This is the report of a phase 1 study assessing safety and preliminary activity of ivosidenib in 73 patients with previously treated IDH1-mutant advanced cholangiocarcinoma (CCA). 46 (63%) patients had treatment-related adverse events (TRAEs), grade ≥3 TRAEs were: 2 fatigue, 1 decreased blood phosphorus, and 1 increased blood alkaline phosphatase. 4 (5%) patients had a partial response; median progression-free survival (PFS) was 3.8 months (95%CI 3.6-7.3), 6-month PFS was 40.1% (95%CI 28.4-51.6), and 12-month PFS was 21.8% (95%CI 12.3-33.0). Median overall survival was 13.8 months (95%CI 11.1-29.3) (data censored for 48 (66%) patients). It has been recently announced that the randomized phase 3 ClarIDHy trial of ivosidenib versus placebo in previously treated IDH1-mutant CCA patients met its primary endpoint of PFS.

(Lowery MA et al. – Lancet Gastroenterology & Hepatology, 9 July 2019)

Read more