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BackgroundNext-generation sequencing has identified actionable genetic aberrations in intrahepatic cholangiocarcinomas (iCCA), including the fibroblast growth factor receptor 2 (FGFR2) fusions. Derazantinib (ARQ 087), an orally bioavailable, multi-kinase inhibitor with potent pan-FGFR activity, has shown preliminary therapeutic activity against FGFR2 fusion-positive iCCA.MethodsThis multicentre, phase 1/2, open-label study enrolled adult patients with unresectable iCCA with FGFR2 fusion, who progressed, were intolerant or not eligible to first-line chemotherapy (NCT01752920). Subjects received derazantinib in continuous daily doses. Tumour response was assessed according to RECIST 1.1 every 8 weeks.ResultsTwenty-nine patients (18 women/11 men; median age, 58.7 years), 2 treatment-naive and 27 who progressed after at least one prior systemic therapy, were enrolled. Overall response rate was 20.7%, disease control rate was 82.8%. Estimated median progression-free survival was 5.7 months (95% CI: 4.04–9.2 months). Treatment-related adverse events (AE) were observed in 27 patients (93.1%, all grades), including asthenia/fatigue (69.0%), eye toxicity (41.4%), and hyperphosphatemia (75.9%). Grade ≥ 3 AEs occurred in 8 patients (27.6%).ConclusionDerazantinib demonstrated encouraging anti-tumour activity and a manageable safety profile in patients with advanced, unresectable iCCA with FGFR2 fusion who progressed after chemotherapy. A pivotal trial of derazantinib in iCCA is ongoing (NCT03230318).

Although clinical trial research is required for the development of improved treatment strategies, very few cancer patients participate in these studies. The purpose of this study was to describe psychosocial barriers to clinical trial participation among oncologists and their cancer patients. A survey was distributed to all medical oncologists in Pennsylvania and a subset of their patients. Relevant background information and assessment of practical and psychosocial barriers to clinical trial participation were assessed. Among 137 oncologists and 170 patients who completed the surveys, 84% of patients were aware of clinical trials, and oncologists and patients generally agreed that clinical trials are important to improving cancer treatment. However, oncologists and patients were more likely to consider clinical trials in advanced or refractory disease. When considering 7 potential barriers to clinical trials, random assignment and fear of receiving a placebo were ranked highly by both patients and oncologists. Patients identified fear of side effects as the greatest barrier to clinical trial participation, whereas oncologists ranked this psychosocial barrier as least important to their patients. Overall, the study found that although oncologists and patients are aware of clinical trials and have favorable attitudes toward them, psychosocial barriers exist for patients that may impact participation in clinical trials. Furthermore, important discrepancies exist between the perceptions of oncologists and those of patients regarding what the psychosocial barriers are. We concluded that characterizing oncologist and patient perceived barriers can help improve communication and decision making about clinical trials, such that participation may be optimized.