A Phase 1 Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
City of Hope Medical Center
Summary
This phase I trial studies the side effects and best dose of BSB-2002 when given after cyclophosphamide and fludarabine and tests how well it works in treating NPM1-mutated acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). BSB-2002 is a type of personalized autologous T cell receptor-modified T cell therapy. T cells are infection fighting blood cells that can kill cancer cells. The T cells given in this study come from the patient and have a new gene put in them that makes them able to recognize mutated NPM1, a protein on the surface of cancer cells. These NPM1 mutated-specific T cells may help the body's immune system identify and kill NPM1-mutated AML cells. Giving chemotherapy, such as cyclophosphamide and fludarabine, before BSB-2002 helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. Giving BSB-2002 after cyclophosphamide and fludarabine may be safe, tolerable, and/or effective in treating relapsed or refractory AML in patients with NPM1 mutation.
Description
PRIMARY OBJECTIVE: I. Evaluate the safety of BSB-2002. SECONDARY OBJECTIVES: I. Cellular kinetics of BSB-2002 in peripheral blood through day 365. II. Evaluate the efficacy of BSB-2002 based on IIa. Percentage of patients with complete remission (CR; including CR with measurable residual disease negative \[CRMRD-\], CR, CR with incomplete hematologic recovery \[CRi\], CR with partial hematologic recovery \[CRh\]) or partial remission (PR) as determined by the investigator according to the European LeukemiaNet (ELN) criteria for AML; IIb. Overall survival; IIc. Duration of response (time fro…