ClarIDHy

ClarIDHy, trial NCT02989857 sponsored by Agios, now Servier, and reported in 2020, was the first randomised trial of a targeted drug in bile duct cancer, testing ivosidenib against placebo in previously treated IDH1-mutant cholangiocarcinoma. It randomised 187 patients, met its primary progression-free survival endpoint with a large relative effect, and showed an overall survival benefit once adjusted for crossover, but the response rate was only about two percent, so it slowed the disease without shrinking it. OnCo links it to biliary tract cancer, IDH1 and IDH2 as targets, ivosidenib and Ghassan K. Abou-Alfa. Whether a drug that stabilises rather than shrinks tumours is worth its cost in a setting with few alternatives is the open question.