{"database":"biostudies-literature","file_versions":[],"scores":{"citationCount":0,"reanalysisCount":0,"viewCount":53,"searchCount":0},"additional":{"omics_type":["Unknown"],"volume":["5(6)"],"submitter":["Bono P"],"pubmed_abstract":["<b>Background: </b>Genetic alterations in fibroblast growth factor receptor (FGFR) and vascular endothelial growth factor receptor (VEGFR) signalling are observed in various tumours. We report a first-in-human phase I/IIa trial evaluating tolerability, pharmacokinetics and preliminary antitumour activity of ODM-203, a novel FGFR and VEGFR inhibitor.<br><br><b>Methods: </b>Open-label, non-randomised, multicentre, phase I/IIa dose escalation and expansion study in patients with advanced or metastatic solid tumours.<br><br><b>Results: </b>Overall, 84 patients received treatment; optimal tablet dose was found to be 400 mg/day with food. All patients experienced at least one adverse event; the majority (89.2%) were grade 1 or 2% and 70.4% were considered treatment related. The most commonly reported events were bilirubin increase-related events (75%) and diarrhoea (50%).Overall response rate was 9.2% and median progression-free survival was 16.1 and 12.4 weeks for patients with aberrant or non-aberrant FGFR tumours. Median time on treatment was 10.1 weeks for all patients and 14.5 weeks for patients who received 400 mg tablets.<br><br><b>Conclusion: </b>This study suggests ODM-203 400 mg/day results in sufficient plasma concentrations and acceptable tolerability in most patients. Preliminary signs of therapeutic activity of ODM-203 in patients with solid tumours was observed.<br><br><b>Trial registration number: </b>NCT02264418."],"journal":["ESMO open"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7709506"],"repository":["biostudies-literature"],"pubmed_title":["Phase I/IIa, open-label, multicentre study to evaluate the optimal dosing and safety of ODM-203 in patients with advanced or metastatic solid tumours."],"pmcid":["PMC7709506"],"pubmed_authors":["Kristeleit RS","Garratt C","Lassen U","Arkenau HT","Ikonen T","Mustonen MVJ","Italiano A","Bono P","Hakulinen P","Peltola KJ","Curigliano G","Azaro A","Rodon JA","Massard C"],"view_count":["53"],"additional_accession":[]},"is_claimable":false,"name":"Phase I/IIa, open-label, multicentre study to evaluate the optimal dosing and safety of ODM-203 in patients with advanced or metastatic solid tumours.","description":"<b>Background: </b>Genetic alterations in fibroblast growth factor receptor (FGFR) and vascular endothelial growth factor receptor (VEGFR) signalling are observed in various tumours. We report a first-in-human phase I/IIa trial evaluating tolerability, pharmacokinetics and preliminary antitumour activity of ODM-203, a novel FGFR and VEGFR inhibitor.<br><br><b>Methods: </b>Open-label, non-randomised, multicentre, phase I/IIa dose escalation and expansion study in patients with advanced or metastatic solid tumours.<br><br><b>Results: </b>Overall, 84 patients received treatment; optimal tablet dose was found to be 400 mg/day with food. All patients experienced at least one adverse event; the majority (89.2%) were grade 1 or 2% and 70.4% were considered treatment related. The most commonly reported events were bilirubin increase-related events (75%) and diarrhoea (50%).Overall response rate was 9.2% and median progression-free survival was 16.1 and 12.4 weeks for patients with aberrant or non-aberrant FGFR tumours. Median time on treatment was 10.1 weeks for all patients and 14.5 weeks for patients who received 400 mg tablets.<br><br><b>Conclusion: </b>This study suggests ODM-203 400 mg/day results in sufficient plasma concentrations and acceptable tolerability in most patients. Preliminary signs of therapeutic activity of ODM-203 in patients with solid tumours was observed.<br><br><b>Trial registration number: </b>NCT02264418.","dates":{"release":"2020-01-01T00:00:00Z","publication":"2020 Dec","modification":"2021-02-20T09:58:22Z","creation":"2021-02-20T09:58:22Z"},"accession":"S-EPMC7709506","cross_references":{"pubmed":["33262202"],"doi":["10.1136/esmoopen-2020-001081"]}}