A Phase 1 interventional study of BCMA-CD19 cCAR T cells in Refractory Immune Cytopenia, sponsored by iCell Gene Therapeutics. Recruiting at 1 site in China. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2025-01-22.
Sponsored by iCell Gene Therapeutics · Phase 1, Interventional, and Treatment
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.
697 studies on the registry are indexed under Thrombocytopenia; 153 are open to participants now.
This study's planned enrollment of 20 is below the median of 55 across 472 interventional studies indexed under Thrombocytopenia.
Browse Thrombocytopenia studies →iCell Gene Therapeutics is the lead sponsor of 16 studies on the registry; 7 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Dose escalation phase: patient's T cells will be transduced with a retroviral vector to express a BCMA-CD19 cCAR. with an escalation approach.
Biological: BCMA-CD19 cCAR T cells
• 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.
The 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
Plan to share: No
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iCell Gene Therapeutics