Dabrafenib plus trametinib in BRAFV600E-mutated rare cancers: the phase 2 ROAR trial
The University of Texas MD Anderson Cancer Center · National Institutes of Health · +29 more institutions
Abstract
BRAFV600E alterations are prevalent across multiple tumors. Here we present final efficacy and safety results of a phase 2 basket trial of dabrafenib (BRAF kinase inhibitor) plus trametinib (MEK inhibitor) in eight cohorts of patients with BRAFV600E-mutated advanced rare cancers: anaplastic thyroid carcinoma (n = 36), biliary tract cancer (n = 43), gastrointestinal stromal tumor (n = 1), adenocarcinoma of the small intestine (n = 3), low-grade glioma (n = 13), high-grade glioma (n = 45), hairy cell leukemia (n = 55) and multiple myeloma (n = 19). The primary endpoint of investigator-assessed overall response rate in these cohorts was 56%, 53%, 0%, 67%, 54%, 33%, 89% and 50%, respectively. Secondary endpoints…
Citation impact
- FWCI
- 41.87
- Percentile
- 100%
- References
- 59
Authors
23Topics & keywords
- Trametinib
- Dabrafenib
- Medicine
- Cancer research
- Oncology
- Internal medicine
- Biology
- Genetics
- Good health and well-being
Funding
- AAmgen
- BSBristol-Myers Squibb
- ELEli Lilly and CompanyAward: P30 CA016672
- PPfizer
- AAstraZenecaAward: CA016672
- GGlaxoSmithKline
- SSanofi
- CPCancer Prevention and Research Institute of TexasAwards: 1U01 CA180964, RP1100584, P30 CA016672, CA016672, UL1 TR000371
- GSGilead Sciences
- ASAmerican Society of Clinical Oncology
- CCelgene
- FHF. Hoffmann-La RocheAward: P30 CA016672
- ABArray BioPharma
- NPNovartis Pharmaceuticals Corporation
- TPTaiho Pharmaceutical
- ASAndrew Sabin Family Foundation
- LLLes Laboratories Pierre Fabre
- HHalozyme
- BBeiGene
- IIncyteAward: CA016672
- DDaiichi-Sankyo
- EEisai
- KHKyowa Hakko Kirin
- APAstellas Pharma
- ESEuropean Society for Medical Oncology
- SESociedad Española de Oncología Médica
- SServier
- NFNovartis Farmacéutica
- IIpsen
- PPlexxikon
- NINational Institutes of HealthAwards: CA016672, 1U01 CA180964, UL1 TR000371, P30 CA016672, R01CA242845, R01CA273168, TR000371, RP1100584
- GGenentech
- UOUniversity of Texas MD Anderson Cancer CenterAwards: 1U01 CA180964, UL1 TR000371, RP1100584, CA016672, P30 CA016672
- CPChugai Pharmaceutical
- CFCenter for Clinical and Translational Sciences, University of Texas Health Science Center at HoustonAwards: UL1 TR000371, 1U01 CA180964, RP1100584, TR000371, P30 CA016672
- NCNational Cancer InstituteAward: P30CA016672
- SKSheikh Khalifa Bin Zayed Al Nahyan Institute for Personalized Cancer TherapyAwards: UL1 TR000371, 1U01 CA180964, P30 CA016672, RP1100584
- CCilag