Liver transplantation for patients with advanced intrahepatic cholangiocarcinoma

A Single-Arm Nonrandomized Phase II Study of Liver Transplantation in Locally Advanced Unresectable Non-Metastatic Intrahepatic Cholangiocarcinoma Treated With Neoadjuvant Systemic Therapy

Observational Rutgers, The State University of New Jersey · NCT06140134

This study is testing if liver transplantation can help patients with advanced bile duct cancer live longer after they’ve had treatment, compared to those who only received chemotherapy.

Quick facts

Study typeObservational
Enrollment30 (estimated)
Ages18 Years to 89 Years
SexAll
SponsorRutgers, The State University of New Jersey Academic / other
Drugs / interventionschemotherapy
Locations2 sites (Newark, New Jersey and 1 other locations)
Trial IDNCT06140134 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the effectiveness of liver transplantation in improving overall survival for patients with locally advanced non-metastatic intrahepatic cholangiocarcinoma (iCCA) who have undergone neoadjuvant systemic therapy. It compares the outcomes of these patients to those who have historically received chemotherapy alone. The research focuses on patients with biologically responsive tumors, assessing the potential for transplantation as a viable treatment option where traditional surgical resection has shown limited success. The study is observational and will gather data on patient outcomes over time.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with histologically confirmed locally advanced unresectable iCCA who have shown disease stability or regression after neoadjuvant therapy.

Not a fit: Patients with metastatic disease or those who do not meet the criteria for organ and marrow function acceptable for transplantation may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve survival rates for patients with advanced iCCA who currently have limited treatment options.

How similar studies have performed: Previous studies have shown poor outcomes for liver transplantation in iCCA, making this approach novel and potentially groundbreaking if successful.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Age ≥18 years of age on the day of consenting to the study.
* Patients must have histologically confirmed diagnosis of locally advanced intrahepatic cholangiocarcinoma
* Confirmed diagnosis of locally advanced unresectable iCCA with no vascular invasion, lymph node, or extrahepatic disease.
* Unresectable disease based on tumor location or underlying liver disease
* Patients must have ≥ 6 months of disease stability or tumor regression on neoadjuvant therapy. In cases in which patients had received second-line therapy, disease must also have been controlled for ≥ 6 months on that regimen.
* Patients who had previous surgical resection for iCCA are eligible if surgery occurred more than 6 months prior to listing, and patients have had ≥ 6 months of disease stability or response on therapy.
* ECOG performance status ≤1 (Karnofsky ≥70%, see Appendix A).
* Patients must have organ and marrow function acceptable for liver transplantation per institutional protocol:
* If history of chronic hepatitis B virus (HBV) infection, viral load should be undetectable on suppressive therapy.
* If history of chronic hepatitis C virus (HCV) infection, patients should have undetectable HCV viral load.
* Women of child-bearing years must have contraception plan in place from the time of study enrollment until at least one year following liver transplant.
* Ability to understand and the willingness to sign a written informed consent document
* Meets all other medical and psychosocial criteria for liver transplant
* Demonstrate ability to comply with study procedures

Exclusion Criteria:

* Age \<18 years of age on the day of consenting to the study.
* Patients who have extrahepatic metastases, lymph node involvement, invasion or encasement of major hepatic vascular structures, perforation of the visceral peritoneum, invasion of extrahepatic structures, invasion of perihilar fat, periductular invasion, concurrent hepatoma or mixed hepatocellular cholangiocarcinoma.
* Concurrent severe and/or uncontrolled concurrent illness including, but not limited to, ongoing or active infection, acute fulminant liver failure, symptomatic congestive heart failure, unstable angina pectoris, severe uncorrected coronary artery disease, severe cerebrovascular disease, severe pulmonary disease, or psychiatric illness/social situations that would limit compliance with study requirements and that would exclude the patient from eligibility for liver transplantation per institutional protocol.
* Prior solid organ or bone marrow transplant
* Dependent on ≥2 IV inotropic support to maintain hemodynamics
* Previous (within the past 5 years) or concurrent presence of other cancer, except non-melanoma skin cancer and in situ carcinomas.
* Eastern Cooperative Oncology Group (ECOG) Performance Status Scale score \>1 (Karnofsky \<70%, see Appendix A).
* Unable to understand and sign a written informed consent document
* Untreated viral hepatitis
* Pregnant or breast-feeding women
* HIV-infected patients

Where this trial is running

Newark, New Jersey and 1 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Intrahepatic CholangiocarcinomaCholangiocarcinoma, Intrahepaticliver transplantationneoadjuvant systemic therapyintrahepatic cholangiocarcinomaiCCAlocally advanced non-metastatic iCCAnon-metastatic intrahepatic cholangiocarcinoma
Last reviewed 2026-06-13 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.