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TOPAZ-1 was the first immunotherapy success in bile duct cancer, with a small median gain but a growing tail of long survivors.
TOPAZ-1, trial NCT03875235 sponsored by AstraZeneca and reported in 2022, was the first immunotherapy success in bile duct cancer, adding durvalumab to gemcitabine and cisplatin in first-line advanced biliary tract cancer. It randomised 685 patients, met its primary overall survival endpoint with a small median gain but a growing tail of long survivors, confirmed at two years and in a three-year update in 2025, and led to approval in September 2022. OnCo links it to biliary tract and gallbladder cancer, durvalumab, gemcitabine plus cisplatin, the pairing of chemotherapy with PD-1 or PD-L1 blockade, Juan W. Valle and Do-Youn Oh, and KEYNOTE-966 with pembrolizumab reproduced the class effect. Which patients populate the durable tail, and whether a biomarker can find them in advance, is the open question.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
685 enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Overall survival (updated)primary | Durvalumab + GemCis | 341 | 12.9 months | 0.76 (0.64 to 0.91) | - | link |
| Placebo + GemCis | 344 | 11.3 months | ||||
| 24-month overall survival | Durvalumab + GemCis | - | 23.6% | - | - | link |
| Placebo + GemCis | - | 11.5% | ||||
| Overall survival (primary analysis) | Durvalumab + GemCis | 341 | - | 0.8 (0.66 to 0.97) | 0.021 | link |
| Placebo + GemCis | 344 | - | ||||
| 36-month overall survival (3-year update, median follow-up 41.3 months) | Durvalumab + GemCis | 341 | 14.6% | 0.74 (0.63 to 0.87) | - | link |
| Placebo + GemCis | 344 | 6.9% |
The tail of long survivors is the case for chemo-immunotherapy in gallbladder cancer, where the median gain is under two months. Who lands in that tail is still unknown; no biomarker in the trial predicts it.
A second publication from the TOPAZ-1 trial, later than the first and described in its title as an update or longer-term analysis. Read it with the primary publication linked from the trial page; the record was linked automatically and its figures have not been checked by hand.
Durvalumab with gemcitabine-cisplatin is a first-line standard for advanced biliary tract cancer, with pembrolizumab (KEYNOTE-966) as the alternative.
Shares A clinical trial or standard treatment for gallbladder cancer, Gemcitabine-cisplatin + PD-(L)1 blockade in biliary cancer, Report gallbladder cancer as its own population in every biliary trial, Gallbladder adenocarcinoma.
Shares Juan W. Valle, Gemcitabine + cisplatin, Extrahepatic cholangiocarcinoma (perihilar and distal), Intrahepatic cholangiocarcinoma.
Shares Report gallbladder cancer as its own population in every biliary trial, Gemcitabine + cisplatin, Extrahepatic cholangiocarcinoma (perihilar and distal), Intrahepatic cholangiocarcinoma.
Shares Juan W. Valle, Report gallbladder cancer as its own population in every biliary trial, Extrahepatic cholangiocarcinoma (perihilar and distal), Intrahepatic cholangiocarcinoma.
Shares Report gallbladder cancer as its own population in every biliary trial, Gemcitabine + cisplatin, Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, AstraZeneca.
Shares Gemcitabine + cisplatin, Extrahepatic cholangiocarcinoma (perihilar and distal), Intrahepatic cholangiocarcinoma, Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups).
Shares A clinical trial or standard treatment for gallbladder cancer, Gemcitabine + cisplatin, Extrahepatic cholangiocarcinoma (perihilar and distal), Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials.
Shares Extrahepatic cholangiocarcinoma (perihilar and distal), Intrahepatic cholangiocarcinoma, Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Durvalumab.