Clinical trial NCT06988592: GemCis Plus PD-1/PD-L1 Inhibitor in Advanced Biliary Tract Cancer: Efficacy and Immune Microenvironment （BTC-IM-1）
Official title: A Prospective, Non-interventional Study of Gemcitabine/Cisplatin Combined With PD-1/PD-L1 Inhibitor as First-line Treatment in Treatment-naïve Advanced Biliary Tract Cancer Patients: Efficacy and Immune Microenvironment Dynamics
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Advanced Biliary Tract Cancer
Lead sponsor: Fudan University
Sponsor class: OTHER
First posted: 2025-05-25
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: This is a prospective, non-interventional, observational study evaluating the efficacy and immune microenvironment changes in treatment-naïve advanced biliary tract cancer (BTC) patients receiving first-line standard therapy with gemcitabine/cisplatin (GemCis) combined with PD-1/PD-L1 inhibitors.

Brief summary: To evaluate the efficacy and immune microenvironment changes in treatment-naïve advanced biliary tract cancer (BTC) patients receiving first-line standard therapy with gemcitabine/cisplatin (GemCis) combined with PD-1/PD-L1 inhibitors.

Detailed description: This is a prospective, non-interventional, observational study evaluating the efficacy and immune microenvironment changes in treatment-naïve advanced biliary tract cancer (BTC) patients receiving first-line standard therapy with gemcitabine/cisplatin (GemCis) combined with PD-1/PD-L1 inhibitors. The primary endpoint is objective response rate (ORR), with secondary endpoints including disease control rate (DCR), duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS), and immune profiling of tumor tissue and peripheral blood before and after treatment.

Eligibility criteria: Inclusion Criteria:

* Age ≥18 years.
* Histologically confirmed unresectable/metastatic cholangiocarcinoma (intrahepatic, extrahepatic, or gallbladder).
* No prior systemic anticancer therapy (chemotherapy, targeted therapy, or immunotherapy).
* Planned to receive GemCis+PD-1/PD-L1 inhibitor as standard first-line treatment.
* ≥1 measurable lesion per RECIST 1.1.
* ECOG performance status 0-1.
* Adequate organ function:

  * ANC ≥1.5 × 10⁹/L, platelets ≥100 × 10⁹/L, hemoglobin ≥9 g/dL.
  * Total bilirubin ≤1.5 × ULN, AST/ALT ≤3 × ULN (≤5 × ULN if liver metastases).
  * Creatinine ≤1.5 × ULN or CrCl ≥60 mL/min.
* Willing to provide archival/fresh tumor tissue and peripheral blood samples.
* Signed informed consent.

Exclusion Criteria:

* Prior systemic therapy.
* Active autoimmune disease requiring immunosuppression.
* Active infection requiring IV antibiotics.
* HIV-positive or active HBV/HCV infection (HBsAg+ with HBV DNA ≥2000 IU/mL; HCV RNA+).
* Symptomatic CNS metastases.
* Pregnancy/lactation.
* Any condition compromising protocol compliance or data interpretation per investigator.

Primary outcomes:
measure: Overall survival (OS) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: max 42 months
Secondary outcomes:
measure: Objective Response Rate (ORR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: max 24 months
measure: Disease control rate (DCR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: max 24 months
measure: Duration of Response (DOR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: max 24 months
measure: Time to Response (TTR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: max 24 months
measure: Progression Free Survival (PFS) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: max 24 months
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
timeFrame: max 42 months
measure: Translational study
timeFrame: max 24 months
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Zhongshan Hospital, Fudan University
state: Shanghai
Study officials:
References: