The objective of this study is to evaluate the efficacy and safety of BCMA/CD19 chimeric antigen receptor (CAR)-modified T cells in the treatment of autoimmune diseases.
Age, 18-65 years old (inclusive), weight >=45kg, male and female;
Additional enrollment criteria for systemic lupus erythematosus A. The SLEDAI score of the patient before enrollment >= 7 points B. Failure to receive the following treatments: oral prednisone >=20 mg/d; Cyclophosphamide 0.4 to 0.6 g/m2 once every two weeks for 6 months, or other immunosuppressants such as mycophenolate mofetil 2 g/day for 3 months without remission.
Additional enrollment criteria for antiphospholipid syndrome A. Cardiolipin antibody, lupus anticoagulant factor, and anti-β2-glycoprotein 1 antibody were all positive before enrollment.
Sjögren's disease additional enrollment criteria A. Positive anti-Ro/SSA antibody screen. B. ESSDAI>= 6 POINTS
Use of rituximab or other monoclonal antibodies within 1.6 months.
Received high-dose glucocorticoids (>1 mg/kg/d) within 1 month.
Serious complications: including heart failure (>= NYHA Class III), renal insufficiency (creatinine clearance <=30 ml/min), hepatic insufficiency (serum ALT or AST greater than three times the upper limit of normal, or total bilirubin greater than the upper limit of normal)
Other severe, progressive, or uncontrollable hematologic, gastrointestinal, endocrine, pulmonary, cardiac, neurological, or cerebral diseases (including demyelinating diseases such as multiple sclerosis).
Single group
BCMA-CD19 CAR-T therapy
DrugPatients received the BCMA-CD19-targeted CAR-T cells with a dosage of 3×10\^6/kg CAR-T cells per kilogram.
Systemic Lupus Erythematosus primary outcome
Percentage of SLE participants who had a response on the SLE Responder Index (SRI-4) at 24 weeks. The Systemic Lupus Erythematosus Responder Index-4 (SRI-4) was used to assess the treatment efficacy in systemic lupus erythematosus (SLE).The scale ranges from 0 to 105, with higher scores indicating worse disease activity. A response is defined as a ≥4-point improvement from baseline at 24 weeks
Sjogren syndrome primary outcome
Proportion of participants with a Sjögren's Tool for Assessing Response (STAR) score of greater than or equal to five at 24 weeks. The STAR is a composite score used to evaluate the clinical response in patients with Sjögren's syndrome. The STAR score ranges from 0 to 9, with higher scores indicating better clinical response. A score of ≥5 at 24 weeks indicates a positive response to treatment.
Inflammatory Myopathy primary outcome
TIS ranges from 0 to 100, with a higher score denoting greater improvement. In adults, TIS can be stratified into the following improvement categories for each patient at each timepoint (minimal improvement \[TIS ≥20\]; moderate improvement \[TIS ≥40\]; major improvement \[TIS ≥60\]). Thus, in the above example the patient would be classified as having moderate improvement \[TIS ≥40\].
Systemic Sclerosis primary outcome
Change in the Modified Rodnan Skin Score (mRSS) at 24 weeks compared to baseline. The mRSS is a clinical tool used to assess the severity of skin thickening in patients with systemic sclerosis (scleroderma). The mRSS ranges from 0 to 51, with higher scores indicating more severe skin thickening. A decrease in the mRSS score indicates an improvement in skin involvement
Systemic Lupus Erythematosus primary secondary Outcome
Change in SLEDAI-2K at 24 weeks compared to baseline. The SLEDAI-2K is a composite index used to assess disease activity in systemic lupus erythematosus. The score ranges from 0 to 105, with higher scores indicating more severe disease activity. An improvement of ≥4 points in the SLEDAI-2K score at 24 weeks compared to baseline is considered a clinically meaningful response, reflecting a reduction in disease activity
Sjogren syndrome secondary outcome
Change in the EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) score at 24 weeks compared to baseline. The ESSDAI is a composite score used to assess disease activity in patients with Sjögren's syndrome. The score ranges from 0 to 123, with higher scores indicating more severe disease activity. A decrease in the ESSDAI score at 24 weeks compared to baseline indicates an improvement in disease activity.
Inflammatory Myopathy secondary outcome
Change in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) score at 24 weeks compared to baseline. The CDASI is a composite score used to assess the severity and extent of skin involvement in patients with cutaneous dermatomyositis. The score ranges from 0 to 132, with higher scores indicating more severe disease activity. A decrease in the CDASI score at 24 weeks compared to baseline indicates an improvement in skin involvement and disease severity.
Systemic Sclerosis secondary outcome
Change in the Health Assessment Questionnaire Disability Index (HAQDI) score at 24 weeks compared to baseline. The HAQDI is a widely used measure of disability in patients with chronic diseases, assessing the impact of disease on daily functioning. The score ranges from 0 to 3, with higher scores indicating greater disability and more severe functional impairment. A decrease in the HAQDI score at 24 weeks compared to baseline indicates an improvement in the patient's functional status and a reduction in disability.
Click on the lead sponsor to view all of their trials.
