Evaluating Olutasidenib Treatment for Adults with IDH1-mutated CCUS, Lower-risk MDS, or CMML

What this Trial Is About

Researchers are evaluating olutasidenib, an investigational drug, in adults with IDH1-mutated Clonal Cytopenia of Undetermined Significance CCUS and lower-risk Myelodysplastic Syndromes MDS or Chronic Myelomonocytic Leukemia CMML. The study aims to determine the response rate to olutasidenib monotherapy and assess its safety. Secondary goals include evaluating transfusion independence, survival, leukemia transformation rates, and reduction in IDH1 clone size. Exploratory objectives focus on gene expression and DNA methylation profiles to identify predictors of treatment response or resistance. Participants take olutasidenib orally twice daily on days 1 to 28 of each 28-day cycle. Those with CCUS receive treatment for up to 18 months, while patients with lower-risk MDS or CMML may continue treatment until disease progression or unacceptable side effects occur. Blood samples and bone marrow biopsies are collected during the study to monitor disease status and treatment effects. During the study, participants are closely monitored for safety and adverse events for about one year. After finishing treatment, follow-up visits occur every three months for up to three years to track long-term outcomes. Researchers assess blood counts, mutation levels, and other health measures to understand the drugs impact and any risks involved. Total participation may last several years, including treatment and follow-up periods.

A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.