A phase II placebo-controlled study of the effect and safety of nanvuranlat in patients with advanced biliary tract cancers previously treated by systemic chemotherapy

Nanvuranlat is a L-type amino acid transporter 1 inhibitor. A randomized phase 2 trial at 14 centers in Japan evaluated nanvuranlat for advanced biliary tract cancer (aBTC) after failure of systemic chemotherapy. Compared with placebo, nanvuranlat improved the progression-free survival (HR 0.56; p=0.02), with grade 3 or higher adverse events in 30.0% and 22.9% of patients in the nanvuranlat group and the placebo group, respectively. The overall survival was not statistically different between the nanvuranlat group and the placebo group.