Clinical trial · Interventional
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
NCT05282459CI-TRIAL-00125661completedPhase 1 / Phase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Leukemia, Myeloid | Myeloid Leukemia | ALIAS | 0.90 |
| Monocytic Leukemia | Acute Monocytic Leukemia | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Enasidenib mesylat dose escalation | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Enasidenib mesylat
- description
- Participants will self administer the enasidenib orally everyday.
- interventionNames
- Drug: Enasidenib mesylat dose escalation
Primary outcomes (1)
- measure
- Clinical Response: Hematological Improvement - Erythroid (HI-E)
- timeFrame
- 16 weeks
- description
- Clinical response was assessed as the number of participants achieving a hematological improvement - erythroid (HI-E). Participants were characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows. * NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements * LTB = 0 units of RBC transfusions * HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions
Secondary outcomes (6)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria:
1. 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 \< 13.0/microL)
2. No disease-modifying therapy (HMA, hydrea) within 2 months of starting study
3. Age ≥ 18 years of age
4. ECOG ≤ 3
5. Negative for IDH2 mutation by NGS or multiplex PCR (SNaPshot)
6. Has symptomatic anemia defined as hemoglobin \< 10.5 g/dL with any of the following.
* Tachypnea
* Shortness of breath
* Fatigue
* Malaise
* Worsening of cardiovascular function
* Asthenia
* Dyspnea on exertion
* Angina
* Other subject symptoms the subject reports as being associated with being anemic.
7. Stated willingness to comply with all study procedures and availability for the duration of the study
8. Ability to take oral medication and be willing to adhere to the medication regimen.
9. Females of reproductive potential need to either commit to true abstinence from heterosexual contact or agree to use, and be able to comply with highly effective contraception without interruption, 28 days prior to starting enasidenib, during the study therapy, and for 30 days after last dose of enasidenib
10. For males of reproductive potential: agreement to use of condoms
11. Adequate organ function defined as:
* Hepatic function: total bilirubin \<1.5 x ULN (unless attributable to Gilbert's disease), AST or ALT \< 3x ULN
* Renal function: creatinine clearance \> 30 mL/minute, calculated by Cockcroft-Gault formula
12. Ability to understand and the willingness to sign the IRB approved informed consent document.
13. Women of childbearing potential must have negative urine or serum pregnancy test
Exclusion Criteria:
1. Use of concurrent other erythropoietic agents (including epoetin, darbepoetin), G-CSF within 30 days of study enrollment
2. Less than 3 months of life expectancy
3. Significant cardiac disease (NYHA Class IV congestive heart failure, or unstable angina or myocardial infarction within the last 6 months
4. Harbor IDH2 somatic mutations by NGS or PCR
5. Pregnant or breast feeding
6. Any uncontrolled bacterial, fungal, viral or other infection.
7. 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.
8. 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.
9. 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
10. Pregnant or breast feedingReferences
Publications (0)
Data not yet available
No reference posted for this study.