Vanderbilt-Ingram Cancer Center
This phase I trial tests the safety, side effects, and best dose of eltanexor in combination with venetoclax for the treatment of patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Eltanexor works by trapping "tumor suppressing proteins" within the cell, thus causing the cancer cells to die or stop growing. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving eltanexor together with venetoclax may be safe, tolerable and/or effective in treating patients with relapsed or refractory MDS or AML.
Primary objective: • To establish the safe and biologically effective dose (BED) of eltanexor in combination with venetoclax in patients with R/R MDS and/or AML Secondary objectives: * To estimate the complete remission (CR) rate with eltanexor and venetoclax in patients with R/R MDS and/or AML * To assess the overall response rate (ORR) following treatment with eltanexor/venetoclax * To assess the overall survival of patients * To assess the progression free survival (PFS) and duration of response (DOR) in patients treated with eltanexor/venetoclax Exploratory objectives: * To assess dif…
Inclusion Criteria: \- Age \>/= 18 years at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be able to meet all study requirements. For Myelodysplastic Syndrome (MDS): Morphologically confirmed diagnosis of MDS with increased blasts (\>/= 5%), with a prior DNA methyltransferase inhibitor (DNMTi) treatment and progression after 2 cycles or stable disease after 4 cycles For Acute Myeloid Leukemia (AML): Morphologically confirmed diagnosis of AML in accordance with WHO diagnostic criteria that is relapsed or refractory following \>/= 1 line(s) of t…
Eltanexor will be taken by mouth
Venetoclax will be taken by mouth
Undergo bone marrow aspiration and biopsy
Undergo blood sample collection