Clinical trial NCT04318834: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Biliary Tract Cancer
Keywords: Biliary tract cancer
Cholangiocarcinoma
Molecular Profiling
Whole Genome Sequencing
Whole Transcriptome Sequencing
Lead sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre
Sponsor class: OTHER
Collaborators: name: Cancer Research Society
First posted: 2020-03-24
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief 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.


Eligibility criteria: Inclusion Criteria:

* Patients must have a histological or radiological diagnosis of inoperable or metastatic BTC.
* Patient must have a tumour that is amenable to a core needle biopsy.
* Patients must have a measurable lesion by RECIST 1.1 in addition to the lesion that is going to be biopsied.
* Patients must be fit to safely undergo a tumour biopsy as judged by the investigator.
* Eastern Cooperative Group (ECOG) performance status ≤ 1.
* Life expectancy of greater than 90 days.
* Within 14 days of the proposed biopsy date, patients must have normal organ and marrow function.
* Patients must undergo systemic treatment with gemcitabine-based regimens as first-line standard systemic palliative treatment with or without other investigational agents within a clinical trial.
* Ability to understand and willing to sign a written informed consent document.

Exclusion Criteria:

* Patients with one or more contraindications to tumour biopsy.
* Patients who have had any prior chemotherapy or other anti-cancer agent in the advanced stage setting.
* Patients who are currently on anti-cancer treatment.
* Patients with known brain metastases.
* Uncontrolled concurrent illness that would limit compliance with study requirements.
* Any other condition that would contraindicate the patient's participation due to safety concerns or compliance with clinical study procedures.

Primary outcomes:
measure: Number of participants with tumour whole genome sequencing returned within 8 weeks
timeFrame: 2 years
Secondary outcomes:
measure: Disease control rate
timeFrame: 4 years
measure: Progression-free survival rate
timeFrame: 4 years
measure: Overall survival rate
timeFrame: 4 years
measure: Number of patients in whom at least 1 actionable mutation is identified
timeFrame: 4 years
measure: Number of patients who received a targeted therapy (after first-line treatment)
timeFrame: 4 years
Other outcomes:
Locations:
city: Montréal
country: Canada
facility: McGill University Health Centre
state: Quebec
Study officials:
References: