Phase 2
N=17
Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation
Leukemia · Leukemia, Myeloid · Monocytic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT05282459 ↗Enrolled (actual)
17
Serious AEs
58.8%
Results posted
Apr 2026
Primary outcome: Primary: Clinical Response: Hematological Improvement - Erythroid (HI-E) — 0; 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Enasidenib mesylat dose escalation (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Tian Yi Zhang
- Primary completion
- Dec 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Response: Hematological Improvement - Erythroid (HI-E) |
0; 0 | — |
| SECONDARY Related Adverse Events |
0; 0; 20; 38 | — |
| SECONDARY Time to Hematological Improvement - Erythroid (HI-E) |
— | — |
| SECONDARY Duration of Hematological Improvement - Erythroid (HI-E) |
— | — |
| SECONDARY Clinical Response: Hematological Improvement - Platelets (HI-P) |
0; 1 | — |
| SECONDARY Clinical Response: Hematological Improvement - Neutrophils (HI-N) |
0; 1 | — |
| SECONDARY Red Blood Cell (RBC) Transfusion Independence (RBC TI) |
0; 0 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Documented diagnosis of
- MDS according to WHO/FAB classification that meets IRSS-R classification of low or intermediate risk disease; and a diagnosed as denovo or secondary MDS (MDS-RS eligible if refractory to or declined luspatercept therapy) OR
- Dysplastic (nonproliferative) CMML with WBC 30 mL/minute, calculated by Cockcroft-Gault formula
- Ability to understand and the willingness to sign the IRB approved informed consent document.
- Women of childbearing potential must have negative urine or serum pregnancy test
Exclusion Criteria
- Use of concurrent other erythropoietic agents (including epoetin, darbepoetin), G-CSF within 30 days of study enrollment
- Less than 3 months of life expectancy
- Significant cardiac disease (NYHA Class IV congestive heart failure, or unstable angina or myocardial infarction within the last 6 months
- Harbor IDH2 somatic mutations by NGS or PCR
- Pregnant or breast feeding
- Any uncontrolled bacterial, fungal, viral or other infection.
- No known HIV+ or active hepatitis B or C infection, defined as positive viral load for HBV or HCV or a positive surface antigen (HBsAg) test for hepatitis B.
- Have other causes of anemia: deficiencies in iron, B12, folate; nutritional deficiencies related to gastric surgery, anorexia nervosa, excessive zinc supplementation; gastrointestinal bleed. If nutritional deficiencies can be corrected, potential subject can be rescreened and enrolled if nutritionally replete and still meets eligibility criteria.
- Any other medical history, including laboratory results, deemed by the Principal Investigator likely to interfere with their participation in the study, or to interfere with the interpretation of the results
- Pregnant or breast feeding
Data sourced from ClinicalTrials.gov (NCT05282459). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.