A Randomized Phase II Study Of Sapacitabine In MDS Refractory To Hypomethylating Agents

Sapacitabine is an orally administered nucleoside analogue which causes single-strand DNA breaks and induces G2 cell cycle arrest. A multi-center, randomized phase 2 study was conducted to evaluate 3 dose regimens in older patients with MDS refractory to hypomethylating agents. The primary endpoint was 1-year survival. Secondary endpoints included the rate of CR, CRp, PR, major hematological improvement (HI) or stable disease and their corresponding durations. The study used a selection design to identify a dose regimen which produced a better 1-year survival rate in the event that all three dose regimens were active. The planned sample size was 60 patients (20 patients in each arm).