Gene-Edited Stem Cell Transplant and CAR T-Cell Therapy Targeting CD33 for Adults with Difficult or Relapsed Acute Myeloid Leukemia

What this Trial Is About

Researchers are studying a new treatment for adults with hard-to-treat or relapsed acute myeloid leukemia AML. This approach combines a modified stem cell transplant with specially engineered immune cells called CAR T-cells that target leukemia cells. The stem cell transplant is altered so that healthy bone marrow cells are not attacked by the CAR T-cells, aiming to make the therapy safer and more effective. Participants receive a gene-edited stem cell transplant from a healthy donor, followed by one to three infusions of CAR T-cells from the same donor. The gene editing removes a protein called CD33 from the transplanted stem cells, preventing them from being targeted by the CAR T-cells. The CAR T-cells are designed to recognize and attack leukemia cells that express CD33. This phase 1 study evaluates the feasibility and safety of this combined approach. During the trial, participants will be closely monitored with tests to assess manufacturing success, side effects related to both the stem cell transplant and CAR T-cell treatments, and the effectiveness of these therapies. Researchers will track survival, disease progression, and response duration over time, including long-term follow-up of up to 15 years. The study involves multiple visits and assessments over months and years to gather detailed safety and outcome information.

CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML