Advanced Cholangiocarcinoma (Part 1)NCT07164703
A Real-World Study of Precision Treatment for Advanced Cholangiocarcinoma Based on Molecular Subtyping
An observational study of Advanced Cholangiocarcinoma (Part 1), run by Mao-Lin Yan at 1 site in China.
Trial summary
This is a multi-center, real-world study aiming to evaluate the efficacy and safety of precision treatment for advanced cholangiocarcinoma (CCA) with different molecular subtypes. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Advanced Cholangiocarcinoma (Part 1)
- What is being tested?
- Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations.
- How many people
- 55 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2025to 31 Oct 2029
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study population will include male and female adults aged 18 to 75 years. Participants must have histologically confirmed, unresectable advanced biliary tract carcinoma, which encompasses intrahepatic or extrahepatic cholangiocarcinoma (CCA) and gallbladder cancer.A key criterion for inclusion is the presence of a documented targetable gene mutation, such as FGFR2 fusion/rearrangement, IDH-1 or IDH-2 mutations, ERBB2 (HER2) amplification/overexpression, BRAF V600E mutation, or others.Eligible patients are required to have at least one measurable lesion according to RECIST 1.1 criteria and a good performance status, indicated by an Eastern Cooperative Oncology Group (ECOG) score of 0 or 1. Furthermore, participants must have a life expectancy of more than 3 months and adequate organ function, including Child-Pugh class A or B liver function and specified levels of hematologic and renal function.
Who may be able to join
- Age ≥ 18 and ≤ 75 years.
- Histologically confirmed, unresectable advanced biliary tract carcinoma, including cholangiocarcinoma (intrahepatic or extrahepatic) and gallbladder cancer (diagnosed according to the 2025 Chinese Society of Clinical Oncology (CSCO) Guidelines for Biliary Tract Malignancies and confirmed by a multidisciplinary team). Must have a documented targetable gene mutation, including but not limited to: Fibroblast Growth Factor Receptor 2 (FGFR2) fusion/rearrangement, Isocitrate Dehydrogenase (NADP(+)) 1 (IDH-1) and Isocitrate Dehydrogenase (NADP(+)) 2 (IDH-2) mutations, Erb-B2 Receptor Tyrosine Kinase 2 (ERBB2/HER2) amplification and/or overexpression, B-Raf Proto-Oncogene, Serine/Threonine Kinase (BRAF) V600E mutation, Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) mutation, Neurotrophic Tyrosine Receptor Kinase (NTRK) fusion, Rearranged during Transfection (RET) fusion, Ring Finger Protein 43 (RNF43) mutation, MET Proto-Oncogene, Receptor Tyrosine Kinase (MET) amplification, Epidermal Growth Factor Receptor (EGFR) mutation, Phosphatidylinositol-4,5-Bisphosphate 3-Kinase Catalytic Subunit Alpha (PIK3CA) mutation, BRCA1 DNA Repair Associated (BRCA1)/BRCA2 DNA Repair Associated (BRCA2) mutation, Partner and Localizer of BRCA2 (PALB2) mutation, Vascular Endothelial Growth Factor Receptor (VEGFR) mutation, deficient Mismatch Repair (dMMR)/Microsatellite Instability-High (MSI-H), or Neuregulin 1 (NRG1) mutation.
- At least one measurable lesion according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (measurable lesion defined as longest diameter ≥10 mm on Computed Tomography (CT)/Magnetic Resonance Imaging (MRI) scan).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
- Child-Pugh class A or B liver function. For patients with obstructive jaundice, total bilirubin must be ≤ 50 µmol/L. Biliary drainage is recommended if total bilirubin is > 50 µmol/L.
- Adequate hematologic function: Absolute Neutrophil Count (ANC) ≥ 1.5 × 10^9/L, Hemoglobin (Hb) ≥ 8.5 g/L, Platelets (PLT) ≥ 75 × 10^9/L.
- No history of severe arrhythmia or heart failure; no severe ventilation dysfunction or severe pulmonary infection; no acute or chronic renal failure with creatinine clearance > 40 mL/min.
- Life expectancy of more than 3 months.
Who cannot join
- Major surgery within 28 days prior to enrollment.
- Presence of any unresolved toxicity of Grade ≥ 2 (according to Common Terminology Criteria for Adverse Events [CTCAE] v4.0) from prior anticancer therapy at the time of enrollment, with the exception of alopecia or Grade 2 anemia.
- Known hypersensitivity to any components or excipients of the study drugs.
- Presence of any active autoimmune disease or a history of autoimmune disease with an expected recurrence. Patients currently receiving immunosuppressants or systemic corticosteroids for immunosuppressive purposes.
- Presence of other concurrent malignancies.
- Presence of brain metastases or spinal cord compression.
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- Presence of any severe and/or unstable pre-existing medical conditions, mental illness, or other conditions that could compromise patient safety, the ability to provide informed consent, or adherence to the study procedures.
- Pregnant or breastfeeding women.
- History of organ transplantation.
- Patients with proteinuria ≥ 1+ on urinalysis must undergo a 24-hour urine protein test. Patients with a 24-hour urine protein level ≥ 1g are excluded.
- Clinically significant corneal or retinal disease confirmed by ophthalmologic examination.
- Liver tumor burden exceeding 50%.
- Hypothyroidism or hyperthyroidism.
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
Affiliated Provincial Hospital of Fuzhou University
Fuzhou, Fujian, 350001
Maolin Yan, Doctor, 15960066307, yanmaolin74@163.com
About this study
Official title: Department of Hepatobiliary Pancreatic Surgery, Fujian Provincial Hospital
This is a multi-center, real-world study aiming to evaluate the efficacy and safety of precision treatment for advanced cholangiocarcinoma (CCA) with different molecular subtypes.
- Conditions
- Advanced Cholangiocarcinoma (Part 1)
- Being tested
- Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations.Drug
Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations.
| Study group | Role | What they receive |
|---|---|---|
| Precision Treatment Based on Molecular Subtyping (or Gene Mutations) | Not described Receives: Drug: Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate, ORR
The Objective response rate (ORR) was defined as the complete response (CR) rate or the partial response (PR) rate according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria.
Measured: Four weeks after the initiation of medication
Secondary outcomes (5)
Overall survival, OS
The Overall survival (OS) was defined as the time between receiving treatment and observing death or loss of follow-up for any reason.
Measured: From date of enrollment until the date of death from any cause, assessed up to 48 months
Progression-free survival, PFS
The Progression free survival (PFS) was defined as the time between the start of treatment and the progression of intrahepatic and/or extrahepatic tumors, or the occurrence of death or loss of follow-up for any reason.
Measured: From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months
Disease control rate, DCR
The Disease control rate (DCR) was defined as the complete response (CR) rate or the partial response (PR) rate or stable disease (SD) rate according to RECIST v1.1 criteria.
Measured: Four weeks after the initiation of medication
Treatment-related adverse events, TRAEs treatment-related adverse events
The incidence, spectrum and severity of adverse events (AE) and serious adverse events (SAE) were determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) V5.0 standard. Dose suspension rate and dose termination rate due to adverse events.
Measured: From the initiation of medication, with recordings made whenever an adverse reaction occurs, assessed up to 48 months
Conversion Resection Rate, CRR
The proportion of all enrolled patients who meet the criteria for resectability and undergo surgical resection after receiving study treatment.
Measured: Four weeks after the initiation of medication until the day before surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2025
- Study start1 Oct 2025 (estimated)
- Primary completion1 Oct 2029 (estimated)
- Study completion31 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Cholangiocarcinoma (Part 1)Studies 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
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mao-Lin Yan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Mao-Lin Yan, Fujian Provincial Hospital
- Investigators
- Maolin Lin, Doctor, Study chair, Department of Hepatobiliary and Pancreatic Surgery, Affiliated Provincial Hospital of Fuzhou University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07164703
- Sponsor's protocol ID
- FJPH-CCA-PT-2025
- Other IDs
- FJPH-CCA-PT-2025
- Status last verified
- Sep 2025
- First submitted
- 31 Aug 2025
- Last updated
- 10 Sep 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Department of Hepatobiliary Pancreatic Surgery, Fujian Provincial Hospital
- ClinicalTrials.gov ID
- NCT07164703
- Acronym / sponsor ID
- FJPH-CCA-PT-2025
- Conditions
- Advanced Cholangiocarcinoma (Part 1)
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Mao-Lin YanUniversity, hospital or other
- Study start
- 1 Oct 2025 (estimated)
- Primary completion
- 1 Oct 2029 (estimated)
- Study completion
- 31 Oct 2029 (estimated)
- First posted
- 10 Sep 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 is a multi-center, real-world study aiming to evaluate the efficacy and safety of precision treatment for advanced cholangiocarcinoma (CCA) with different molecular subtypes.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Precision Treatment Based on Molecular Subtyping (or Gene Mutations) | – | Drug: Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population will include male and female adults aged 18 to 75 years. Participants must have histologically confirmed, unresectable advanced biliary tract carcinoma, which encompasses intrahepatic or extrahepatic cholangiocarcinoma (CCA) and gallbladder cancer.A key criterion for inclusion is the presence of a documented targetable gene mutation, such as FGFR2 fusion/rearrangement, IDH-1 or IDH-2 mutations, ERBB2 (HER2) amplification/overexpression, BRAF V600E mutation, or others.Eligible patients are required to have at least one measurable lesion according to RECIST 1.1 criteria and a good performance status, indicated by an Eastern Cooperative Oncology Group (ECOG) score of 0 or 1. Furthermore, participants must have a life expectancy of more than 3 months and adequate organ function, including Child-Pugh class A or B liver function and specified levels of hematologic and renal function.
Key inclusion criteria (8 listed)
- Age ≥ 18 and ≤ 75 years.
- Histologically confirmed, unresectable advanced biliary tract carcinoma, including cholangiocarcinoma (intrahepatic or extrahepatic) and gallbladder cancer (diagnosed according to the 2025 Chinese Society of Clinical Oncology (CSCO) Guidelines for Biliary Tract Malignancies and confirmed by a multidisciplinary team). Must have a documented targetable gene mutation, including but not limited to: Fibroblast Growth Factor Receptor 2 (FGFR2) fusion/rearrangement, Isocitrate Dehydrogenase (NADP(+)) 1 (IDH-1) and Isocitrate Dehydrogenase (NADP(+)) 2 (IDH-2) mutations, Erb-B2 Receptor Tyrosine Kinase 2 (ERBB2/HER2) amplification and/or overexpression, B-Raf Proto-Oncogene, Serine/Threonine Kinase (BRAF) V600E mutation, Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) mutation, Neurotrophic Tyrosine Receptor Kinase (NTRK) fusion, Rearranged during Transfection (RET) fusion, Ring Finger Protein 43 (RNF43) mutation, MET Proto-Oncogene, Receptor Tyrosine Kinase (MET) amplification, Epidermal Growth Factor Receptor (EGFR) mutation, Phosphatidylinositol-4,5-Bisphosphate 3-Kinase Catalytic Subunit Alpha (PIK3CA) mutation, BRCA1 DNA Repair Associated (BRCA1)/BRCA2 DNA Repair Associated (BRCA2) mutation, Partner and Localizer of BRCA2 (PALB2) mutation, Vascular Endothelial Growth Factor Receptor (VEGFR) mutation, deficient Mismatch Repair (dMMR)/Microsatellite Instability-High (MSI-H), or Neuregulin 1 (NRG1) mutation.
- At least one measurable lesion according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (measurable lesion defined as longest diameter ≥10 mm on Computed Tomography (CT)/Magnetic Resonance Imaging (MRI) scan).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
- Child-Pugh class A or B liver function. For patients with obstructive jaundice, total bilirubin must be ≤ 50 µmol/L. Biliary drainage is recommended if total bilirubin is > 50 µmol/L.
Key exclusion criteria (13 listed)
- Major surgery within 28 days prior to enrollment.
- Presence of any unresolved toxicity of Grade ≥ 2 (according to Common Terminology Criteria for Adverse Events [CTCAE] v4.0) from prior anticancer therapy at the time of enrollment, with the exception of alopecia or Grade 2 anemia.
- Known hypersensitivity to any components or excipients of the study drugs.
- Presence of any active autoimmune disease or a history of autoimmune disease with an expected recurrence. Patients currently receiving immunosuppressants or systemic corticosteroids for immunosuppressive purposes.
- Presence of other concurrent malignancies.
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 |
|---|---|---|
| Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations.Targeted drugs or immunotherapies for cholangiocarcinoma based on targetable gene mutations. | Drug | Precision Treatment Based on Molecular Subtyping (or Gene Mutations) |
Primary outcome
Objective response rate, ORR
Time frame: Four weeks after the initiation of medication
The Objective response rate (ORR) was defined as the complete response (CR) rate or the partial response (PR) rate according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria.
5 secondary outcomes
Overall survival, OS
Time frame: From date of enrollment until the date of death from any cause, assessed up to 48 months
Progression-free survival, PFS
Time frame: From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months
Disease control rate, DCR
Time frame: Four weeks after the initiation of medication
Treatment-related adverse events, TRAEs treatment-related adverse events
Time frame: From the initiation of medication, with recordings made whenever an adverse reaction occurs, assessed up to 48 months
Conversion Resection Rate, CRR
Time frame: Four weeks after the initiation of medication until the day before surgery
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 10 Sep 2025.
- Individual participant dataSharing planned: No
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.
Maolin Lin, Doctor study chair
Department of Hepatobiliary and Pancreatic Surgery, Affiliated Provincial Hospital of Fuzhou 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 Advanced Cholangiocarcinoma (Part 1), run by Mao-Lin Yan 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 8 inclusion and 13 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: opening soon, as of the sponsor's last update on 10 Sep 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 Maolin Yan, Doctor (15960066307, yanmaolin74@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate, ORR (time frame: Four weeks after the initiation of medication). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Sep 2025; AzTrials last refreshed its copy on 7 Nov 2026.