Omacetaxine and Venetoclax for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1

Summary

This phase Ib/II trial best dose, possible benefits and/or side effects of omacetaxine and venetoclax in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory) and have a genetic change RUNX1. Drugs used in chemotherapy, such as omacetaxine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving omacetaxine and venetoclax may help to control the disease.

Timeline

Start

2021-12-17

Primary completion

2024-09-16

Completion

2024-09-16

Publications

• DiNardo CD, Jen WY, Montalban-Bravo G, Wang X, Loghavi S, Lavu S, Short NJ, Chien K, Issa GC, Pemmaraju N, Yilmaz M, Andreeff M, Borthakur G, Kadia TM, Daver NG, Garcia-Manero G, Mill CP, Su X, Fiskus W, Bhalla KN. Omacetaxine and venetoclax in relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome with mutant RUNX1. Blood Neoplasia. 2025 Jul 25;2(4):100145. doi: 10.1016/j.bneo.2025.100145. eCollection 2025 Nov.

Drugs

Indications