Study design
The methods of this open-label, nonrandomized, first-in-human study of asciminib (Supplementary Fig. The primary objective was to determine the maximum tolerated dose and/or recommended dose for the expansion of asciminib monotherapy. Secondary objectives included assessing safety, tolerability, preliminary antileukemic activity, and pharmacokinetic profile in plasma of asciminib. Additional details are in the Supplementary Methods.