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Molecular targeted maintenance therapy versus standard of care in advanced biliary cancer: an international, randomised, controlled, open-label, phase III umbrella trial (SAFIR-ABC10-Precision Medicine)
Malka, D. Borbath, I. Lopes, A. Couch, D. Jimenez, M. Vandamme, T. Valle, J. W. Wason, J. Ambrose, E. Dewever, L.
Malka, D.
Borbath, I.
Lopes, A.
Couch, D.
Jimenez, M.
Vandamme, T.
Valle, J. W.
Wason, J.
Ambrose, E.
Dewever, L.
Abstract
BACKGROUND: Advanced biliary tract cancers (ABCs) are a heterogeneous group of rare malignancies of the bile ducts and gall-bladder with a poor prognosis and limited treatment options. Cisplatin-gemcitabine (CISGEM) chemotherapy plus immunotherapy (durvalumab or pembrolizumab) is the current first-line standard of care (1L-SoC). ABCs frequently harbour actionable molecular alterations that suggest a high potential for benefit from molecular targeted therapies (MTTs). However, the assessment of potential first-line MTT treatments is hindered by the scarcity of ABCs harbouring a specific alteration and the time required to carry out tumour molecular profiling. MATERIALS AND METHODS: We detail here the design of SAFIR-ABC10, an international, randomised, phase III umbrella trial comparing the efficacy of sequential matched targeted therapy after four cycles (12 weeks) of 1L-SoC versus continued 1L-SoC in patients with ABC and an actionable molecular alteration [European Society for Medical Oncology (ESMO) Scale for Clinical Actionability of molecular Targets (ESCAT) tier I or II]. The primary study endpoint is progression-free survival. Besides initial tumour and circulating DNA next-generation sequencing analysis, sequential blood and tumour sampling will be carried out to identify biomarkers of prognosis, response and acquired resistance. PERSPECTIVES: SAFIR-ABC10 is, to our knowledge, the first randomised, umbrella trial assessing the concept of precision medicine in ABC, the ideal setting for addressing this question with a high rate of targetable alterations.
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Date
2025
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Citation
Malka D, Borbath I, Lopes A, Couch D, Jimenez M, Vandamme T, et al. Molecular targeted maintenance therapy versus standard of care in advanced biliary cancer: an international, randomised, controlled, open-label, phase III umbrella trial (SAFIR-ABC10-Precision Medicine). ESMO open. 2025 May;10(5):104540. PubMed PMID: 40209292. Pubmed Central PMCID: PMC12008684. Epub 2025/04/10. eng.