A Phase II Multicenter Trial of Rucaparib and Nivolumab as Maintenance Therapy in Patients with Advanced Biliary Tract Cancer: BilT-02

This phase II trial investigated rucaparib [poly (ADP-ribose) polymerase (PARP) inhibitor] in combination with nivolumab in patients with advanced biliary tract cancer (BTC) without progression after 4 to 6 months of first-line platinum-based chemotherapy.

The primary endpoint was the 4-month progression-free survival (PFS) rate with null and alternative hypotheses of 63% and 85%. Secondary endpoints included the PFS rate in patients with DNA damage response (DDR) and isocitrate dehydrogenase 1 (IDH1) mutations, median PFS and overall survival (OS) from the start of study treatment (PFS1 and OS1) and first-line chemotherapy (PFS2 and OS2), best overall response, and safety (NCT03639935).