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In the ROAR basket trial, the BRAF-blocking pair dabrafenib and trametinib shrank tumours in about half of 43 people with bile duct or gallbladder cancer carrying the BRAF V600E mutation, which is found in roughly 5 percent of biliary cancers. The pair is approved for any solid tumour with this mutation in the United States and is listed by guidelines for biliary cancer.
ROAR (NCT02034110) was a phase 2, open-label, single-arm, multicentre basket trial of dabrafenib plus trametinib in BRAF V600E-mutated rare cancers across nine cohorts (anaplastic thyroid, biliary tract, gastrointestinal stromal tumour, small-bowel adenocarcinoma, low- and high-grade glioma, hairy cell leukaemia, multiple myeloma and adenocarcinoma of unknown primary). The biliary tract cohort enrolled 43 previously treated patients between 2014 and 2018; investigator-assessed overall response, the primary endpoint, was achieved by 22 of 43 (51 percent, 95 percent confidence interval 36 to 67) and independent-review response by 20 of 43 (47 percent), at a median follow-up of 10 months. The most common grade 3 or worse adverse event was raised gamma-glutamyltransferase (12 percent); 40 percent had serious adverse events, 21 percent treatment-related, most often pyrexia. BRAF V600E is found in about 5 percent of biliary tract tumours and is less common in gallbladder cancer than in intrahepatic cholangiocarcinoma. The FDA approved dabrafenib with trametinib for BRAF V600E solid tumours on a tumour-agnostic basis in June 2022 with ROAR among the supporting trials; NCCN lists the pair for biliary cancer, and in the UK the combination is not appraised by NICE for biliary cancer.
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.
A second publication from the ROAR 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.
This is the paper Europe PMC returns for registry id NCT02034110 with the most citations, so it is the natural first reading for anyone following the ROAR trial. Read the abstract above alongside the trial page and the registry entry; the record was linked automatically and its figures have not been checked by hand.
Shares Dabrafenib, Dabrafenib + trametinib, Trametinib, BRAF.
Shares Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer.
Shares Dabrafenib, Trametinib, Novartis, Small-molecule kinase inhibitors.
Shares Rare cancers, Tumour-agnostic (tissue-agnostic) approval, Basket, umbrella, and platform trials, Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups).
Shares Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer.
Shares Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer.
Shares Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer.
Shares Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups), Biliary tract cancer (all types), Biliary tract cancer (cholangiocarcinoma), Gallbladder cancer.