The goal of this prospective, open, single-arm clinical trial was to evaluate the safety and potential efficacy of CAR T cell therapy in children with refractory/recurrent lupus nephritis. The persistence and cell phenotype of CAR-T cells in vivo and CAR-T treatment-related inflammatory factors were evaluated after treatment. To explore new therapeutic methods, in order to reduce the side effects of traditional therapeutic drugs, increase curative effect, and finally make patients obtain long-term survival and improve survival quality.
Age 6-18 years old (including critical value);
Diagnosed with SLE according to the 2019 EULAR/ACR SLE classification criteria;
According to the 2018 ISN/RPS LN standards diagnosed with active Class III or IV LN, with or without a membranous component and the biopsy must be performed within 6 months prior to screening;
SLEDAI-2000 score ≥8 points;
Received kidney transplant previously;
Serious drug allergy history or allergy;
Presence or suspicion of fungal, bacterial, viral or other infections that cannot be controlled or require treatment;
Complicated with severe organ dysfunction of heart, liver, lung or coagulation dysfunction;
Single group
Low-dose CAR-T cells group
Biological/VaccineThis group of patients received low dose novel structure of Chimeric Antigen Receptors T (CAR-T) cells therapy with an infusion dose of approximately 5×100,000 cells/Kg.
High-dose CAR-T cells group
Biological/VaccineThis group of patients received high dose novel structure of Chimeric Antigen Receptors T (CAR-T) cells therapy with an infusion dose of approximately 1×1000,000 cells/Kg.
Number of participants with CAR-T cells treatment-related adverse events(AE)
Incidence and severity of treatment-related AE, including adverse event of special interest (AESI), as assessed by CTCAE v5.0.
Overall renal response (ORR) rete
ORR rate: Number of participants meet the complete renal response (CRR) or partial renal response (PRR) as a percentage of total participants. Follow-up for 2 years after CAR-T cells reinfusion, monitored and recorded ORR at study set time points (Month 3, Month 6, Month 12, Month 24, or participant withdrawal from the study).
Number of participants with SRI-4 response
Number of participants with SLE response Index 4(SRI-4) response: including Systemic Lupus Erythematosus Disease Activity Index(SLEDAI) 2000 scores decreased by ≥4 points from baseline, PGA with no worsening (VAS increased by \<0.30 points from baseline), British Isles Lupus Assessment Group(BILAG) 2004 with no new A domain score and no more than 1 new B domain scores. Follow-up for 2 years after CAR-T cells reinfusion, monitored and recorded SRI-4 response at study set time points (Month 3, Month 6, Month 12, Month 24, or participant withdrawal from the study).
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