Omacetaxine and azacitidine for untreated patients with myelodysplastic syndromes and excess blasts: a phase I/II clinical trial.

ABSTRACT:

Background

Patients with myelodysplastic syndromes (MDS) with excess blasts (MDS-EB) have poor long-term outcomes. Our preclinical studies showed MDS-EB stem cells are dependent upon protein synthesis. We designed a phase 1/2 clinical trial to examine the safety/efficacy of the protein synthesis inhibitor omacetaxine mepesuccinate (oma) with the hypomethylating agent (HMA) azacitidine (aza) for patients with untreated MDS-EB.

Methods

Enrollment occurred from September 2018 to March 2024 and the study was registered at clinicaltrials.gov (NCT03564873). The phase 1 primary endpoint was to determine the maximum tolerated dose (MTD) and the phase 2 primary endpoint was to determine the overall response rate. Aza 75 mg/m² was administered daily and oma twice daily days

SUBMITTER: Pollyea DA 

PROVIDER: S-EPMC12547833 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature