Phase 1 Study of Fresh Allogeneic Second-generation CD19-CAR T Cells to Treat Children and Young Adults With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

What this Trial Is About

Researchers are evaluating fresh, donor-derived CD19-directed second-generation Chimeric Antigen Receptor T CAR T cells in pediatric and young adult patients with relapsed or refractory B-cell precursor Acute Lymphoblastic Leukemia BCP-ALL. This phase 1, open-label, single-center study aims to assess the safety, determine the recommended dose, and gather preliminary evidence of efficacy in patients who have relapsed after or before allogeneic hematopoietic stem cell transplantation alloHSCT with a fully matched donor available. Participants receive a single intravenous infusion of CD19-CARLentiALLO cells on Day 0 following a lymphodepletion regimen with fludarabine and cyclophosphamide given on days -5, -4, and -3. Patients are assigned to two cohorts based on donor HLA matching cohort A with fully matched family or unrelated donors and cohort B with haploidentical donors. Dose levels vary by cohort, and an additional lower dose level may be explored if dose-limiting toxicities occur. During the study, participants will be closely monitored for safety, including dose-limiting toxicities over 28 days, and for graft-versus-host disease occurrence and severity over one year. Researchers will also evaluate treatment responses such as complete response and minimal residual disease negativity. Follow-up includes survival assessment over one year. The study involves detailed clinical evaluations and laboratory tests to ensure thorough monitoring of each participant.

Allogeneic Second-generation CD19-CAR T Cells for Pediatric Relapsed/Refractory B-ALL