Study of CD123-Directed T-Cell Therapy for Patients 21 Years and Younger with Relapsed or Refractory AML, MDS, ALL, or BPDCN

What this Trial Is About

Researchers are studying CD123-CAR T-cell therapy as a new treatment for patients 21 years old or younger with certain blood cancers, including acute myelogenous leukemia AML, myelodysplastic syndrome MDS, T- or B-cell acute lymphoblastic leukemia ALL, and blastic plasmacytoid dendritic cell neoplasia BPDCN. The main goal is to find the highest safe dose of these modified immune cells after chemotherapy, while monitoring side effects and overall patient survival. This is a phase 1 trial sponsored by St. Jude Childrens Research Hospital. The study has two main parts first, blood cells are collected from patients and modified in the lab to better target cancer cells. Then, patients receive chemotherapy called lymphodepleting chemotherapy for several days to prepare their body for the treatment. This includes fludarabine given on days -4, -3, and -2 and cyclophosphamide on days -3 and -2. After a rest day on day -1, patients receive one infusion of the CD123-CAR T cells on day 0 or day 1. The trial tests up to four dose levels of the CAR T cells, with special groups for patients with or without a transplant donor. Participants will have blood drawn for cell collection and then receive chemotherapy and the CAR T-cell infusion under close medical supervision. They will be monitored for side effects, and their disease status will be regularly evaluated. Safety and maximum tolerated dose will be assessed four weeks after infusion. Other tests include monitoring immune cell profiles, tumor changes, and comparing donor-derived to autologous CAR T cells. The total involvement lasts through treatment and follow-up to measure safety and disease response.

CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)