CD19 CAR-T Therapy for Refractory Childhood-Onset Systemic Lupus Erythematosus
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
AgeUp to 25
SponsorSeoul National University Hospital
About this trial
This study is evaluating the safety and potential benefits of CD19 chimeric antigen receptor T-cell (CAR-T) therapy in children and young adults with severe systemic lupus erythematosus (SLE) that began during childhood. The study will include up to 8 participants aged 25 years or younger whose disease remains active despite treatment with multiple immune-suppressing medicines and has affected at least one major organ.
CAR-T cells will be made from each participant's own T cells and modified to recognize CD19, a protein found on B cells that contribute to SLE. Participants will receive chemotherapy to reduce existing immune cells, followed by a single intravenous infusion of SNUH-CD19-CAR-T cells. They will be monitored for side effects and changes in disease activity, organ involvement, laboratory findings, and the need for steroids or other immune-suppressing medicines. The main assessment will be performed 1 year after the CAR-T cell infusion.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participants must be 25 years of age or younger at the time of SNUH-CD19-CAR-T infusion.
Participants must have a diagnosis of systemic lupus erythematosus and meet the 2019 European Alliance of Associations for Rheumatology/American College of Rheumatology classification criteria for systemic lupus erythematosus.
Participants must be currently receiving or have previously received at least three of the following therapies, including corticosteroids: hydroxychloroquine, methotrexate, cyclophosphamide, mycophenolate mofetil, cyclosporine, tacrolimus, rituximab, or belimumab.
Kidney: class II-V lupus nephritis confirmed by biopsy or proteinuria greater than 0.5 g/day
Disqualifiers
Severe active central nervous system lupus requiring current treatment, including uncontrolled seizures, psychotic symptoms, or cognitive impairment.
Inability to obtain an adequate leukapheresis product suitable for the manufacture of SNUH-CD19-CAR-T cells.
Known human immunodeficiency virus infection.
Uncontrolled active infection, as determined by the investigator. An infection will be considered controlled if appropriate treatment has been administered and there is no evidence of progression at enrollment. Persistent fever without other signs or symptoms of infection will not, by itself, be considered evidence of progressive infection.
Trial design
Design model
Single group
Treatments tested in this trial
SNUH-CD19-CAR-T Cells
Biological/VaccineAutologous T cells genetically modified to express a CD19-targeted chimeric antigen receptor. Participants will receive a single intravenous infusion at a dose of 0.2 to 1.0 × 10\^6 CAR-positive viable T cells/kg.
Lymphodepleting Chemotherapy
DrugParticipants will receive protocol-defined lymphodepleting chemotherapy before CAR-T cell infusion. The preferred regimen consists of intravenous fludarabine 30 mg/m²/day for 4 days and cyclophosphamide 500 mg/m²/day for 2 days. Alternative regimens, dose reduction, partial administration, or omission may be used according to the protocol and the investigator's clinical judgment
Treatment groups
8 Participants
are divided into 1 treatment groupGroup A: SNUH-CD19-CAR-T TreatmentExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
Proportion of Participants Achieving Lupus Low Disease Activity State
Time frame
1 year after SNUH-CD19-CAR-T infusionSecondary outcomes
1
Proportion of Participants With Lupus Nephritis Achieving a Renal Response
Among participants with lupus nephritis at baseline, renal response is defined as achievement of either a complete or partial renal response according to protocol-specified criteria. Complete renal response requires eGFR ≥90 mL/min/1.73 m² or ≥90% of baseline, urine protein-to-creatinine ratio \<0.5 g/g, normal or inactive urinary sediment, and normal serum creatinine or a value within 15% of baseline. Partial renal response requires eGFR ≥80% of baseline or an improvement of ≥25%, a ≥50% reduction in urine protein-to-creatinine ratio to \<3.0 g/g, and serum creatinine ≤125% of baseline.
Time frame
1 year after SNUH-CD19-CAR-T infusionSponsors and contacts
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Source ClinicalTrials.gov