This is a single-center, single-arm, open-label phase 1/2 study of CART19 in children and young adults with refractory Systemic lupus erythematosus (SLE), including both patients diagnosed with lupus nephritis (LN) and patients with non-renal Systemic lupus erythematosus (SLE).
Phase 1 will evaluate the safety of CART19 in 6-12 patients with Systemic lupus erythematosus (SLE). There is no planned dose escalation, but a dose de-escalation will be made based on the incidence of Dose Limiting Toxicities. Phase 2 will evaluate the efficacy and further evaluate the safety of CART19 in this population.
Signed informed consent form must be obtained prior to any study procedure. Labs or other procedures obtained during routine clinical care may be used for eligibility if obtained within the protocol required window.
Patient age must be 10-29 years, inclusive, at time of enrollment.
Meeting ACR/EULAR Classification Criteria for SLE
ANA positive > 1:80 and/or double-stranded DNA (dsDNA) positive
Active, untreated infections
HIV infection
Active Hepatitis B
Active Hepatitis C
Single group
CART19
Biological/VaccineCART19 cells transduced with a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ, administered by IV injection.
Frequency of the dose limiting toxicities of CART19
Frequency of the dose limiting toxicities of CART19
Rate of childhood SLE Clinical Remission off steroids (cCR-0) within 12 months
2-year overall survival rate
2-year flare free survival rate
Feasibility of manufacturing CART19 for participants with SLE
Feasibility of manufacturing CART19 measured by the percentage of manufactured products that do not meet release criteria for vector transduction efficiency, T cell product purity, viability, or sterility
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