6/30/23, 2:17 AM TOPAZ-1 Pathway Feedback Search Clinical Topics Home Studies TOPAZ 1 TOPAZ 1 Disease Disease Cholangiocarcinoma Gallbladder cancer Trial question What is the role of durvalumab plus chemotherapy in patients with advanced biliary tract cancer? Study design Multi-center Double blinded RCT Population Characteristics of study participants 50.0% female N = 685 50.0% male 685 patients (340 female, 345 male) Inclusion criteria: patients with previously untreated unresectable or metastatic biliary tract cancer or with recurrent disease Key exclusion criteria: ampullary carcinoma; active or prior documented autoimmune or inflammatory disorders; known allergy or hypersensitivity to any study treatment Interventions N=341 durvalumab (1500 mg durvalumab plus gemcitabine and cisplatin) N=344 placebo (matching placebo plus gemcitabine and cisplatin) Primary outcome Overall survival https://web.pathway.md/studies/rec3OAXMDoELlr2at 1/2 6/30/23, 2:17 AM TOPAZ-1 Pathway 41.9 % 41.9 34.3 31.4 % 20.9 % Significant increase 10.5 % NNT = 13 0.0 % Durvalumab Placebo Significant increase in overall survival (41.9% vs. 34.3%; HR 1.22, 95% CI 1.03 to 1.52) Secondary outcomes Significant increase in progression-free survival (7.2 months vs. 5.7 months; HR 1.33, 95% CI 1.12 to 1.59) Borderline significant increase in objective response rate (26.7% vs. 18.7%; OR 1.6, 95% CI 1.11 to 2.31) Safety outcomes No significant difference in grade 3 or 4 adverse events. Conclusion In patients with previously untreated unresectable or metastatic biliary tract cancer or with recurrent disease, durvalumab was superior to placebo with respect to overall survival. Reference Do-Youn Oh, M.D., Ph.D. et al. Durvalumab plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer. NEJM Evid. 2022;1(8). Open reference URL https://web.pathway.md/studies/rec3OAXMDoELlr2at 2/2