Final overall survival efficacy results of ivosidenib for patients with advanced cholangiocarcinoma with IDH1 mutation – The phase 3 randomized clinical ClarIDHy trial
This is the report of final overall survival (OS) results from the randomized phase 3 ClarIDHy trial, demonstrating the efficacy of ivosidenib vs placebo for patients with unresectable or metastatic cholangiocarcinoma with IDH1 mutation. 187 patients were randomized 2:1 to ivosidenib (N=126) or placebo (N=61); 43 patients crossed over from placebo to ivosidenib. Median OS was 10.3 months (95%CI 7.8-12.4 months) with ivosidenib vs 7.5 months (95%CI 4.8-11.1 months) with placebo (HR 0.79, 95%CI 0.56-1.12, P=0.09). When adjusted for crossover, median OS with placebo was 5.1
months (95%CI 3.8-7.6 months; HR 0.49, 95%CI 0.34-0.70, P<0.001). The most common grade ³3 treatment-emergent adverse event (TEAE) in both groups was ascites (11 patients [9%] receiving ivosidenib, 4 patients [7%] receiving placebo). Serious ivosidenib related-TEAEs were reported in 3 patients (2%). Patients receiving ivosidenib reported no decline in quality of life compared with placebo.