A randomized, double-blind, multicenter phase II AIO trial with gemcitabine plus sorafenib versus gemcitabine plus placebo in patients with chemotherapy-naive advanced or metastatic biliary tract cancer: First safety and efficacy data.

4077 Background: Despite recent progress in chemotherapy (Valle, 2010), new therapy options are urgently needed for advanced or metastatic biliary tract cancer (mBTC). The objectives of this randomized, double-blind, multicenter phase II trial were safety and clinical activity of gemcitabine (GEM) combined with sorafenib versus placebo (Sor/Pl) as first-line therapy in mBTC. Methods: GEM (1,000 mg/m²) was given on day 1, 8, 15, 22, 29, 36, 43 in first cycle and day 1, 8, 15 of all other cycles. Sor/Pl tablets were administered as a dose of 400 mg bid orally. Preliminary incomplete data of 103 full recruited, but still blinded patients could be analysed. Since patients are still under treatment, data are shown for both groups together. Results: Out of 62 patients, 9/40/13 had gallbladder, intrahepatic or hepatic BTC, respectively. Median age was 64.5 years (range 36-93). Median size of target lesions was 80 mm (range 10-271). Median time after diagnosis was 25 days (range 1-2633). Reasons for treatment end...