Biliary Tract CancerNCT04871321
Biomarker Discovery in Patients With Advanced Biliary Tract Cancer
An observational study of Biliary Tract Cancer and Cholangiocarcinoma, run by CHA University at 1 site in South Korea.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
- What is being tested?
- nab-paclitaxel plus gemcitabine-cisplatin
- How many people
- 119 participants
- Where
- 1 siteSouth Korea
- Study dates
- 14 Apr 2021to 31 Dec 2023
- How long will I take part?
- 12 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with advanced biliary tract cancer will be recruited at CHA Bundang Medical Center
Who may be able to join
- 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
Show all 9Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
South Korea1 site
CHA Bundang Medical Center
Seongnam-si, Gyeonggi-do, 13496
About this study
Official title: 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.
Read more
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.
Read the 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).
- Conditions
- Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
- Being tested
- nab-paclitaxel plus gemcitabine-cisplatin
- Keywords
- Biliary Tract cancer, Gemcitabine, Cisplatin, nab-paclitaxel, RNA sequencing, biomarker
- Medical subject headings
- Biliary Tract Neoplasms, Cholangiocarcinoma, Gallbladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nab-paclitaxel plus gemcitabine-cisplatinDrug
Also called immunohistochemistry, NGS, RNA-sequencing, Liquid biopsy
- gemcitabine 800mg/m2 + cisplatin 25 mg/m2 + nab-paclitaxel 100mg/m2 on day 1 and day 8, every 21 days
| Study group | Role | What they receive |
|---|---|---|
| Gem/Cis/nab-P | Gemcitabine + Cisplatin + Nab-Paciltaxel Receives: Drug: nab-paclitaxel plus gemcitabine-cisplatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
collected tumor samples
tumor samples from patients with hepatobiliary cancers (incidence of genetic alteration, association with treatment response and survival duration)
Measured: through study completion, an average of 2 years
collected blood samples
blood samples from patients with hepatobiliary cancers (incidence of genetic alteration, association with treatment response and survival duration)
Measured: through study completion, an average of 2 years
Secondary outcomes (2)
Multi-omics analysis
Multi-omics analysis to further subtype and find therapeutic targets of biliary tract cancer
Measured: 2 years
Biomarkers on the efficacy of Gemcitabine/Cisplatin/Nab-paclitaxel for advanced biliary tract cancer
Molecular biomarker associated with overall survival, progression-free survival and objective response rate in patients who receive Gemcitabine/Cisplatin/Nab-paclitaxel chemotherapy
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2021
- Study start14 Apr 2021
- Primary completion31 Dec 2022
- Study completion31 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Biliary Tract CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 119 participants (actual)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- CHA University
- Sponsor type
- University, hospital or other
- Collaborators
- Dong-A Pharmaceutical
- Responsible party
- Sponsor
- Investigators
- Hongjae Chon, MD,PhD, Principal investigator, CHA University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04871321
- Sponsor's protocol ID
- 2021-03-045
- Status last verified
- Aug 2025
- First submitted
- 26 Apr 2021
- Last updated
- 26 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Jarnagin WR, Fong Y, DeMatteo RP, Gonen M, Burke EC, Bodniewicz BS J, Youssef BA M, Klimstra D, Blumgart LH. Staging, resectability, and outcome in 225 patients with hilar cholangiocarcinoma. Ann Surg. 2001 Oct;234(4):507-17; discussion 517-9. doi: 10.1097/00000658-200110000-00010.
PubMed 11573044Valle J, Wasan H, Palmer DH, Cunningham D, Anthoney A, Maraveyas A, Madhusudan S, Iveson T, Hughes S, Pereira SP, Roughton M, Bridgewater J; ABC-02 Trial Investigators. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010 Apr 8;362(14):1273-81. doi: 10.1056/NEJMoa0908721.
PubMed 20375404Kim BJ, Hyung J, Yoo C, Kim KP, Park SJ, Lee SS, Park DH, Song TJ, Seo DW, Lee SK, Kim MH, Park JH, Cho H, Ryoo BY, Chang HM. Prognostic factors in patients with advanced biliary tract cancer treated with first-line gemcitabine plus cisplatin: retrospective analysis of 740 patients. Cancer Chemother Pharmacol. 2017 Jul;80(1):209-215. doi: 10.1007/s00280-017-3353-2. Epub 2017 Jun 8.
PubMed 28597043Shroff RT, Javle MM, Xiao L, Kaseb AO, Varadhachary GR, Wolff RA, Raghav KPS, Iwasaki M, Masci P, Ramanathan RK, Ahn DH, Bekaii-Saab TS, Borad MJ. Gemcitabine, Cisplatin, and nab-Paclitaxel for the Treatment of Advanced Biliary Tract Cancers: A Phase 2 Clinical Trial. JAMA Oncol. 2019 Jun 1;5(6):824-830. doi: 10.1001/jamaoncol.2019.0270.
PubMed 30998813Chae H, Kim D, Yoo C, Kim KP, Jeong JH, Chang HM, Lee SS, Park DH, Song TJ, Hwang S, Kim KH, Song GW, Ahn CS, Lee JH, Hwang DW, Kim SC, Jang SJ, Hong SM, Kim TW, Ryoo BY. Therapeutic relevance of targeted sequencing in management of patients with advanced biliary tract cancer: DNA damage repair gene mutations as a predictive biomarker. Eur J Cancer. 2019 Oct;120:31-39. doi: 10.1016/j.ejca.2019.07.022. Epub 2019 Aug 30.
PubMed 31476489Nakamura H, Arai Y, Totoki Y, Shirota T, Elzawahry A, Kato M, Hama N, Hosoda F, Urushidate T, Ohashi S, Hiraoka N, Ojima H, Shimada K, Okusaka T, Kosuge T, Miyagawa S, Shibata T. Genomic spectra of biliary tract cancer. Nat Genet. 2015 Sep;47(9):1003-10. doi: 10.1038/ng.3375. Epub 2015 Aug 10.
PubMed 26258846Chu KJ, Ma YS, Jiang XH, Wu TM, Wu ZJ, Li ZZ, Wang JH, Gao QX, Yi B, Shi Y, Wang HM, Gu LP, Zhang SQ, Wang GR, Liu JB, Fu D, Jiang XQ. Whole-Transcriptome Sequencing Identifies Key Differentially Expressed mRNAs, miRNAs, lncRNAs, and circRNAs Associated with CHOL. Mol Ther Nucleic Acids. 2020 Sep 4;21:592-603. doi: 10.1016/j.omtn.2020.06.025. Epub 2020 Jun 27.
PubMed 32721879Cheon J, Lee CK, Sang YB, Choi HJ, Kim MH, Ji JH, Ko KH, Kwon CI, Kim DJ, Choi SH, Kim C, Kang B, Chon HJ. Real-world efficacy and safety of nab-paclitaxel plus gemcitabine-cisplatin in patients with advanced biliary tract cancers: a multicenter retrospective analysis. Ther Adv Med Oncol. 2021 Aug 7;13:17588359211035983. doi: 10.1177/17588359211035983. eCollection 2021.
PubMed 34394748
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Discovery of Molecular Biomarkers for Predicting Response Through RNA Sequencing in Patients With Advanced Biliary Tract Cancer Who Received Nab-paclitaxel Plus Gemcitabine-cisplatin
- ClinicalTrials.gov ID
- NCT04871321
- Acronym / sponsor ID
- 2021-03-045
- Conditions
- Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
- Keywords
- Biliary Tract cancer, Gemcitabine, Cisplatin, nab-paclitaxel, RNA sequencing, biomarker
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- CHA UniversityUniversity, hospital or other
- Collaborators
- Dong-A Pharmaceutical
- Study start
- 14 Apr 2021 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 31 Dec 2023 (actual)
- First posted
- 4 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 12 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gem/Cis/nab-P | – | Drug: nab-paclitaxel plus gemcitabine-cisplatinGemcitabine + Cisplatin + Nab-Paciltaxel |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with advanced biliary tract cancer will be recruited at CHA Bundang Medical Center
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| nab-paclitaxel plus gemcitabine-cisplatinAlso: immunohistochemistry, NGS, RNA-sequencing, Liquid biopsy- gemcitabine 800mg/m2 + cisplatin 25 mg/m2 + nab-paclitaxel 100mg/m2 on day 1 and day 8, every 21 days | Drug | Gem/Cis/nab-P |
Primary outcome
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)
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)
2 secondary outcomes
Multi-omics analysis
Time frame: 2 years
Biomarkers on the efficacy of Gemcitabine/Cisplatin/Nab-paclitaxel for advanced biliary tract cancer
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2025.
- Background: PubMed 11573044Jarnagin WR, Fong Y, DeMatteo RP, Gonen M, Burke EC, Bodniewicz BS J, Youssef BA M, Klimstra D, Blumgart LH. Staging, resectability, and outcome in 225 patients with hilar cholangiocarcinoma. Ann Surg. 2001 Oct;234(4):507-17; discussion 517-9. doi: 10.1097/00000658-200110000-00010.
- Background: PubMed 20375404Valle J, Wasan H, Palmer DH, Cunningham D, Anthoney A, Maraveyas A, Madhusudan S, Iveson T, Hughes S, Pereira SP, Roughton M, Bridgewater J; ABC-02 Trial Investigators. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010 Apr 8;362(14):1273-81. doi: 10.1056/NEJMoa0908721.
- Background: PubMed 28597043Kim BJ, Hyung J, Yoo C, Kim KP, Park SJ, Lee SS, Park DH, Song TJ, Seo DW, Lee SK, Kim MH, Park JH, Cho H, Ryoo BY, Chang HM. Prognostic factors in patients with advanced biliary tract cancer treated with first-line gemcitabine plus cisplatin: retrospective analysis of 740 patients. Cancer Chemother Pharmacol. 2017 Jul;80(1):209-215. doi: 10.1007/s00280-017-3353-2. Epub 2017 Jun 8.
- Background: PubMed 30998813Shroff RT, Javle MM, Xiao L, Kaseb AO, Varadhachary GR, Wolff RA, Raghav KPS, Iwasaki M, Masci P, Ramanathan RK, Ahn DH, Bekaii-Saab TS, Borad MJ. Gemcitabine, Cisplatin, and nab-Paclitaxel for the Treatment of Advanced Biliary Tract Cancers: A Phase 2 Clinical Trial. JAMA Oncol. 2019 Jun 1;5(6):824-830. doi: 10.1001/jamaoncol.2019.0270.
- Background: PubMed 31476489Chae H, Kim D, Yoo C, Kim KP, Jeong JH, Chang HM, Lee SS, Park DH, Song TJ, Hwang S, Kim KH, Song GW, Ahn CS, Lee JH, Hwang DW, Kim SC, Jang SJ, Hong SM, Kim TW, Ryoo BY. Therapeutic relevance of targeted sequencing in management of patients with advanced biliary tract cancer: DNA damage repair gene mutations as a predictive biomarker. Eur J Cancer. 2019 Oct;120:31-39. doi: 10.1016/j.ejca.2019.07.022. Epub 2019 Aug 30.
- Background: PubMed 26258846Nakamura H, Arai Y, Totoki Y, Shirota T, Elzawahry A, Kato M, Hama N, Hosoda F, Urushidate T, Ohashi S, Hiraoka N, Ojima H, Shimada K, Okusaka T, Kosuge T, Miyagawa S, Shibata T. Genomic spectra of biliary tract cancer. Nat Genet. 2015 Sep;47(9):1003-10. doi: 10.1038/ng.3375. Epub 2015 Aug 10.
- Background: PubMed 32721879Chu KJ, Ma YS, Jiang XH, Wu TM, Wu ZJ, Li ZZ, Wang JH, Gao QX, Yi B, Shi Y, Wang HM, Gu LP, Zhang SQ, Wang GR, Liu JB, Fu D, Jiang XQ. Whole-Transcriptome Sequencing Identifies Key Differentially Expressed mRNAs, miRNAs, lncRNAs, and circRNAs Associated with CHOL. Mol Ther Nucleic Acids. 2020 Sep 4;21:592-603. doi: 10.1016/j.omtn.2020.06.025. Epub 2020 Jun 27.
- Background: PubMed 34394748Cheon J, Lee CK, Sang YB, Choi HJ, Kim MH, Ji JH, Ko KH, Kwon CI, Kim DJ, Choi SH, Kim C, Kang B, Chon HJ. Real-world efficacy and safety of nab-paclitaxel plus gemcitabine-cisplatin in patients with advanced biliary tract cancers: a multicenter retrospective analysis. Ther Adv Med Oncol. 2021 Aug 7;13:17588359211035983. doi: 10.1177/17588359211035983. eCollection 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hongjae Chon, MD,PhD principal investigator
CHA University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Biliary Tract Cancer and Cholangiocarcinoma, run by CHA University at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
collected tumor samples (time frame: through study completion, an average of 2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04871321, which also lists 8 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2025.