Clinical trial Recruiting at 1 site Phase2; Phase3 Interventional Ages 18 and up

Neoadjuvant gemcitabine and cisplatin with perioperative pembrolizumab vs upfront surgery for cancers of the bile ducts

Official title: Neoadjuvant Gemcitabine and Cisplatin in Combination With Perioperative Pembrolizumab Versus Upfront Surgery for Patients With Primary Resectable and Borderline Resectable Perihilar and Distal Cholangiocarcinoma (NEODISCO): A Multicentre Phase 2B/3 Randomized Controlled Trial

Run by Amsterdam UMC, location VUmc · NCT06923475 · Data updated 2026-11-12

This trial tests whether giving chemotherapy with pembrolizumab before surgery helps people with resectable or borderline resectable extrahepatic cholangiocarcinoma see if they have better outcomes than going straight to surgery.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Ages18 Years and up
SexAll
Enrollment150 (estimated)
SponsorAmsterdam UMC, location VUmc
Academic / other
Drugs / interventionspembrolizumab
Locations1 site (Amsterdam)
StatusRecruiting
StartedApril 17, 2026
Main results expectedNovember 6, 2028
Study endsNovember 6, 2028
Registry record updatedAugust 25, 2026
Trial IDNCT06923475 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Adults (18+) with histologically or cytologically confirmed resectable or borderline resectable perihilar or distal cholangiocarcinoma, adequate ECOG 0-1, and suitable biliary drainage when needed.

Probably not a fit

Patients whose disease progresses during neoadjuvant therapy or do not reach resection may not benefit from the perioperative approach.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, randomized study compares a neoadjuvant regimen of gemcitabine and cisplatin plus perioperative pembrolizumab against upfront surgical resection in patients with resectable or borderline resectable perihilar and distal cholangiocarcinoma. The trial aims to determine if the preoperative approach can improve event-free survival and increase the likelihood of a complete (R0) resection. Eligible participants are adults with confirmed disease, adequate performance status, and suitable biliary drainage when needed. The study is conducted across multiple centers in the Netherlands, led by Amsterdam UMC with collaboration from Maastricht, UMC Utrecht, and UMC Groningen.

Why it matters

Potential benefit: If successful, patients may have longer progression-free and overall survival and a higher chance of a complete tumor removal.

How similar studies have gone: Previous approaches using neoadjuvant regimens in biliary cancers have shown signals of feasibility and potential improvements in surgical outcomes, but results are variable and this trial tests a novel combination with pembrolizumab.

Who can join: the full rules

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Histologically or cytologically confirmed resectable or borderline resectable pCCA and dCCA. These are all the patients considered candidates for upfront surgical exploration, with intent to resect, as confirmed by local MDT and the study expert panel also taking into consideration endoscopic and radiological findings. In cases where drainage is not required, patients with a disease highly suspicious for extrahepatic cholangiocarcinoma, as determined by the expert MDT, may be included without histological proof to prevent unnecessary post-ERCP complications.
* Successful drainage, in case of clinical significant bile duct obstruction.
* MidCCA inclusion in the NEODISCO-trial will be permitted and will be included according to the proposed type of resection.
* Male/female participants who are at least 18 years of age on the day of signing informed consent.
* Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1. Evaluation of ECOG is to be performed within 7 days prior to the first dose of study intervention.

Exclusion Criteria:

* Upfront "clearly" unresectable pCCA: circumferential unreconstructable vascular involvement of the FLR and/or insufficient FLR for potential radical resection. Insufficient FLR is defined as \<30% residual volume or a function \<2.7 min/m2. Patients considered borderline resectable but, by the discretion of the MDT, not a candidate for upfront exploration/resection, are considered ineligible for NEODISCO.
* Upfront clearly unresectable dCCA (following DPCG criteria).
* Patients with proven N2 lymph nodes (according to the AJCC 8th edition).
* PCCA eligible for liver transplantation.
* Intrahepatic cholangiocarcinoma with hilar involvement.
* Cancer suspicious for ampullary carcinoma (for instance involvement of papilla during endoscopy).
* Local recurrence following prior resection of eCCA (patients who develop local recurrence during the study are however not excluded).
* Patients with underlying liver diseases: PSC, untreated hepatitis, cirrhosis child-Pugh B, C.
* Previous malignancy unless no evidence of disease, or diagnosed more than 3 years before diagnosis of eCCA, or with a life expectancy of more than 5 years from date of inclusion.
* Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (eg, CTLA-4, OX-40, CD137).

Where it's running

  • Amsterdam University Medical Center
    Amsterdam, Netherlands
    Recruiting

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.