A Phase 1 Study of Ziftomenib Combinations in People With Acute Myeloid Leukemia


Full Title

Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed/Refractory Acute Myeloid Leukemia


Researchers want to find the best doses of ziftomenib to use with other drugs to treat leukemia. The people in this study have acute myeloid leukemia (AML) that keeps growing even with treatment. In addition, they have AML with changes in the NPM1, KMT2A, or FLT3 genes.

A protein called menin can promote the growth and survival of cancer cells. Ziftomenib blocks the menin pathway, which may help slow or stop AML growth. Ziftomenib is taken orally (by mouth).

If you join this study, you will take ziftomenib plus one of these other leukemia treatments:

  • FLAG-IDA (fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin)
  • LDAC (low-dose cytarabine)
  • Gilteritinib

Who Can Join

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

  • Have AML that keeps growing or came back after treatment.
  • Have AML with changes in the NPM1, KMT2A, or FLT3 genes.
  • Have completed prior leukemia treatment at least 2 weeks before getting the study treatment.
  • Be able to walk and do routine activities for more than half the time you are awake.
  • Be age 18-75.


For more information and to see if you can join this study, please call Dr. Aaron Goldberg’s office at 646-608-3752.




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID