Combining GemCis with PD-1/PD-L1 inhibitors for advanced biliary tract cancer

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

This study is testing if combining a chemotherapy treatment with immune therapy can help people with advanced biliary tract cancer feel better and live longer.

Quick facts

• Study type: Observational
• Enrollment: 100 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Fudan University (other)
• Drugs / interventions: chemotherapy, immunotherapy
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06988592 on ClinicalTrials.gov

What this trial studies

This observational study aims to evaluate the efficacy and changes in the immune microenvironment of treatment-naïve patients with advanced biliary tract cancer receiving first-line therapy with gemcitabine/cisplatin (GemCis) combined with PD-1/PD-L1 inhibitors. The study will assess the objective response rate as the primary endpoint, while secondary endpoints will include disease control rate, duration of response, time to response, progression-free survival, overall survival, and immune profiling of tumor tissue and blood samples before and after treatment. Participants will provide both archival and fresh tumor tissue and blood samples for comprehensive analysis.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with histologically confirmed unresectable or metastatic cholangiocarcinoma who have not received prior systemic therapy.

Not a fit: Patients with prior systemic therapy or active autoimmune diseases requiring immunosuppression may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could improve treatment outcomes for patients with advanced biliary tract cancer.

How similar studies have performed: Other studies combining chemotherapy with PD-1/PD-L1 inhibitors have shown promising results, suggesting potential for success in this approach.

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.

Where this trial is running

Shanghai, Shanghai Municipality

• Zhongshan Hospital, Fudan University — Shanghai, Shanghai Municipality, China (RECRUITING)

Study contacts

• Study coordinator: Peng Wang, MD

Conditions: Advanced Biliary Tract Cancer