A Phase IB/II Study of Pinometostat and Azacitidine Chemotherapy for Acute Myeloid Leukemia with a Genetic Mutation


Full Title

A Phase Ib/II Study of the Histone Methyltransferase Inhibitor Pinometostat in Combination with Azacitidine in Patients with 11q23-Rearranged Acute Myeloid Leukemia (10200) (CIRB)


Azacitidine chemotherapy is a standard treatment for acute myeloid leukemia (AML). Pinometostat is an investigational drug that, when given with azacitidine, may help AML cells turn into healthy blood cells. The purpose of this study is to find the highest dose of pinometostat that can be given safely with azacitidine in patients with AML containing a genetic mutation called an 11q23 gene rearrangement, and to observe the effectiveness of this drug combination in patients.

Pinometostat works by inhibiting a protein called DOT1L that is involved in AML growth. Both medications used in this study are given intravenously (by vein).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have AML that continues to grow despite prior treatment and contains the 11q23 gene rearrangement.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Kamal Menghrajani at 212-639-2000.





Disease Status

Newly Diagnosed & Relapsed/Refractory