biostudies-literatureUnknown10(6)Ueno MYakult Honsha<h4>Purpose</h4>Effective second-line chemotherapy options are limited in treating advanced biliary tract cancers (BTCs). Resminostat is an oral histone deacetylase inhibitor. Such inhibitors increase sensitivity to fluorouracil, the active form of S-1. In the phase I study, addition of resminostat to S-1 was suggested to have promising efficacy for pre-treated BTCs. This study investigated the efficacy and safety of resminostat plus S-1 in second-line therapy for BTCs.<h4>Methods</h4>Patients were randomly assigned to receive resminostat or placebo (200 mg orally per day; days 1-5 and 8-12) and S-1 group (80-120 mg orally per day by body surface area; days 1-14) over a 21-day cycle. The primary endpoint was progression-free survival (PFS). Secondary endpoints comprised overall survival (OCancer medicine2088-2099https://www.ebi.ac.uk/biostudies/studies/S-EPMC7957161biostudies-literatureA randomized, double-blind, phase II study of oral histone deacetylase inhibitor resminostat plus S-1 versus placebo plus S-1 in biliary tract cancers previously treated with gemcitabine plus platinum-based chemotherapy.PMC7957161Furukawa MNemoto NFukutomi AKomatsu YNakai YMoriwaki TKobayashi SShimizu STsuda MIshii HMorizane CUeno MKatanuma ANegoro YFuruse JSakai DIkeda MMakino KMizuno NKajiwara TMuto MOzaka MTsuji AfalseA randomized, double-blind, phase II study of oral histone deacetylase inhibitor resminostat plus S-1 versus placebo plus S-1 in biliary tract cancers previously treated with gemcitabine plus platinum-based chemotherapy.<h4>Purpose</h4>Effective second-line chemotherapy options are limited in treating advanced biliary tract cancers (BTCs). Resminostat is an oral histone deacetylase inhibitor. Such inhibitors increase sensitivity to fluorouracil, the active form of S-1. In the phase I study, addition of resminostat to S-1 was suggested to have promising efficacy for pre-treated BTCs. This study investigated the efficacy and safety of resminostat plus S-1 in second-line therapy for BTCs.<h4>Methods</h4>Patients were randomly assigned to receive resminostat or placebo (200 mg orally per day; days 1-5 and 8-12) and S-1 group (80-120 mg orally per day by body surface area; days 1-14) over a 21-day cycle. The primary endpoint was progression-free survival (PFS). Secondary endpoints comprised overall survival (O2021-01-01T00:00:00Z2021 Mar2025-04-20T02:30:22.194Z2025-04-20T02:30:22.194ZS-EPMC79571613363560510.1002/cam4.3813