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.
This study adopts the "3+3" design, with a total of three dosage groups (1 × 107/kg, 3 × 107/kg, 6 × 107/kg). The study will start from the low dose group. Only when the present dose group has completed enrollment and no DLT has been observed, the study could escalate to the next dose group based on the evaluation of efficacy and cell kinetic data. If no DLT is observed even in the highest dose group, the escalation will be terminated.Each dose group is expected to enroll 3 to 6 patients, with a total of 9 to 18 patients anticipated in the trial.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
A single infusion of CD19 UCAR-T cells will be administered intravenously after lymphodepletion chemotherapy.
Children's Hospital Zhejiang University School of Medicine, Hangzhou, zhejiang
Hangzhou, Zhejiang, China
RECRUITINGIncidence 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.
Time frame: 28 dyas
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.
Time frame: 6 months
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
Time frame: 6 months
Changes of myositis specific antibody levels and serum muscle enzyme levels after anti-CD19 UCAR-T cells infusion.
Time frame: 6 months
Improvement in muscle inflammation infiltration by MRI after anti-CD19 UCAR-T cells infusion.
Time frame: 6 months
Childhood Myositis Assessment Scale (CMAS) score after anti-CD19 UCAR-T cells
The total score is 52 points, the lower the score, the weaker the muscle strength and the more severe the disease.
Time frame: 6 months
Cmax of anti-CD19 UCAR-T cells [Cell dynamics]
Time frame: 1 month
Tmax of anti-CD19 UCAR-T cells [Cell dynamics]
Time frame: 1 month
AUCs of anti-CD19 UCAR-T cells [Cell dynamics]
Time frame: 1 month
Changes of myth enzyme after anti-CD19 UCAR-T cells
Time frame: 1 year
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