Phase 2 Study of Enasidenib Treatment for Adults with Clonal Cytopenia of Undetermined Significance and IDH2 Mutations

What this Trial Is About

Researchers are studying enasidenib, a drug that targets the mutated IDH2 protein, to see if it is a safe and effective treatment for people with clonal cytopenia of undetermined significance CCUS. This condition involves low blood cell counts without a clear cause, and the study aims to evaluate whether blocking the mutated protein can improve blood cell levels. This pilot study is conducted by Washington University School of Medicine and focuses on adults with specific IDH2 mutations. Participants will take enasidenib at a dose of 100 mg daily for up to 18 cycles, with each cycle lasting 28 days. Treatment will continue until the disease progresses to acute myeloid leukemia AML or myelodysplastic syndrome MDS, unacceptable side effects occur, or disease progression is suspected but the patient is still benefiting clinically. The study is designed as a single-group phase 2 trial where all participants receive the drug. During the study, researchers will monitor blood counts to assess the best hematologic response over approximately 17 months of treatment. They will also track side effects and changes in the mutated IDH2 gene levels at several points during treatment. Participants will undergo regular health evaluations including laboratory tests and will be observed for the duration of treatment and 30 days after the last dose to assess safety and effectiveness.

Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial