Study of BCOR and ZC3H12 Gene Knock-out CD19-targeting CAR-T Cell Therapy in Adults with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

What this Trial Is About

Researchers are evaluating a new CAR T-cell therapy for adults with refractory or relapsed B-cell acute lymphoblastic leukemia rr B-ALL. This Phase 12 single-center, single-arm study focuses on the safety and effectiveness of CAR T cells with BCOR and ZC3H12 genes knocked out, called CAR19TIF cells, to improve treatment persistence and long-term response in patients who have limited options after prior therapies. The study aims to establish the best dose and assess treatment outcomes in this patient group. The study includes two phases. Phase 1 involves dose escalation or decline with 6 to 18 patients receiving CAR19TIF cells at varying doses starting from 5105 cellskg, using a staggered 28-day administration schedule to evaluate safety and efficacy. Phase 2 will enroll 10 to 12 patients treated at the recommended Phase 2 dose determined by Phase 1 results. Before CAR19TIF infusion, patients receive conditioning chemotherapy with fludarabine and cyclophosphamide. The study monitors dose-limiting toxicities and adjusts dosing to find the optimal balance of safety and effectiveness. Participants will undergo regular assessments including blood tests to measure CAR-positive T cells and CD19-positive cells over 12 to 24 months. The study tracks adverse events, response rates, survival, and progression-free survival up to two years after treatment. Patients are monitored closely for safety and treatment effects, with follow-up visits and evaluations scheduled throughout the study. The full duration of participation varies by individual treatment phase and follow-up timelines.

BCOR and ZC3H12 Genes Knock-out CD19-targeting CAR-T Cell Therapy in r/r B-ALL