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Clinical Study on the Safety, Efficacy and Pharmacokinetics of Universal CLL1 Chimeric Antigen Receptor T-Cell in Relapsed/Refractory Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a common type of acute leukemia in adults. Although the treatment of AML has improved in recent decades, the 5-year survival rate remains below 50% due to the chemoresistance or toxicity of these treatments. Most patients eventually die from relapse and/or progressive disease, and these patients urgently need new treatment strategies. Chimeric antigen receptor T-cell (CAR-T cell) therapy is an adoptive immunotherapy that expresses one or more specific chimeric antigen receptors (CARs) on T cells through genetic engineering, enabling them to target tumor cells. CAR-T cell immunotherapy has been a milestone in tumor immunotherapy in recent years and has achieved remarkable efficacy in the treatment of hematological malignancies. Human C-type lectin-like molecule 1 (CLL-1) is specifically expressed on the tumor cells of more than 90% of AML patients. CLL1 is selectively expressed on the surface of leukemia stem cells but not on normal hematopoietic stem cells, making it an ideal target for AML. Autologous CLL1 CAR-T cells have shown strong therapeutic effects in previous studies. However, autologous CAR-T cells have disadvantages such as long preparation time and high cost. Universal CAR-T cells have effectively solved this problem. In this study, universal CAR-T cells targeting the CLL1 target were prepared based on the non-gene editing intracellular membrane protein retention technology, further expanding the application of CAR-T in the treatment of acute myeloid leukemia.
Details
| Lead sponsor | Mingfeng Zhao |
|---|---|
| Phase | Phase 1 |
| Status | NOT_YET_RECRUITING |
| Enrolment | 20 |
| Start date | 2026-02 |
| Completion | 2028-12-12 |
Conditions
- Acute Myeloid Leukemia
- Chimeric Antigen Receptor T-cell
- Universal CLL1 CAR-T
Interventions
- universal CLL1 CAR-T cells
Primary outcomes
- Bone marrow tumor burden — Day14; Day 28；Month 3；Month 6；Month 12；Month 18；Month 24
At key time points following CAR-T cell infusion (e.g., Day 14), bone marrow samples are collected from patients via bone marrow puncture. The proportion of blast cells in the total bone marrow cells is calculated using flow cytometry to assess the therapeutic effect of CAR-T cells. A blast cell percentage of less than 5% is defined as complete remission. - Overall survival time — From the date of CAR-T cell infusion to the date of death from any cause, assessed up to 60 months.
Overall survival time：Evaluate the time from CAR-T cell infusion to death or the end of follow-up for the patients. - Progression-Free-Survival — From date of CAR-T cell infusion until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 60months.
Progression-free survival time: The time interval from receiving CAR-T cell infusion to disease progression or death.