A Phase 1 Study of Uproleselan Plus Conditioning Chemotherapy and Stem Cell Transplantation in People With Very High-Risk Acute Myeloid Leukemia


Full Title

A Phase 1/2 Trial of Uproleselan Combined with High Dose Busulfan Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for Patients with Chemotherapy Resistant Acute Myeloid Leukemia


Researchers want to find the best dose of uproleselan to give to people undergoing a stem cell transplant (SCT). The people in this study have acute myeloid leukemia (AML) that is very hard to treat. It keeps growing even after intensive chemotherapy. SCT involves giving healthy blood-forming cells (stem cells) from a donor to replace diseased cells in your bone marrow.

Before SCT, people normally get standard conditioning chemotherapy to weaken their immune system. This prepares your body for the transplant. Uproleselan blocks a protein called E-selectin, which helps protect cancer cells from chemotherapy. By blocking this protein, uproleselan may make AML cells more sensitive to conditioning chemotherapy. Uproleselan is given intravenously (by vein).

Who Can Join

To join this study, there are a few conditions. You must:

  • Have AML that keeps growing even after chemotherapy.
  • Be planning to have a stem cell transplant.
  • Be well enough to walk and take care of yourself. You must be able to do activities such as office work or light housework.
  • Be age 1-30 years.


For more information about this study and to see if you or your child can join, please call 833-MSK-KIDS.




Phase I/II (phases 1 and 2 combined)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID