HERIZON-BTC-01
The single-arm trial that won zanidatamab its approval: about four in ten patients with HER2-positive bile duct or gallbladder cancer responded after chemotherapy had failed.
Overview
HERIZON-BTC-01 enrolled 87 patients at 32 sites in nine countries between September 2020 and March 2022; 80 were HER2 immunohistochemistry 2+ or 3+ (cohort 1). Zanidatamab 20 mg/kg every two weeks produced confirmed objective responses in 33 of 80 (41.3 percent, 95 percent CI 30.4 to 52.8) by independent central review; 18 percent had grade 3 treatment-related adverse events and there were no treatment-related deaths. It supported the FDA accelerated approval of November 2024 and is being confirmed in first line by HERIZON-BTC-302.
- 41.3 out of 100 people had their tumour shrink with Zanidatamab.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- 18 out of 100 people reached this endpoint with Zanidatamab.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- These results apply to the people the trial enrolled: HER2-amplified unresectable or metastatic biliary tract cancer after gemcitabine-based therapy: single-arm zanidatamab. People in a different situation may not see the same effect.
- The trial selected people by a biomarker (HER2); the result should not be assumed for people whose cancer does not have it.
- Only 87 people took part, so the numbers are less certain than in a large trial.
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.
87 enrolled.
Notes
top- Key figures: 87 enrolled at 32 sites in nine countries (80 in cohort 1, HER2 IHC 2+ or 3+); confirmed response by independent central review 41.3 percent (33 of 80; 30.4 to 52.8) at a median follow-up of 12.4 months; grade 3 treatment-related adverse events 18 percent (diarrhoea 5, reduced ejection fraction 3), no grade 4 or treatment-related deaths. Approvals: FDA accelerated November 2024 (IHC 3+), European Commission conditional 27 June 2025, MHRA 19 February 2026, NICE TA1153 7 May 2026 (HER2 IHC 3+, after at least one line, commercial arrangement; FOLFOX or active symptom control as comparators).
- UK sites (3, registry): University College London Hospitals, Royal Free London, Imperial College Healthcare (all London).
- Tumour type split from the Ziihera label: gallbladder cancer 53%, intrahepatic cholangiocarcinoma 27%, extrahepatic cholangiocarcinoma 19%.
A lower, more conservative prevalence than the 2017 meta-analysis, using the trial's own definition. Heterogeneity means a small biopsy can miss HER2, which argues for testing resection specimens and for reflex testing rather than testing on request.
One cancer page on OnCo cites this paper by its DOI; this record gives the citation a page of its own so a reader can follow it without leaving OnCo. Read the abstract above alongside the citing page listed under Related; the record was created automatically from the Europe PMC entry and its figures have not been checked by hand.
Similar pages
not linked directly; found by shared links- TrialACTICCA-1
Shares Extrahepatic cholangiocarcinoma (perihilar and distal), Intrahepatic cholangiocarcinoma, Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups) and the tag gallbladder-evidence.
- Key paperHER2/HER3 pathway in biliary tract malignancies; systematic review and meta-analysis: a potential therapeutic target?
Shares Reflex HER2 testing of every advanced gallbladder and extrahepatic biliary cancer, Extrahepatic cholangiocarcinoma (perihilar and distal), Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Biliary tract cancer (all types) and the tag gallbladder-evidence.
- TrialSWOG S0809
Shares Extrahepatic cholangiocarcinoma (perihilar and distal), Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (cholangiocarcinoma) and the tag gallbladder-evidence.
- TrialNIFTY
Shares Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma) and the tag gallbladder-evidence.
- Key paperLiposomal irinotecan plus fluorouracil and leucovorin versus fluorouracil and leucovorin for metastatic biliary tract cancer after progression on gemcitabine plus cisplatin (NIFTY): a multicentre, open-label, randomised, phase 2b study
Shares Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer and the tag gallbladder-evidence.
- Key paperSecond-line FOLFOX chemotherapy versus active symptom control for advanced biliary tract cancer (ABC-06): a phase 3, open-label, randomised, controlled trial
Shares Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer and the tag gallbladder-evidence.
- Key paperBiliary tract cancer: ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up
Shares Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer and the tag gallbladder-evidence.
- Key paperReal-world analysis of ctDNA and other biomarkers in patients with curatively resected stage I-III biliary tract cancer
Shares Gallbladder cancer roadmap: from a chance finding at gallstone surgery to a disease with its own trials, Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer and the tag gallbladder-evidence.