GemCis Plus PD-1/PD-L1 Inhibitor in Advanced Biliary Tract Cancer: Efficacy and Immune Microenvironment （BTC-IM-1）

NCT06988592 | Recruiting

• 1 other identifier: BTC-IM-1
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

Advanced Biliary Tract Cancer

Outcome Measures

Primary Outcomes (1)

• Overall survival (OS) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

  OS is defined as the time from study treatment to the date of death of the subject, regardless of the cause of death.

  max 42 months

Secondary Outcomes (7)

• Objective Response Rate (ORR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

  max 24 months

• Disease control rate (DCR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

  max 24 months

• Duration of Response (DOR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

  max 24 months

• Time to Response (TTR) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

  max 24 months

• Progression Free Survival (PFS) evaluated by the investigator per Response Evaluation Criteria in Solid Tumors Version 1.1

  max 24 months

Study Arms (1)

BTC cohort

Drug: GemCis plus PD-1/PD-L1 inhibitor

Interventions

GemCis plus PD-1/PD-L1 inhibitor DRUG

Gemcitabine: 1000 mg/m², intravenous infusion, on Day 1 and Day 8 Cisplatin: 25 mg/m², intravenous infusion, on Day 1 and Day 8 PD-1/PD-L1 inhibitor: An approved agent (e.g., Pembrolizumab, Nivolumab, Atezolizumab, etc.) will be selected based on clinical practice and administered at standard doses and schedules. Each treatment cycle lasts 21 days, continuing until disease progression, unacceptable toxicity, or patient/physician decision to discontinue.

BTC cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Fudan University: lead

Study Sites (1)

Zhongshan Hospital, Fudan University

Shanghai, Shanghai Municipality, 200032, China

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

A baseline tumor biopsy and blood collection before initiation of treatment, followed by a second tumor biopsy with paired blood collection after the first response evaluation will be planned.

Central Study Contacts

Peng Wang, MD

CONTACT

86-21-64041990; peng_wang@fudan.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 16, 2025
• First Posted: May 25, 2025
• Study Start: May 23, 2025
• Primary Completion (Estimated): May 18, 2027
• Study Completion (Estimated): December 30, 2027
• Last Updated: May 29, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)