Testing a new medication for rare blood cell disorders with a specific genetic mutation

This trial is testing a medication called olutasidenib in people who have rare blood disorders—specifically clonal cytopenia of undetermined significance, myelodysplastic syndromes, or chronic myelomonocytic leukemia—and who have a specific genetic change called an IDH1 mutation. Researchers want to see if this medication can help your bone marrow make more healthy blood cells, reduce the need for blood transfusions, and slow or stop the disease from progressing. You would take the medication twice a day as a capsule for as long as it's helping and you're tolerating it well.

People with these rare blood disorders often have low blood cell counts that require frequent transfusions and can be life-threatening. This trial exists because early research suggests that blocking the effects of the IDH1 gene mutation might help the bone marrow produce healthy blood cells again, potentially improving patients' quality of life and survival.