A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance (NCT05102370) | Clinical Trial Compass
CompletedPhase 1
A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance
United States4 participantsStarted 2021-10-06
Plain-language summary
Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* Age ≥18 years at the time of signing the informed consent form.
* Willing and able to adhere to the study visit schedule and other protocol requirements.
* Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds:
* Hgb \<10 g/dL
* ANC \<1.8 × 10\^9/L
* Platelets \<100 × 10\^9/L
* Results of bone marrow biopsy within 1 month of study entry (screening bone marrow biopsy) must not indicate hematologic disease.
* IDH2 gene mutation (R140 or R172), performed locally, at a frequency \>2%.
* ECOG performance status of 0-2.
* Adequate organ function, defined as:
* Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤3x upper limit of normal (ULN), unless the subject has Gilbert's syndrome.
* Serum total bilirubin \<1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis. In these cases, approval from the study Principal Investigator is required.
* Creatinine clearance greater than 50 mL/min based on the Cockroft-Gault glomerular filtration rate estimation.
* Patients being enrolled on study on the basis of anemia, will only be eligible if folate, B12, serum iron, serum ferritin, total iron binding capacity, haptoglobin and peripheral smear within normal limits
* Women of childbearing potential may participate provided they have a negative serum pregnancy test at …