BCOR and ZC3H12 Genes Knock-out CD19-targeting CAR-T Cell Therapy in r/r B-ALL
Summary
In this single-center, single-arm, prospective, Phase 1/2 study, the safety and efficacy of autologous BCOR and ZC3H12 genes knock-out CD19-targeting chimeric antigen receptor (CAR) T-cell therapy will be evaluated in patients with refractory/relapsed (r/r) B-cell acute lymphoblastic leukaemia (B-ALL). In phase 1, 3 eligible patients will be enrolled and receive BCOR and ZC3H12 genes knock-out CD19 CAR T cell therapy at a initial dose of 5×10\^5 cells/kg. Based on the results, . Subsequently an additional 3-15 patients will be enrolled in a "3+3" dose-escalation/decline design to adjust the dose of BCOR and ZC3H12 genes knock-out CD19 CAR T cells to achieve optimal safety and efficacy. The recommended Phase 2 dose (RP2D) will then be established. 10 to 12 subjects will be enrolled and receive BCOR and ZC3H12 genes knock-out CD19 CAR T cell infusion at dose of RP2D.
Timeline
- Start
- 2025-06-20
- Primary completion
- 2026-05-31
- Completion
- 2027-05-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CAR19TIF | Cell therapy | 50000 cells/kg | — |
| Subject | CAR19TIF | Cell therapy | 100000 cells/kg | — |
| Subject | CAR19TIF | Cell therapy | 500000 cells/kg | — |
| Subject | CAR19TIF | Cell therapy | 2e+06 cells/kg | — |
| Subject | CAR19TIF | Cell therapy | 6e+06 cells/kg | — |
| Background | Cyclophosphamide | Other / unclassified | 250 mg/m2 | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | 500 mg/m2 | Intravenous |
| Background | Fludarabine | Small molecule | 25 mg/m2 | Intravenous |
| Background | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |