A Phase 1 Study of Uproleselan Plus Fludarabine and Cytarabine to Treat Acute Myeloid Leukemia, Myelodysplastic Syndromes, and Mixed Phenotype Acute Leukemia in Children


Full Title

A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy (PEPN2113) (CIRB)


In this study, researchers are seeking the best dose of uproleselan to give with fludarabine and cytarabine in children with cancer. The children in this study have acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or mixed phenotype acute leukemia. Their cancers keep growing even after treatment. In addition, their cancer cells make a protein called E-selectin ligand.

Uproleselan binds to E-selectin in the bone marrow. It prevents bone marrow cells from interacting with cancer cells that make E-selectin ligand. This may keep leukemia cells from hiding in the bone marrow and escaping the effect of chemotherapy. Giving uproleselan with fludarabine and cytarabine may therefore make these drugs work better against cancer cells.

Who Can Join

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

  • Have AML, MDS, or mixed phenotype acute leukemia that keeps growing or came back after treatment. The cancer must be in second or greater relapse.
  • Have cancer that makes the E-selectin ligand protein.
  • Have recovered from the serious side effects of previous treatments before getting the study treatment.
  • Be able to walk and do routine activities for more than half the time your child is awake.
  • Be under age 18.


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




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID