Biliary tract cancer (BTC) accounts for \<1% of all cancers, but remains a highly fatal malignancy. Surgical resection is the only hope for cure, but most patients present with advanced disease when curative-intent surgery is not possible. The therapeutic options for patients with advanced disease are limited, primarily to chemotherapeutic regimens, which are based on empiric evidence without the use of biomarkers. These current treatment strategies have been largely ineffective in controlling the disease, resulting in poor survival outcomes of less than 1 year. An understanding of the molecular characteristics of biliary tract cancer may enable stratification of patients into therapies that target specific molecular alterations with greater efficacies and improved clinical outcomes. This study aims to investigate the feasibility and clinical utility of prospective molecular profiling of advanced biliary tract cancer. The primary endpoint of this study is to demonstrate the feasibility of returning whole genome sequencing results within 8 weeks of tumour biopsy for second-line treatment consideration (n=30 patients). In parallel, tumour whole transcriptome sequencing will be performed to identify actionable molecular alterations (e.g., fusion transcripts). Once the primary endpoint is met, the study will be expanded. Current funding allows expansion to 40 patients in total.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
40
Tumour whole genome sequencing, germline whole genome sequencing, tumour whole transcriptome sequencing
McGill University Health Centre
Montreal, Quebec, Canada
RECRUITINGNumber of participants with tumour whole genome sequencing returned within 8 weeks
We have estimated that 30 patients will be required to reach the primary end point, which will be met if we demonstrate that tumor whole genome sequencing data is available at 8 weeks from the tumour biopsy for 80% of patients.
Time frame: 2 years
Disease control rate
Number of patients that achieve stability of disease (cancer) by imaging (RECIST criteria) with first-line standard chemotherapy, consisting of gemcitabine backbone.
Time frame: 4 years
Progression-free survival rate
Progression free survival (PFS) defined as the interval between the date of registration and the earliest date of disease progression or death due to any cause of patients treated with 1st line chemotherapy.
Time frame: 4 years
Overall survival rate
Overall survival (OS) defined as the interval between the date of registration and the date of death of patients treated with 1st line chemotherapy.
Time frame: 4 years
Number of patients in whom at least 1 actionable mutation is identified
based on whole genome sequencing or RNA sequencing analyses.
Time frame: 4 years
Number of patients who received a targeted therapy (after first-line treatment)
based on the identification of an actionable mutation by whole genome sequencing or RNA sequencing.
Time frame: 4 years
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