{"entity":{"id":"roar","kind":"trial","name":"ROAR (Rare Oncology Agnostic Research) basket: BRAF V600E biliary tract cancer cohort","aka":["ROAR"],"tldr":"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.","summary":"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.","status":"positive","asOf":"2026-09-24","links":[{"label":"ClinicalTrials.gov NCT02034110","url":"https://clinicaltrials.gov/study/NCT02034110"},{"label":"Subbiah et al., Lancet Oncology 2020: dabrafenib plus trametinib in BRAF V600E biliary tract cancer (ROAR)","url":"https://doi.org/10.1016/S1470-2045(20)30321-1"}],"tags":[],"related":[],"cancers":["gallbladder","cholangiocarcinoma","biliary-tract-cancer","rare-cancers"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":["dabrafenib-trametinib","dabrafenib","trametinib"],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["tumour-agnostic","basket-umbrella-platform"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-roar-lancet-oncol-2020","paper-roar-ann-oncol-2022-update"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT02034110","phase":"2","setting":"BRAF V600E-mutated rare cancers in nine histology cohorts, including previously treated unresectable, metastatic or recurrent biliary tract cancer (n=43): dabrafenib 150 mg twice daily plus trametinib 2 mg daily, single arm","sponsor":"Novartis Pharmaceuticals","result":"Biliary cohort ORR 51% (22/43, investigator) and 47% (independent review); grade 3 or worse GGT rise 12%.","yearReported":2020,"enrolled":206,"enrolledBasis":"registry","outcomes":[{"endpoint":"Overall response rate, biliary tract cancer cohort (investigator-assessed)","primary":true,"unit":"%","arms":[{"name":"Dabrafenib + trametinib","n":43,"value":51,"note":"95% CI 36 to 67"}],"source":"https://doi.org/10.1016/S1470-2045(20)30321-1"},{"endpoint":"Overall response rate, biliary tract cancer cohort (independent review)","unit":"%","arms":[{"name":"Dabrafenib + trametinib","n":43,"value":47,"note":"95% CI 31 to 62"}],"source":"https://doi.org/10.1016/S1470-2045(20)30321-1"}]},"route":"/trials/roar/","neighbours":{"cancer":[{"id":"biliary-tract-cancer","kind":"cancer","name":"Biliary tract cancer (all types)","route":"/cancers/biliary-tract-cancer/"},{"id":"cholangiocarcinoma","kind":"cancer","name":"Biliary tract cancer (cholangiocarcinoma)","route":"/cancers/cholangiocarcinoma/"},{"id":"gallbladder","kind":"cancer","name":"Gallbladder cancer","route":"/cancers/gallbladder/"}],"term":[{"id":"basket-umbrella-platform","kind":"term","name":"Basket, umbrella, and platform trials","route":"/terms/basket-umbrella-platform/"},{"id":"gallbladder-cancer-in-biliary-trials","kind":"term","name":"Gallbladder cancer in biliary tract cancer trials (eligibility and subgroups)","route":"/terms/gallbladder-cancer-in-biliary-trials/"},{"id":"rare-cancers","kind":"term","name":"Rare cancers","route":"/terms/rare-cancers/"},{"id":"tumour-agnostic","kind":"term","name":"Tumour-agnostic (tissue-agnostic) approval","route":"/terms/tumour-agnostic/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"braf","kind":"target","name":"BRAF","route":"/targets/braf/"}],"drug":[{"id":"dabrafenib","kind":"drug","name":"Dabrafenib","route":"/drugs/dabrafenib/"},{"id":"dabrafenib-trametinib","kind":"drug","name":"Dabrafenib + trametinib","route":"/drugs/dabrafenib-trametinib/"},{"id":"trametinib","kind":"drug","name":"Trametinib","route":"/drugs/trametinib/"}],"company":[{"id":"novartis","kind":"company","name":"Novartis","route":"/companies/novartis/"}],"paper":[{"id":"paper-roar-lancet-oncol-2020","kind":"paper","name":"Dabrafenib plus trametinib in patients with BRAF V600E -mutated biliary tract cancer (ROAR): a phase 2, open-label, single-arm, multicentre basket trial","route":"/key-papers/paper-roar-lancet-oncol-2020/"},{"id":"paper-roar-ann-oncol-2022-update","kind":"paper","name":"Dabrafenib plus trametinib in patients with BRAF V600E-mutant anaplastic thyroid cancer: updated analysis from the phase II ROAR basket study","route":"/key-papers/paper-roar-ann-oncol-2022-update/"}]}}