Autoimmune diseases occur when the immune system mistakenly attacks the body's own tissues, leading to chronic inflammation and damage to organs such as the kidneys, lungs, muscles, nerves, or blood cells. Although many treatments are available, some patients do not respond adequately or experience repeated disease flares despite long-term therapy. New treatment approaches are therefore needed for patients with relapsed or refractory autoimmune diseases. This study is an exploratory clinical trial designed to evaluate the safety and potential benefits of a novel cell-based therapy called autologous CD19-BCMA dual-target CAR T-cell therapy. This treatment uses a patient's own immune cells, which are collected from the blood, modified in the laboratory to recognize specific immune cells involved in autoimmune disease, and then infused back into the patient. The study includes adult patients with certain relapsed or refractory autoimmune diseases, such as systemic lupus erythematosus, systemic sclerosis, inflammatory muscle diseases, Sjögren's syndrome, autoimmune hemolytic anemia, and multiple sclerosis. After cell collection and preparative treatment, participants will receive a single infusion of the investigational CAR T-cell therapy and will be closely monitored for safety. The main purpose of this study is to better understand the safety of this treatment, including possible side effects. The study will also explore how the disease responds to treatment over time. Participants will be followed for up to two years after treatment to assess safety and clinical outcomes. The results of this study may help researchers better understand whether this type of cell therapy could be a feasible treatment option for patients with difficult-to-treat autoimmune diseases in the future.
Age 18 to 70 years (inclusive), of any sex.
Patients with failure of prior single-target CD19 or BCMA therapy, with a washout period of at least 6 months since the last treatment.
Diagnosis of SLE according to the 2019 EULAR/ACR Classification Criteria for Systemic Lupus Erythematosus.
At screening, positive antinuclear antibody (ANA) (titer ≥1:80), and/or positive anti-dsDNA antibody, and/or positive anti-Sm antibody.
Use of B-cell-depleting therapy within 1 month prior to screening and assessed by the investigator as not having failed therapy, including agents targeting CD19, CD20, CD22, CD52, CD38, or BCMA (monoclonal antibodies or bispecific antibodies).
High-dose intravenous human immunoglobulin (IVIG) within 1 month prior to screening.
Therapeutic-dose systemic corticosteroids within 24 hours prior to lymphodepleting conditioning (prednisone >20 mg/day or equivalent).
Corticosteroid pulse therapy within 2 weeks (defined as prednisone ≥500 mg/day or equivalent).
Single group
Autologous CD19-BCMA Dual-Target CAR T-Cell Therapy
DrugAutologous CD19-BCMA dual-target CAR T-cell therapy is a personalized cell-based immunotherapy manufactured from each participant's own peripheral blood T lymphocytes. Following leukapheresis, autologous T cells are genetically modified ex vivo to express a chimeric antigen receptor targeting both CD19 and B-cell maturation antigen (BCMA), enabling recognition and elimination of B-lineage cells and antibody-producing plasma cells implicated in autoimmune disease pathogenesis. The modified T cells are expanded under controlled conditions and administered as a single intravenous infusion after lymphodepleting conditioning. This dual-target CAR-T approach is intended to provide broad and sustained depletion of pathogenic B-cell populations and to promote immune system rebalancing in patients with relapsed or refractory autoimmune diseases.
Number and percentage of participants with adverse events (AEs), serious adverse events (SAEs), laboratory abnormalities, and adverse events of special interest (AESIs: CRS and ICANS), graded per CTCAE v5.0 and ASTCT 2019 criteria
AEs will be collected from the time of signing the informed consent form through 2 years after CAR-T infusion or the exit visit, whichever occurs first; if disease relapse occurs within 6 months after infusion, AEs will be collected as much as possible through 6 months post-infusion with participant cooperation. All AEs will be coded using MedDRA and summarized by System Organ Class (SOC) and Preferred Term (PT) as the number and percentage of participants with events. AE severity will be summarized using CTCAE v5.0. CRS and ICANS will be summarized using ASTCT 2019 grading criteria. AESIs (including CRS and ICANS) and SAEs (including death) will be listed separately.
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