OnCo
trialsTrialPositive

TOPAZ-1

TOPAZ-1 was the first immunotherapy success in bile duct cancer, with a small median gain but a growing tail of long survivors.

OS 12.9 vs 11.3 months (HR 0.76 updated); 24-month OS 23.6% vs 11.5%; 3-year OS update (J Hepatol 2025) confirmed the durable tail. Approved September 2022.

Setting
First-line advanced biliary tract cancer: gemcitabine-cisplatin + durvalumab vs + placebo
Phase
Phase 3
Sponsor
AstraZeneca
Registry
Headline result
OS HR 0.76; 2-year OS 23.6% vs 11.5%.
Reported
2022
Enrolled
685
Replication
KEYNOTE-966 (pembrolizumab) reproduced the class effect.

Outcomes

2top
In plain words
What these results mean for people, not percentages
685 people took part
Overall survival (updated)primarysurvival endpoint
  • Median 12.9 vs 11.3 months with Durvalumab + GemCis compared with Placebo + GemCis; about 1.6 months longer for half the group.
  • A median is a midpoint: half the people did better than this and half did worse, so it is not a prediction for any one person.
  • Put another way, the treated group had about 24 percent lower chance of the event at any given time (hazard ratio 0.76, likely range 0.64 to 0.91).
24-month overall survivalsurvival endpoint
  • 23.6 vs 11.5 out of 100 alive with Durvalumab + GemCis compared with Placebo + GemCis; 12.1 more per 100.
  • Roughly one extra person helped for every 8 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
Be careful
  • Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
  • These results apply to the people the trial enrolled: First-line advanced biliary tract cancer: gemcitabine-cisplatin + durvalumab vs + placebo. People in a different situation may not see the same effect.

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 participants enrolled.

Overall survival (updated)primary
HR 0.76 (0.64–0.91)
Durvalumab + GemCis
12.9 mo
Placebo + GemCis
11.3 mo
24-month overall survival
Durvalumab + GemCis23.6 of 100
Placebo + GemCis11.5 of 100
EndpointArmnValueHR (95% CI)pSource
Overall survival (updated)primaryDurvalumab + GemCis34112.9 months0.76 (0.64–0.91)
Placebo + GemCis34411.3 months
24-month overall survivalDurvalumab + GemCis23.6%
Placebo + GemCis11.5%
Replication
KEYNOTE-966 (pembrolizumab) reproduced the class effect.

Connected

5top