Trial in progress: A feasibility study of multi-platform profiling using biospecimens from patients with resected biliary tract cancer.

Abstract

TPS359 Background: Biliary tract cancers (BTC) are rare malignancies with a high recurrence rate after resection of localized disease. FDA-approved systemic therapies for advanced disease are limited to combination chemotherapy and FGFR inhibitors for FGFR2-altered BTC. The tools of personalized medicine may guide management and expand treatment options for this disease. In multiple tumor types, circulating tumor DNA (ctDNA) has shown significant predictive power for recurrence after resection, but its utility has not yet been established in BTC. Organoid drug sensitivity screening may also inform and expand treatment options, particularly for patients without targetable mutations on next generation sequencing (NGS). Prospective concordance between organoid drug sensitivity and in vivo clinical responses to a given drug is needed to establish the value of this testing in BTC. We designed a clinical trial to determine the feasibility and physician-assessed utility of multi-platform profiling of resected BTC. Methods: This is an observational, non-randomized feasibility study to assess the ability to obtain NGS, ctDNA monitoring, and organoid drug sensitivity screening on patients with resected localized BTC. Secondary endpoints include physician-adjudicated utility of the profiling results for patient management, prospective concordance of organoid drug sensitivity with in vivo drug activity, and value of ctDNA in prediction of recurrence or response to therapy. Key eligibility criteria include a biopsy or clinical presentation consistent with BTC (excluding mixed HCC-cholangiocarcinoma), surgical candidacy for resection of measurable disease, and no prior targeted or investigational therapies. ctDNA is collected pre- and post-operatively, during adjuvant chemotherapy, during surveillance for up to 5 years and during systemic therapy for recurrent disease. Physician surveys are conducted before and after receipt of profiling results and then at time points where a change in management would be considered. Enrollment began in July 2020 with 5 of a planned 20 patients enrolled at the time of submission. Clinical trial information: NCT04561453.