CholangiocarcinomaNCT07291063
Anti-PD-1/PD-L1 Antibodies Plus Gemcitabine and Cisplatin for Advanced CCA
An observational study of Cholangiocarcinoma, run by Shanghai Zhongshan Hospital at 1 site in China.
Trial summary
This study is a prospective, observational study designed to analyze the safety, tolerability, and efficacy of first-line treatment using the combination of gemcitabine and cisplatin plus anti-PD-1/PD-L1 antibodies for patients with advanced cholangiocarcinoma. Read more about this study
- Who is it for?
- Ages 18 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Cholangiocarcinoma
- What is being tested?
- Anti-PD-1/PD-L1 antibody and Gemcitabine and Cisplatin Chemotherapy
- Type of treatment
- Injection or infusion
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 19 Jul 2024to 31 Dec 2029
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 to 90 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Patients with locally advanced or metastatic biliary tract cancer diagnosed histologically or cytologically.
Who may be able to join
- 1. ≥18 years old, male or female 2. Histologically or cytologically confirmed locally advanced or metastatic biliary tract cancer (including ICC, ECC, or GBC) that is not amenable to curative surgery or local therapies.
- 3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1 4. Patient has given written informed consent. 5. The function of important organs meets the requirements 6. Expected survival ≥12 weeks 7. Non-surgical sterilization or women of childbearing age need to use a medically-accepted contraceptive (such as an intrauterine device, contraceptive or condom) during the study period and within 3 months after the end of the study treatment period.
Who cannot join
- 1. The patient has any active autoimmune disease or a history of autoimmune disease (such as the following, but not limited to: autoimmune hepatitis, interstitial pneumonia, uveitis, enteritis, hepatitis, pituitary inflammation, vasculitis, nephritis, thyroid Hyperfunction; patients with vitiligo; complete remission of asthma in childhood, can be included without any intervention after adulthood; asthma patients who require bronchodilators for medical intervention cannot be included); 2. The patient is using immunosuppressive agents or systemic hormonal therapy to achieve immunosuppressive purposes (agents amount > 10 mg / day of prednisone or other therapeutic hormones), and continue to use within 2 weeks before enrollment; 3. Have clinical symptoms or disease that are not well controlled; 4. Significant clinically significant bleeding symptoms or a clear bleeding tendency within 3 months prior to randomization; 5. Arterial/venous thrombosis in the first 6 months of randomization 6. According to the investigator, the patient has other factors that may affect the results of the study or lead to the termination of the study, such as alcohol abuse, drug abuse, other serious diseases (including mental illness) requiring combined treatment, and serious laboratory abnormalities.#with family or social factors, it will affect the safety of patients. 7. Known hypersensitivity to Gemcitabine, Cisplatin, or platinum-containing compounds; significant hearing impairment (grade ≥2) or peripheral neuropathy (grade ≥2) that contraindicates Cisplatin;
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Zhongshan Hospital Fudan university
Shanghai, Shanghai Municipality, 200032
Hui Li, PhD, 13075929630, vincentxwx34@163.com
24111210117@m.fudan.edu.cn
About this study
Official title: A Phase I/II Study of Anti-PD-1/PD-L1 Antibodies in Combination With Gemcitabine and Cisplatin for Patients With Advanced Cholangiocarcinoma
This study is a prospective, observational study designed to analyze the safety, tolerability, and efficacy of first-line treatment using the combination of gemcitabine and cisplatin plus anti-PD-1/PD-L1 antibodies for patients with advanced cholangiocarcinoma.
Read the detailed description
This is a prospective cohort study of combination anti-PD-1/PD-L1 antibodies plus Gemcitabine and Cisplatin chemotherapy for adult patients (≥18) with advanced cholangiocarcinoma
Gemcitabine and Cisplatin (GC): This chemotherapy doublet has been the historical standard of care for advanced cholangiocarcinoma. It works by interfering with DNA synthesis and causing DNA cross-linking, leading to tumor cell apoptosis.
Anti-PD-1/PD-L1 antibodies: Immunotherapy (including pembrolizumab, durvalumab, envafolimab, tislelizumab, etc.) is designed to block the PD-1/PD-L1 immune checkpoint pathway, thereby reinvigorating T-cells to recognize and attack tumor cells. Recent pivotal trials (e.g., TOPAZ-1, KEYNOTE-966) have demonstrated that adding immunotherapy to GC chemotherapy significantly improves overall survival compared to chemotherapy alone.
This study aims to evaluate the real-world safety, tolerability, and clinical efficacy of this combination regimen in specific clinical practice settings for unresectable late-stage cholangiocarcinoma patients.
- Conditions
- Cholangiocarcinoma
- Being tested
- Anti-PD-1/PD-L1 antibody, Gemcitabine and Cisplatin Chemotherapy
- Keywords
- Cholangiocarcinoma, Gemcitabine, Cisplatin, Anti-PD-1/PD-L1 antibody
- Medical subject headings
- Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-PD-1/PD-L1 antibodyDrug
Anti-PD-1/PD-L1 Intravenous injection for at least 6 months
Gemcitabine and Cisplatin ChemotherapyDrug
Gemcitabine and cisplatin Intravenous injection for at least 6 months
| Study group | Role | What they receive |
|---|---|---|
| Chemotherapy plus Immunotherapy Combination | Not described Receives: Drug: Anti-PD-1/PD-L1 antibody; Drug: Gemcitabine and Cisplatin Chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Safety will be monitored by addressing and recording all adverse events (AEs), serious adverse events (SAEs) and specific laboratory abnormalities (worst grade). Toxicities will be graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0.
Measured: Up to 30 days after last treatment dose
Objective response rate(ORR)
Evaluated by researchers based on the RECIST 1.1 standard
Measured: 5 years
Progression free survival(PFS)
Evaluated by researchers based on the RECIST 1.1 standard
Measured: 5 years
To the relief time (TOR)
Evaluated by researchers based on the RECIST 1.1 standard
Measured: Time Frame: 5 years
Duration of relief(DOR)
Evaluated by researchers based on the RECIST 1.1 standard
Measured: 5 years
Disease Control Rate (DCR)
Evaluated by researchers based on the RECIST 1.1 standard
Measured: Time Frame: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2025
- Study start19 Jul 2024
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for CholangiocarcinomaStudies of the same condition, open and under way.
- More trials run by Shanghai Zhongshan HospitalThe sponsor's other registered studies.
- 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
- 50 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Shanghai Zhongshan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07291063
- Sponsor's protocol ID
- Y2024-752
- Status last verified
- Dec 2025
- First submitted
- 2 Dec 2025
- Last updated
- 18 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Oh DY, Ruth He A, Qin S, Chen LT, Okusaka T, Vogel A, Kim JW, Suksombooncharoen T, Ah Lee M, Kitano M, Burris H, Bouattour M, Tanasanvimon S, McNamara MG, Zaucha R, Avallone A, Tan B, Cundom J, Lee CK, Takahashi H, Ikeda M, Chen JS, Wang J, Makowsky M, Rokutanda N, He P, Kurland JF, Cohen G, Valle JW. Durvalumab plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer. NEJM Evid. 2022 Aug;1(8):EVIDoa2200015. doi: 10.1056/EVIDoa2200015. Epub 2022 Jun 1.
PubMed 38319896Valle 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 20375404Kelley RK, Ueno M, Yoo C, Finn RS, Furuse J, Ren Z, Yau T, Klumpen HJ, Chan SL, Ozaka M, Verslype C, Bouattour M, Park JO, Barajas O, Pelzer U, Valle JW, Yu L, Malhotra U, Siegel AB, Edeline J, Vogel A; KEYNOTE-966 Investigators. Pembrolizumab in combination with gemcitabine and cisplatin compared with gemcitabine and cisplatin alone for patients with advanced biliary tract cancer (KEYNOTE-966): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023 Jun 3;401(10391):1853-1865. doi: 10.1016/S0140-6736(23)00727-4. Epub 2023 Apr 16.
PubMed 37075781
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Anti-PD-1/PD-L1 Antibodies in Combination With Gemcitabine and Cisplatin for Patients With Advanced Cholangiocarcinoma
- ClinicalTrials.gov ID
- NCT07291063
- Acronym / sponsor ID
- Y2024-752
- Conditions
- Cholangiocarcinoma
- Keywords
- Cholangiocarcinoma, Gemcitabine, Cisplatin, Anti-PD-1/PD-L1 antibody
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Shanghai Zhongshan HospitalUniversity, hospital or other
- Study start
- 19 Jul 2024 (actual)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 18 Dec 2025 (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.
This study is a prospective, observational study designed to analyze the safety, tolerability, and efficacy of first-line treatment using the combination of gemcitabine and cisplatin plus anti-PD-1/PD-L1 antibodies for patients with advanced cholangiocarcinoma.
Detailed description
This is a prospective cohort study of combination anti-PD-1/PD-L1 antibodies plus Gemcitabine and Cisplatin chemotherapy for adult patients (≥18) with advanced cholangiocarcinoma
Gemcitabine and Cisplatin (GC): This chemotherapy doublet has been the historical standard of care for advanced cholangiocarcinoma. It works by interfering with DNA synthesis and causing DNA cross-linking, leading to tumor cell apoptosis.
Anti-PD-1/PD-L1 antibodies: Immunotherapy (including pembrolizumab, durvalumab, envafolimab, tislelizumab, etc.) is designed to block the PD-1/PD-L1 immune checkpoint pathway, thereby reinvigorating T-cells to recognize and attack tumor cells. Recent pivotal trials (e.g., TOPAZ-1, KEYNOTE-966) have demonstrated that adding immunotherapy to GC chemotherapy significantly improves overall survival compared to chemotherapy alone.
This study aims to evaluate the real-world safety, tolerability, and clinical efficacy of this combination regimen in specific clinical practice settings for unresectable late-stage cholangiocarcinoma patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Chemotherapy plus Immunotherapy Combination | – | Drug: Anti-PD-1/PD-L1 antibody, Drug: Gemcitabine and Cisplatin Chemotherapy |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with locally advanced or metastatic biliary tract cancer diagnosed histologically or cytologically.
Key inclusion criteria (2 listed)
- 1. ≥18 years old, male or female 2. Histologically or cytologically confirmed locally advanced or metastatic biliary tract cancer (including ICC, ECC, or GBC) that is not amenable to curative surgery or local therapies.
- 3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1 4. Patient has given written informed consent. 5. The function of important organs meets the requirements 6. Expected survival ≥12 weeks 7. Non-surgical sterilization or women of childbearing age need to use a medically-accepted contraceptive (such as an intrauterine device, contraceptive or condom) during the study period and within 3 months after the end of the study treatment period.
Key exclusion criteria (1 listed)
- 1. The patient has any active autoimmune disease or a history of autoimmune disease (such as the following, but not limited to: autoimmune hepatitis, interstitial pneumonia, uveitis, enteritis, hepatitis, pituitary inflammation, vasculitis, nephritis, thyroid Hyperfunction; patients with vitiligo; complete remission of asthma in childhood, can be included without any intervention after adulthood; asthma patients who require bronchodilators for medical intervention cannot be included); 2. The patient is using immunosuppressive agents or systemic hormonal therapy to achieve immunosuppressive purposes (agents amount > 10 mg / day of prednisone or other therapeutic hormones), and continue to use within 2 weeks before enrollment; 3. Have clinical symptoms or disease that are not well controlled; 4. Significant clinically significant bleeding symptoms or a clear bleeding tendency within 3 months prior to randomization; 5. Arterial/venous thrombosis in the first 6 months of randomization 6. According to the investigator, the patient has other factors that may affect the results of the study or lead to the termination of the study, such as alcohol abuse, drug abuse, other serious diseases (including mental illness) requiring combined treatment, and serious laboratory abnormalities.#with family or social factors, it will affect the safety of patients. 7. Known hypersensitivity to Gemcitabine, Cisplatin, or platinum-containing compounds; significant hearing impairment (grade ≥2) or peripheral neuropathy (grade ≥2) that contraindicates Cisplatin;
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 |
|---|---|---|
| Anti-PD-1/PD-L1 antibodyAnti-PD-1/PD-L1 Intravenous injection for at least 6 months | Drug | Chemotherapy plus Immunotherapy Combination |
| Gemcitabine and Cisplatin ChemotherapyGemcitabine and cisplatin Intravenous injection for at least 6 months | Drug | Chemotherapy plus Immunotherapy Combination |
Primary outcome
Incidence of adverse events
Time frame: Up to 30 days after last treatment dose
Safety will be monitored by addressing and recording all adverse events (AEs), serious adverse events (SAEs) and specific laboratory abnormalities (worst grade). Toxicities will be graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0.
Objective response rate(ORR)
Time frame: 5 years
Evaluated by researchers based on the RECIST 1.1 standard
Progression free survival(PFS)
Time frame: 5 years
Evaluated by researchers based on the RECIST 1.1 standard
To the relief time (TOR)
Time frame: Time Frame: 5 years
Evaluated by researchers based on the RECIST 1.1 standard
Duration of relief(DOR)
Time frame: 5 years
Evaluated by researchers based on the RECIST 1.1 standard
Disease Control Rate (DCR)
Time frame: Time Frame: 5 years
Evaluated by researchers based on the RECIST 1.1 standard
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2025.
- Results publication: PubMed 38319896Oh DY, Ruth He A, Qin S, Chen LT, Okusaka T, Vogel A, Kim JW, Suksombooncharoen T, Ah Lee M, Kitano M, Burris H, Bouattour M, Tanasanvimon S, McNamara MG, Zaucha R, Avallone A, Tan B, Cundom J, Lee CK, Takahashi H, Ikeda M, Chen JS, Wang J, Makowsky M, Rokutanda N, He P, Kurland JF, Cohen G, Valle JW. Durvalumab plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer. NEJM Evid. 2022 Aug;1(8):EVIDoa2200015. doi: 10.1056/EVIDoa2200015. Epub 2022 Jun 1.
- Results publication: 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.
- Results publication: PubMed 37075781Kelley RK, Ueno M, Yoo C, Finn RS, Furuse J, Ren Z, Yau T, Klumpen HJ, Chan SL, Ozaka M, Verslype C, Bouattour M, Park JO, Barajas O, Pelzer U, Valle JW, Yu L, Malhotra U, Siegel AB, Edeline J, Vogel A; KEYNOTE-966 Investigators. Pembrolizumab in combination with gemcitabine and cisplatin compared with gemcitabine and cisplatin alone for patients with advanced biliary tract cancer (KEYNOTE-966): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023 Jun 3;401(10391):1853-1865. doi: 10.1016/S0140-6736(23)00727-4. Epub 2023 Apr 16.
- Individual participant dataSharing planned: Yes
Related trials for Cholangiocarcinoma
Not yet recruitingPhase 1
Hepatic Artery Infusion of Carfilzomib in Participants With Liver Metastatic Disease Previously Treated With Hepatic Artery Infusion Pump Therapy
Colorectal Neoplasms, Neoplasms
Active, not recruitingPhase 2/3
A Study of CTX-009 in Combination With Paclitaxel in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers (COMPANION-002)
Biliary Tract Cancer, Cholangiocarcinoma
Active, not recruitingPhase 1
Prospective Exploratory Study of FAPi PET/CT With Histopathology Validation in Patients With Various Cancers
Bladder Carcinoma, Cervical Carcinoma
RecruitingPhase 2
At-Home Cancer Directed Therapy Versus in Clinic for the Treatment of Patients With Advanced Cancer
Advanced Anal Carcinoma, Advanced Biliary Tract Carcinoma
Active, not recruitingObservational study
Liver Cancer Registry Platform
Hepatocellular Carcinoma (HCC), Cholangiocarcinoma
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.
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 Cholangiocarcinoma, run by Shanghai Zhongshan Hospital at 1 site in China.
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 2 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 18 Dec 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 China. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Qinghai Ye, PhD (2164041990, 24111210117@m.fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: Up to 30 days after last treatment dose). The study lists 6 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 NCT07291063, which also lists 3 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 18 Dec 2025.