A Study of Enasidenib in People with Unexplained Low Blood Cell Counts


Full Title

A Pilot Study of Enasidenib for Patients with Clonal Cytopenia of Undetermined Significance and Mutations in IDH2


The purpose of this study is to see how the drug enasidenib works in people with a condition called “clonal cytopenia of undetermined significance” (CCUS). People with CCUS have low levels of certain kinds of blood cells, but it is not known why. Some of these blood cells have a mutation in a gene called IDH2. They may have a higher chance of developing a precancerous condition called myelodysplastic syndrome (MDS) or a blood cancer called acute myeloid leukemia (AML).

Enasidenib blocks the mutated IDH2 protein. It is already used to treat people with AML with an IDH2 mutation. In this study, researchers are assessing the safety and effectiveness of enasidenib in people with CCUS and IDH2 mutations. They hope that enasidenib may improve blood cell counts. Enasidenib is a tablet that is taken orally (by mouth).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have unexplained low blood cell counts for at least 6 months, with blood cells containing an IDH2 mutation.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for people age 18 and older.

For more information about this study and to ask about eligibility, please contact the office of Dr. Eytan Stein at 646-608-3749.




Phase I (phase 1)



ClinicalTrials.gov ID