeficiency disease that has not been corrected;
(3) Accompanied by severe, active, or uncontrolled infectious diseases, including but not limited to active tuberculosis, latent tuberculous infection, active viral hepatitis, etc.;
(4) Known to have active malignant diseases or confirmed malignancies before screening (including hematological malignancies and solid tumors, except for resected and cured cutaneous basal cell carcinoma);
Single group
Anti-CD19 UCAR-T cells
DrugA single infusion of CD19 UCAR-T cells will be administered intravenously after lymphodepletion chemotherapy.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
The incidence of adverse events after CAR-T cell infusion was assessed by CTCAE, version 5.0.
Total improvement score (TIS) after anti-CD19 UCAR-T cells infusion.
The total improvement score between 0-100 also corresponds to the degree of improvement, with higher improvement scores indicating greater improvement.
Disease Activity Score (DAS) after anti-CD19 UCAR-T cells infusion.
Including musculoskeletal system score and skin mucosal score, with a total score of 20 points. The higher the score, the higher the disease activity
Changes of myositis specific antibody levels and serum muscle enzyme levels after anti-CD19 UCAR-T cells infusion.
Cmax of anti-CD19 UCAR-T cells [Cell dynamics]
Tmax of anti-CD19 UCAR-T cells [Cell dynamics]
AUCs of anti-CD19 UCAR-T cells [Cell dynamics]
Changes of myth enzyme after anti-CD19 UCAR-T cells
Duration of disease remission (DOR) after anti-CD19 UCAR-T cells infusion [Long-term Efficacy]
Time to B cell recovery after anti-CD19 UCAR-T cells infusion.
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