A Phase 1 clinical trial to evaluate the safety and efficacy of PiggyBac transposon-mediated Chimeric Antigen Receptor(CAR) T-cells targeting CD19 in refractory Systemic Lupus Erythematosus (SLE) patients who have not responded to standard immunosuppressive treatments.
This is a single-institution phase I study in adults with refractory SLE. Autologous Peripheral Blood Mononuclear Cells will be transduced with a chimeric antigen receptor targeting the B-cell surface antigen CD19 using the PiggyBac Transposon system. Subjects will receive a conditioning lymphodepletion chemotherapy regimen of fludarabine and cyclophosphamide, followed by the infusion of 1x10\^6 cells/kg CD-19 CAR T-cells. Subjects will be evaluated post-treatment for toxicity, SLE disease activity, and the persistence of CAR-expressing T cells in vivo.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
PiggyBac Transposon-Mediated CD19 CAR-T Therapy (1-2 x 10\^6 cells/kg)
King Chulalongkorn Memorial Hospital
Bangkok, Please Select, Thailand
Safety of PiggyBac transposon-mediated CAR T-cell infusion targeting CD19 in adult patients with refractory SLE.
The incidence of adverse events assessed by the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0
Time frame: Up to 28 days after CD-19 CAR-T cell infusion
Disease activity of SLE
The Safety of Estrogens in Lupus Erythematosus National Assessment (SELENA) - Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)
Time frame: 3, 6, and 12 months after CD-19 CAR-T cell infusion
Complete response rate of lupus nephritis
Complete response defined as normal or ≤ 25% decline of estimated glomerular filtration rate (eGFR) Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) from baseline and urinary protein to creatinine index (UPCI) or 24-hour urinary protein ≤ 0.5 g/g or g/day
Time frame: 3, 6, and 12 months after CD-19 CAR-T cell infusion
Partial response rate of lupus nephritis
Partial response defined as normal or ≤25% decline of eGFR CKD-EPI from baseline and at least 50% reduction of proteinuria, with a UPCI or 24-hour urinary protein between 0.5 to 3 g/g or g/day
Time frame: 3, 6, and 12 months after CD-19 CAR-T cell infusion
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