This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of BCMA-CD19 cCAR T cells in patients with refractory ITP associated with autoimmune disease.
Immune thrombocytopenia (ITP) Can be associated with various autoimmune diseases, including SLE, and SS. Patients with refractory thrombocytopenia often have long hospital stays, high medical costs, high demand for blood products, and are prone to complications of other systemic injuries. Such patients require active treatment to reduce the risk of life-threatening bleeding, delay the progression of the disease prognosis Glucocorticoids combined with immunosuppressive agents are still the main treatment strategies. Recently, biological agents targeting abnormal immune cells, such as rituximab and belimumab, which deplete B cells have also achieved some success in the treatment of ITP. However, these agents cannot permanently reverse the production of abnormal antibodies as they are unable to eliminate pathogenic long-lived plasma cells because these agents cannot penetrate lymph nodes and soft tissue. The BCMA-CD19 cCAR T-cells are designed to deplete antibody-producing 'root", B cells and plasma cells.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
• BCMA-CD19 cCAR T cells are used to treat patients. Patient will be administered either fresh or thawed CAR T cells by IV injection after receiving lymphodepleting chemotherapy.
West China Hospital
Chengdu, Sichuan, China
RECRUITINGThe number and incidence of adverse events after BCMA-CD19 cCAR T cell infusion
Evaluation all possible adverse reactions, including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity within 3 months after BCMA-CD19 cCAR infusion.
Time frame: 24 months
Overall remission rate 12 weeks after BCMA-CD19 cCAR
1. Complete response (CR): Platelet count ≥ 100 × 109/L and no bleeding; 2. Partial response (PR): platelet count\<100 × 109/L, but increased by at least 2 times compared to baseline platelet count, with no bleeding symptoms 3. Disease control: Disease control monitored up to 2 years after BCMA-CD19 cCAR T cells infusion)
Time frame: 24 months
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