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

NCT07855302 | Recruiting Phase 1

• 1 other identifier: SNUH-CD19-CART-SLE-01
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Childhood-Onset Systemic Lupus Erythematosus; Refractory SLE; CD19 CAR-T Cell Therapy

Outcome Measures

Primary Outcomes (1)

• Proportion of Participants Achieving Lupus Low Disease Activity State

  1 year after SNUH-CD19-CAR-T infusion

Secondary Outcomes (1)

• Proportion of Participants With Lupus Nephritis Achieving a Renal Response

  1 year after SNUH-CD19-CAR-T infusion

Study Arms (1)

SNUH-CD19-CAR-T Treatment

EXPERIMENTAL

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; Drug: Lymphodepleting Chemotherapy

Interventions

SNUH-CD19-CAR-T Cells BIOLOGICAL

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.

SNUH-CD19-CAR-T Treatment

Lymphodepleting Chemotherapy DRUG

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

SNUH-CD19-CAR-T Treatment

Eligibility Criteria

• Age: Up to 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.
• Pregnancy or breastfeeding.
• Any medical or other condition that, in the investigator's clinical judgment, makes participation in the study inappropriate.

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Hospital

Seoul, South Korea

RECRUITING

Related Publications (2)

• 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.

  PMID: 38381673 BACKGROUND

• 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.

  PMID: 36109639 BACKGROUND

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Hye Ji, CRC

CONTACT

82-10-2957-8895; hyeji91@snu.ac.kr

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2026
• First Posted: October 2, 2026
• Study Start: September 14, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: October 2, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)