Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study
Isocitrate dehydrogenase 1 (IDH1) mutations occur in approximately 13% of patients with intrahepatic cholangiocarcinoma. 185 patients were randomly assigned to ivosidenib (AG-120) or placebo. Median follow-up for progression-free survival was 6·9 months. Progression- free survival was significantly improved with ivosidenib compared with placebo. Serious adverse events were reported in 36 (30%) of 121 patients receiving ivosidanib and 13 (22%) of 59 patients receiving placebo.
