Molecular Profiling of Advanced Biliary Tract Cancers

Official Title

Comprehensive Molecular Profiling of Advanced Biliary Tract Cancers for Better Treatment Selection: a McGill University Health Centre Study (COMPASS-B-MUHC)

Summary:

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.

Trial Description

Primary Outcome:

• Number of participants with tumour whole genome sequencing returned within 8 weeks

• Disease control rate
• Progression-free survival rate
• Overall survival rate
• Number of patients in whom at least 1 actionable mutation is identified
• Number of patients who received a targeted therapy (after first-line treatment)

View this trial on ClinicalTrials.gov