Molecular Profiling for Biliary Tract Cancer

(COMPASS-B-MUHC Trial)

Recruiting at 1 trial location

Overseen By George Zogopoulos, MD, PhD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre

Must be taking: Gemcitabine-based regimens

Disqualifiers: Brain metastases, Uncontrolled illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 4 years

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to better understand the genetic makeup of biliary tract cancer (BTC) to improve treatment options. BTC is a rare but deadly cancer, and most patients cannot undergo surgery to remove it. By analyzing the tumor's genes through tumor and germline molecular profiling, researchers hope to match patients with treatments targeting specific genetic changes. People with advanced BTC that cannot be surgically removed and who have not yet received chemotherapy might be suitable candidates for this trial. As an unphased trial, this study offers patients the opportunity to contribute to groundbreaking research that could lead to more personalized and effective treatments for BTC.

What prior data suggests that this molecular profiling is safe for patients with advanced biliary tract cancer?

Research has shown that molecular profiling in biliary tract cancer (BTC) is generally safe and well-tolerated. This process examines the tumor's genes to identify specific changes. It is important to note that profiling itself is not a treatment but a method to find targets for potential therapies.

While specific safety data for the profiling process is lacking, it resembles a biopsy, a common medical procedure. No major side effects have been reported from molecular profiling. This approach reveals the unique features of each patient's cancer, potentially leading to more effective and personalized treatment options.

Overall, molecular profiling is considered safe and plays a crucial role in customizing cancer treatments. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this approach to treating advanced biliary tract cancer because it leverages molecular profiling to personalize treatment. Unlike standard therapies like gemcitabine alone, this method involves analyzing the genetic makeup of both the tumor and the patient to tailor the treatment plan. This could lead to more effective and targeted therapy options, potentially improving outcomes by matching patients with drugs that are more likely to work for their specific cancer profile. This precision medicine approach represents a significant shift from one-size-fits-all treatments, offering hope for better management of this challenging condition.

What evidence suggests that this molecular profiling is effective for biliary tract cancer?

Research has shown that understanding the unique characteristics of biliary tract cancer can help doctors find better treatment options. In this trial, participants will undergo tumor and germline molecular profiling to identify specific traits of their cancer. This information will guide the selection of treatments, such as targeted therapies or immunotherapy, which may be more effective for each patient. Some studies suggest that this personalized approach could lead to better results than standard chemotherapy, which often proves ineffective for this type of cancer. While more research is needed, early findings offer hope for improving survival rates and treatment responses. ^{2 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults with advanced biliary tract cancer that can't be removed by surgery. They must have a measurable tumor, understand and sign consent, have an ECOG status ≤1 (which means they are able to walk and capable of all self-care), expect to live more than 90 days, and have normal organ/marrow function. They should've had gemcitabine-based treatment first without success.

Inclusion Criteria

I am receiving or will receive gemcitabine as my first treatment.

Ability to understand and willing to sign a written informed consent document

I have a tumor that can be measured and another that can be biopsied.

Exclusion Criteria

Any other condition that would contraindicate the patient's participation due to safety concerns or compliance with clinical study procedures

I have cancer that has spread to my brain.

I have not received chemotherapy or other cancer treatments in the advanced stage.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Molecular Profiling

Tumour biopsy is performed for whole genome and transcriptome sequencing to identify actionable molecular alterations

8 weeks

Treatment

Chemo-naive patients receive first-line gemcitabine-based chemotherapy or an investigational drug

4 years

Follow-up

Participants are monitored for disease control, progression-free survival, and overall survival

4 years

What Are the Treatments Tested in This Trial?

Interventions

• Tumour and germline molecular profiling

Trial Overview The study tests the process of molecular profiling on tumors from patients with advanced biliary tract cancer. It aims to return whole genome sequencing results within 8 weeks after biopsy for second-line treatment consideration. The goal is to find specific molecular changes in the cancer that could lead to more effective treatments.

How Is the Trial Designed?

Group I: Individuals with advanced biliary tract cancer Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

McGill University Health Centre/Research Institute of the McGill University Health Centre

Lead Sponsor

Trials

476

Recruited

170,000+

Cancer Research Society

Collaborator

Trials

8

Recruited

5,400+

Cancer Research Society

Collaborator

Trials

6

Recruited

650+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6657312/

Molecular profiling of biliary cancers reveals distinct ... Recent studies have demonstrated potential novel biliary cancer targets and a possible role for immunotherapy in the treatment of patients with this disease.

2. sciencedirect.com sciencedirect.com/science/article/pii/S0305737223001421

Molecular profiling in cholangiocarcinoma: A practical ... This review aims to provide clinical pathologists and other healthcare professionals with practical step-by-step guidance on the use of NGS for molecular ...

3. link.springer.com link.springer.com/article/10.1007/s12254-025-01050-7

Molecular profiling and targeted therapies in biliary tract ... The median overall survival in advanced stages remains below 13 months despite chemotherapy and recent immunotherapy advances. However, ...

4. nature.com nature.com/articles/s41598-023-42083-4

Scientific Reports - Nature In total, the molecular profile of 159 patients with biliary tract cancer was available. For further analysis, 19 patients were excluded due to ...

5. esmoopen.com esmoopen.com/article/S2059-7029(24)01476-5/fulltext

Evolving therapeutic landscape of advanced biliary tract ... We highlight the importance of molecular profiling in guiding therapeutic decisions and emphasize the need for continued research to optimize ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5056250/

Molecular profiling of biliary tract cancer: a target rich disease Biliary tract cancers (BTCs) are relatively uncommon orphan tumors that have an aggressive disease course and a poor clinical outcome.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04318834

Molecular Profiling of Advanced Biliary Tract Cancers This study aims to investigate the feasibility and clinical utility of prospective molecular profiling of advanced biliary tract cancer. The primary endpoint of ...

8. academic.oup.com academic.oup.com/jnci/article/115/7/870/7114547

Molecular profiling and treatment pattern differences between ... This study examines differences in molecular profiling rates and treatment patterns in these populations, focusing on use of adjuvant, liver- ...