Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation
United States17 participantsStarted 2022-01-12
Plain-language 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.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Documented diagnosis of
✓. No disease-modifying therapy (HMA, hydrea) within 2 months of starting study
✓. Age ≥ 18 years of age
✓. ECOG ≤ 3
✓. Negative for IDH2 mutation by NGS or multiplex PCR (SNaPshot)
✓. Has symptomatic anemia defined as hemoglobin \< 10.5 g/dL with any of the following.
✓. Stated willingness to comply with all study procedures and availability for the duration of the study
✓. Ability to take oral medication and be willing to adhere to the medication regimen.
Exclusion criteria
✕. Use of concurrent other erythropoietic agents (including epoetin, darbepoetin), G-CSF within 30 days of study enrollment
. 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.