CD19 CAR-T Therapy for Refractory Childhood-Onset Systemic Lupus Erythematosus

A Clinical Study of CD19 Chimeric Antigen Receptor T-Cell Therapy for Patients With Refractory Childhood-Onset Systemic Lupus Erythematosus

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.

Study Overview

• Status: Recruiting
• Conditions: Childhood-Onset Systemic Lupus Erythematosus; Refractory SLE; CD19 CAR-T Cell Therapy
• Intervention / Treatment: Biological: SNUH-CD19-CAR-T Cells; Drug: Lymphodepleting Chemotherapy

• Study Type: Interventional
• Enrollment (Estimated): 8
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Hye Ji, CRC; Phone Number: 82-10-2957-8895; Email: hyeji91@snu.ac.kr

Study Locations

• South Korea
  • Seoul, South Korea
    • Recruiting
    • Seoul National University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 25 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Participants must be 25 years of age or younger at the time of SNUH-CD19-CAR-T infusion.

2. 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.

3. 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.

4. Participants must have involvement of at least one of the following major organ systems:

   • Kidney: class II-V lupus nephritis confirmed by biopsy or proteinuria greater than 0.5 g/day
   • Heart: pericarditis, myocarditis, conduction abnormality, or valvular disease
   • Lung: pleuritis, interstitial lung disease, or pulmonary arterial hypertension
   • Hematologic system: platelet count below 50,000/μL or hemolytic anemia

5. Participants must have uncontrolled active disease, defined as a Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score of 10 or higher.

Exclusion Criteria:

1. Severe active central nervous system lupus requiring current treatment, including uncontrolled seizures, psychotic symptoms, or cognitive impairment.
2. Inability to obtain an adequate leukapheresis product suitable for the manufacture of SNUH-CD19-CAR-T cells.
3. Known human immunodeficiency virus infection.
4. 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.
5. Pregnancy or breastfeeding.
6. Any medical or other condition that, in the investigator's clinical judgment, makes participation in the study inappropriate.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: SNUH-CD19-CAR-T Treatment; Participants will receive protocol-defined lymphodepleting chemotherapy followed by a single intravenous infusion of autologous SNUH-CD19-CAR-T cells at a dose of 0.2 to 1.0 × 10^6 CAR-positive viable T cells/kg.

Biological: SNUH-CD19-CAR-T Cells; Autologous 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.; Drug: Lymphodepleting Chemotherapy; Participants 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Proportion of Participants Achieving Lupus Low Disease Activity State	1 year after SNUH-CD19-CAR-T infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	1 year after SNUH-CD19-CAR-T infusion

Collaborators and Investigators

• Sponsor: Seoul National University Hospital

Publications and helpful links

General Publications

• Muller F, Taubmann J, Bucci L, Wilhelm A, Bergmann C, Volkl S, Aigner M, Rothe T, Minopoulou I, Tur C, Knitza J, Kharboutli S, Kretschmann S, Vasova I, Spoerl S, Reimann H, Munoz L, Gerlach RG, Schafer S, Grieshaber-Bouyer R, Korganow AS, Farge-Bancel D, Mougiakakos D, Bozec A, Winkler T, Kronke G, Mackensen A, Schett G. CD19 CAR T-Cell Therapy in Autoimmune Disease - A Case Series with Follow-up. N Engl J Med. 2024 Feb 22;390(8):687-700. doi: 10.1056/NEJMoa2308917.
• Mackensen A, Muller F, Mougiakakos D, Boltz S, Wilhelm A, Aigner M, Volkl S, Simon D, Kleyer A, Munoz L, Kretschmann S, Kharboutli S, Gary R, Reimann H, Rosler W, Uderhardt S, Bang H, Herrmann M, Ekici AB, Buettner C, Habenicht KM, Winkler TH, Kronke G, Schett G. Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus. Nat Med. 2022 Oct;28(10):2124-2132. doi: 10.1038/s41591-022-02017-5. Epub 2022 Sep 15.

Study record dates

Study Major Dates

• Study Start (Actual): September 14, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: September 27, 2026
• First Submitted That Met QC Criteria: September 27, 2026
• First Posted (Actual): October 2, 2026

Study Record Updates

• Last Update Posted (Actual): October 2, 2026
• Last Update Submitted That Met QC Criteria: September 27, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin and Connective Tissue Diseases; Lupus Erythematosus, Systemic
• Other Study ID Numbers: SNUH-CD19-CART-SLE-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No