Phase 1 Open-Label Study of ASTX030 to Evaluate Drug Levels in Adults With Myelodysplastic Syndrome

What this Trial Is About

Researchers are evaluating ASTX030, an oral azacitidine AZA formulation combined with cedazuridine CED tablets, in patients with Myelodysplastic Syndrome MDS. This Phase 1 study aims to identify doses of these oral formulations that achieve a similar total exposure AUC to that of the standard subcutaneous AZA injection at 75 mgm2, including separate parts focusing on tolerability and expansion assessments. During the tolerability assessment part, patients receive subcutaneous AZA on Day 1 of Cycle 1 followed by oral AZA and CED on Days 2 to 7, with oral dosing continued on Days 1 to 7 in subsequent cycles. The expansion assessment part uses a crossover design where patients alternate between ASTX030 and subcutaneous AZA in the first two cycles, then continue ASTX030 from Cycle 3 onward, with each cycle lasting 28 days. Participants will be monitored through pharmacokinetic measurements comparing total AUC of AZA between oral and injection forms up to 2 months for the expansion part and up to 1 month for the tolerability part. Safety and tolerability are assessed, and participants must meet specific organ function and performance status criteria. The study spans multiple cycles with scheduled dosing and evaluations to compare the investigational oral treatment to standard injection therapy.

A Phase 1 Trial of ASTX030 in Patients With Myelodysplastic Syndrome