Biomarker Discovery in Patients With Advanced Biliary Tract Cancer

August 25, 2025 updated by: CHA University

Discovery of Molecular Biomarkers for Predicting Response Through RNA Sequencing in Patients With Advanced Biliary Tract Cancer Who Received Nab-paclitaxel Plus Gemcitabine-cisplatin

Biliary tract cancer is a rare gastrointestinal malignant neoplasm and includes intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, and gall bladder cancer. Curative surgical resection offers the only chance for cure. However, most patients with BTC are diagnosed at an unresectable stage. Therefore, the survival outcomes of patients with advanced biliary tract cancer remain dismal.

The combination of gemcitabine and cisplatin has become the current standard for advanced BTCs since the landmark ABC-02 trial in 2010. However, the median overall survival of Gem/Cis chemotherapy is less than 1 year. Recently, a triplet regimen of gemcitabine, cisplatin, and nab-paclitaxel showed promising results in a single-arm phase II multicenter study.

However, biliary tract cancer is a group of heterogenous diseases by site and genetic alteration, and this diversity may lead differences in response to systemic chemotherapy.

Transcriptome analysis through RNA-sequencing has rarely been performed in advanced biliary tract cancer, and even if it has performed, only small number of patients were included. Further research on multi-omics data is needed on the necessity and clinical significance in treatment of biliary tract cancer.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Using biopsy specimen (formalin fixed paraffin embedded tissue), in-house NGS and RNA-sequencing will be performed simultaneously. Through this, investigators will discover biomarkers based on multi-omics data that predict response to systemic chemotherapy (nab-paclitaxel plus gemcitabine-cisplatin). In addition, blood sampling will be performed in parallel to conduct research on cell-free DNA and circulating tumor cell analysis related to response and progression on chemotherapy; before administration of chemotherapy, 3 months after chemotherapy, 6 months after chemotherapy, the time of disease progression (if possible), before curative resection (if possible).

Study Type

Observational

Enrollment (Actual)

119

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • South Korea
    • Gyeonggi-do
      • Seongnam-si, Gyeonggi-do, South Korea, 13496
        • CHA Bundang Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

19 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with advanced biliary tract cancer will be recruited at CHA Bundang Medical Center

Description

Inclusion Criteria:

  • Age ≥ 19 years old
  • Capable of understanding and complying with the protocol requirements and signed informed consent
  • The patients were confirmed as bilary tract cancer (gall bladder cancer, extrahepatic cholangiocarcinoma, intrahepatic cholangiocarcinoma) by histopathology or cytology
  • Patients with inoperable or metastatic or recurrent biliary tract cancer
  • Patients who underwent in-house next-generation sequencing
  • Patients planning to receive Gemcitabine, Cisplatin, nab-paclitaxel triplet chemotherapy
  • At least one measurable objective lesion was identified based on the RECIST 1.1 criteria
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
  • The expected survival a ≥3 months

Exclusion Criteria:

  • The subject has uncontrolled, significant intercurrent or recent illness including infection for organ failure
  • Prior palliative chemotherapy for biliary tract cancer
  • Dementia, altered mental state, or any mental illness that prevents understanding or informed consent
  • Other conditions that researchers not think to be suitable for enrollment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Gem/Cis/nab-P
Gemcitabine + Cisplatin + Nab-Paciltaxel
Drug: nab-paclitaxel plus gemcitabine-cisplatin
- gemcitabine 800mg/m2 + cisplatin 25 mg/m2 + nab-paclitaxel 100mg/m2 on day 1 and day 8, every 21 days
Other Names:
  • immunohistochemistry, NGS, RNA-sequencing, Liquid biopsy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
collected tumor samples
Time Frame: through study completion, an average of 2 years
tumor samples from patients with hepatobiliary cancers (incidence of genetic alteration, association with treatment response and survival duration)
through study completion, an average of 2 years
collected blood samples
Time Frame: through study completion, an average of 2 years
blood samples from patients with hepatobiliary cancers (incidence of genetic alteration, association with treatment response and survival duration)
through study completion, an average of 2 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Multi-omics analysis
Time Frame: 2 years
Multi-omics analysis to further subtype and find therapeutic targets of biliary tract cancer
2 years
Biomarkers on the efficacy of Gemcitabine/Cisplatin/Nab-paclitaxel for advanced biliary tract cancer
Time Frame: 2 years
Molecular biomarker associated with overall survival, progression-free survival and objective response rate in patients who receive Gemcitabine/Cisplatin/Nab-paclitaxel chemotherapy
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CHA University

Collaborators

Dong-A Pharmaceutical

Investigators

  • Principal Investigator: Hongjae Chon, MD,PhD, CHA University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 14, 2021

Primary Completion (Actual)

December 31, 2022

Study Completion (Actual)

December 31, 2023

Study Registration Dates

First Submitted

April 26, 2021

First Submitted That Met QC Criteria

April 29, 2021

First Posted (Actual)

May 4, 2021

Study Record Updates

Last Update Posted (Estimated)

August 26, 2025

Last Update Submitted That Met QC Criteria

August 25, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2021-03-045

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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