Loading...
Cholangiocarcinoma is a chemotherapy-responsive disease. Although first-line chemotherapy with gemcitabine plus cisplatin is the standard treatment strategy, second- and later-line therapies have limited effectiveness. Genomic profiling indicates that cholangiocarcinoma has a number of targetable genes as well as genomic heterogeneity among intrahepatic, extrahepatic, and gallbladder primary cancers.
Investigators now report results of an industry-sponsored, international, double-blind, placebo-controlled, randomized, phase III trial (ClarIDHy) comparing ivosidenib, a small molecule inhibitor of mutated IDH-1, versus placebo in 185 patients (61%–65% female) with previously treated cholangiocarcinoma harboring an IDH-1 mutation. Most patients…