NCT05282459

Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation

A Phase Ib/II, Single Center, Open-Label, Safety and Efficacy Study to Improve Anemia in Subjects on Enasidenib With Lower Risk Myelodysplastic Syndrome and Non-proliferative Chronic Myelomonocytic Leukemia Without an IDH2 Mutation

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Tian Yi Zhang · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 1b/2, open-label, single arm study to evaluate if enasidenib is safe and effective in improving anemia and decreasing transfusion needs in subjects diagnosed with lower risk myelodysplastic syndrome (MDS) or nonproliferative chronic myelomonocytic leukemia (CMML) without a mutation in isocitrate dehydrogenase type 2 (IDH2 wildtype). Other objectives include assessment of improvements in platelet production and characterization of the mechanism of action of enasidenib in enhancing endogenous erythropoiesis.

Detailed description

Primary Objective(s)- To determine the efficacy (response rate) of enasidenib in improving anemia and decreasing RBC transfusion dependence. Secondary Objective(s)- To determine the tolerability, safety and durability of the erythroid response and identify laboratory parameters as clinical markers of response.

Conditions

• Leukemia
• Leukemia, Myeloid
• Monocytic Leukemia

Interventions

Type	Name	Description
DRUG	Enasidenib mesylat dose escalation	Subjects will participate dose escalation with a starting dose of 100 mg. Enasidenib will be self administered orally and daily.

Timeline

• Start date: 2022-01-12
• Primary completion: 2024-12-03
• Completion: 2025-03-03
• First posted: 2022-03-16
• Last updated: 2026-04-17
• Results posted: 2026-04-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study