Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.
Hgb <10 g/dL
ANC <1.8 × 109/L
Platelets <100 × 109/L
IDH2 gene mutation (R140 or R172), performed locally, at a frequency ≥ 2%.
Indication of hematologic disease by bone marrow biopsy within 6 months of study entry.
Evidence of disease progression from time of bone marrow biopsy to enrollment based on investigator review of symptoms and complete blood counts
Active malignancy (defined as > 1 cm disease on most recent CT scan in the past 6 months).
Currently receiving therapy for solid tumor malignancy or received within the last 6 months.
Single group
Enasidenib
DrugProvided by BMS.
Best hematologic response
Hematologic response to enasidenib will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials
Toxicity as measured by the number of adverse events experienced by participant
Measured by CTCAE v 5.0
Change in mutant IDH2 variant allele fraction
The IDH2 variant allele fraction reflects the clonal dominance in the blood. Blood samples will be drawn at various time points throughout the study to determine the IDH2 variant allele fraction. The lower the IDH2 variant allele fraction the better the outcome.
Duration of hematologic improvement
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Washington University School of Medicine
Lead sponsor
Bristol-Myers Squibb
Collaborator
Damon Runyon Cancer Research Foundation
Collaborator
