Molecular Profiling of Advanced Biliary Tract Cancers

NCT04318834 | Recruiting

• 1 other identifier: 2020-6112
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Biliary Tract Cancer

Keywords

Biliary tract cancer; Cholangiocarcinoma; Molecular Profiling; Whole Genome Sequencing; Whole Transcriptome Sequencing

Outcome Measures

Primary Outcomes (1)

• Number 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.

  2 years

Secondary Outcomes (5)

• Disease control rate

  4 years

• Progression-free survival rate

  4 years

• Overall survival rate

  4 years

• Number of patients in whom at least 1 actionable mutation is identified

  4 years

• Number of patients who received a targeted therapy (after first-line treatment)

  4 years

Study Arms (1)

Individuals with advanced biliary tract cancer

EXPERIMENTAL

Following a tumour biopsy for molecular profiling, chemo-naive patients with advanced biliary tract cancer will receive first-line gemcitabine-based chemotherapy or an investigational drug on a participating clinical trial.

Other: Tumour and germline molecular profiling

Interventions

Tumour and germline molecular profiling OTHER

Tumour whole genome sequencing, germline whole genome sequencing, tumour whole transcriptome sequencing

Individuals with advanced biliary tract cancer

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• McGill University Health Centre/Research Institute of the McGill University Health Centre: lead
• Cancer Research Society: collaborator

Study Sites (1)

McGill University Health Centre

Montreal, Quebec, H4A 3J1, Canada

RECRUITING

MeSH Terms

Conditions

Biliary Tract Neoplasms; Cholangiocarcinoma

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Biliary Tract Diseases; Digestive System Diseases; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type

Central Study Contacts

George Zogopoulos, MD, PhD

CONTACT

514-934-1934; george.zogopoulos@mcgill.ca

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 24, 2020
• First Posted: March 24, 2020
• Study Start: April 1, 2020
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: April 17, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)