Liver-directed chemotherapy plus systemic drugs to downstage biliary tract cancer

HAIC Combined With Systemic Therapy as De-escalation Therapy Strategy for Biliary Tract Cancer: A Conceptual Study

This trial will test whether adding liver-directed arterial chemotherapy to standard systemic treatment can shrink tumors and allow less extensive surgery for adults with untreated biliary tract cancer.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Peking University (Academic / other)
• Drugs / interventions: durvalumab, pembrolizumab, bevacizumab, toripalimab, chemotherapy
• Locations: 2 sites (Beijing and 1 other locations)
• Trial ID: NCT07466238 on ClinicalTrials.gov

What this trial studies

This interventional protocol combines hepatic arterial infusion chemotherapy (HAIC) — using regimens such as GOLF or FOLFOX — with systemic agents including gemcitabine, bevacizumab, and PD‑1/PD‑L1 inhibitors to try to downstage biliary tract cancers. The goal is to increase objective tumor responses and convert unresectable or borderline tumors into candidates for curative resection. Eligible patients are treatment‑naive adults with intrahepatic or perihilar cholangiocarcinoma or gallbladder cancer, limited or no distant metastasis, ECOG <2, and preserved liver and organ function. Treatment is delivered at Peking University Cancer Hospital and involves repeated locoregional infusions and systemic therapy with monitoring for response and safety.

Who should consider this trial

Good fit: Ideal candidates are treatment‑naive adults (18–80 years) with intrahepatic or perihilar cholangiocarcinoma or gallbladder cancer, ECOG 0–1, Child‑Pugh A or B (≤7), and no widespread extrahepatic metastases.

Not a fit: Patients with distal cholangiocarcinoma, multiple extrahepatic metastases, poor organ function, prior systemic therapy, pregnancy, or allergy to contrast agents or oxaliplatin are unlikely to benefit.

Why it matters

Potential benefit: If successful, this approach could increase tumor shrinkage and resectability, allowing more patients to receive potentially curative surgery and improving survival.

How similar studies have performed: Recent phase III trials (TOPAZ‑1, KEYNOTE‑966) showed survival gains by adding immune checkpoint inhibitors to GemCis, but combining HAIC with systemic and immunotherapies as a de‑escalation strategy remains relatively novel.

Eligibility criteria

Inclusion Criteria:

* Age: 18-80 years, both genders.
* Diagnosis of biliary tract cancer (including intrahepatic cholangiocarcinoma, perihilar cholangiocarcinoma, and gallbladder cancer) and confirmed by histopathological or cytopathological examination.
* Without distant metastasis or limited distant metastasis
* Treatment-naive.
* ECOG PS score \< 2.
* Child-Pugh score: Class A or B (≤7).
* Normal major organ function, meeting the following standards:(1) Blood routine examination:A. Hb≥90 g/L;B. ANC≥1.5×10\^9/L;C. PLT≥75×10\^9/L;(2) Biochemical examination:A. ALB ≥30g/L;B. ALT and AST\<5×ULN;C. TBiL ≤5×ULN;D. Creatinine ≤1.5×ULN;(3) Coagulation function:A. International normalized ratio (INR) ≤1.5×ULN;B. Activated partial thromboplastin time (APTT) ≤1.5×ULN.

Exclusion Criteria:

* Coexistent or synchronous malignancies.
* Distal cholangiocarcinoma.
* Allergic to contrast agents or oxaliplatin.
* Pregnant or lactating women.
* Multiple extrahepatic metastases or combined malignant pleural and peritoneal effusions.
* History of organ transplantation.
* With infections requiring anti-infection treatment.
* Severe and irreparable coagulation dysfunction.

Where this trial is running

Beijing and 1 other locations

• Peking University Cancer Hospital — Beijing, China (Recruiting)
• Peking University Cancer Hospital — Beijing, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Xiaodong Wang

Conditions Biliary Tract Cancer; biliary tract cancer; hepatic arterial infusion chemotherapy; systemic therapy