Eltanexor and Venetoclax in Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia

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.

NCT Number	NCT06399640
Lead Sponsor	Vanderbilt-Ingram Cancer Center Collaborators: Karyopharm Therapeutics Inc, National Cancer Institute (NCI)
Conditions	Relapsed Myelodysplastic Syndrome, Refractory Myelodysplastic Syndrome, Acute Myeloid Leukemia, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Enrollment	60 participants
Start Date	2024-08-14
Primary Completion	2027-02-01 (estimated)
Study Completion	2027-10-01 (estimated)
Updated on ClinicalTrials.gov	2026-06-24