Liver Transplantation in Intrahepatic Cholangiocarcinoma

NCT06140134 | Recruiting DMC

• 1 other identifier: Pro2021000973
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 2

Brief Summary

The aim of the current study is to determine the potential efficacy of liver transplantation in the form of patients' overall survival (OS) after neoadjuvant systemic therapy in patients with biologically responsive locally advanced non-metastatic intrahepatic cholangiocarcinoma (iCCA) in comparison to patients historically treated with chemotherapy alone.

Conditions

Intrahepatic Cholangiocarcinoma; Cholangiocarcinoma, Intrahepatic

Keywords

liver transplantation; neoadjuvant systemic therapy; intrahepatic cholangiocarcinoma; iCCA; locally advanced non-metastatic iCCA; non-metastatic intrahepatic cholangiocarcinoma; liver transplant; unresectable intrahepatic cholangiocarcinoma; locally advanced intrahepatic cholangiocarcinoma

Outcome Measures

Primary Outcomes (1)

• 5-year Overall Survival Rate (ORS)

  5-year ORS is the length of time that patients diagnosed with locally advanced non-metastatic intrahepatic cholangiocarcinoma (iCCA) treated with neoadjuvant systemic therapy followed by liver transplant (LT) are still alive post-LT.

  5 years from the time of transplant or until death of patient, whichever came first

Secondary Outcomes (3)

• Recurrence-free survival (RFS)

  From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 5 years

• Intent-to-treat Overall Survival Rate (ITT ORS)

  5 years from time of waitlist or until death of patient, whichever came first

• Post-liver transplant functional assessments to identify postoperative complications, short-term morbidities, and long-term morbidities

  From date of emergence of a post-transplant adverse event until the date the adverse event is designated as resolved by investigator or its effect on patient's condition stabilizes, whichever came first, assessed up to 5 years

Study Arms (1)

Intrahepatic cholangiocarcinoma listed for liver transplant

Listed patients enrolled with diagnosis of intrahepatic cholangiocarcinoma will be enrolled on an intent to treat basis with comparison between patients receiving transplant versus those who expire or are removed from the waitlist.

Procedure: Liver Transplant

Interventions

Liver Transplant PROCEDURE

Whole liver allotransplantation will be performed for patients on the liver transplant waiting list

Also known as: Orthotopic liver transplantation

Intrahepatic cholangiocarcinoma listed for liver transplant

Eligibility Criteria

• Age: 18 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients with biologically responsive locally advanced non-metastatic intrahepatic cholangiocarcinoma (iCCA) treated with neoadjuvant systemic chemotherapy with a radiographic disease stability or regression.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Rutgers, The State University of New Jersey: lead

Study Sites (2)

Rutgers New Jersey Medical School

Newark, New Jersey, 07103, United States

RECRUITING

University Hospital

Newark, New Jersey, 07103, United States

RECRUITING

Related Publications (29)

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Biospecimen

Retention: SAMPLES WITH DNA

Serial collection of patient blood for fractionation into serum/plasma and PBMCs (peripheral blood mononuclear cells) will be cryopreserved. Liver explant tissue (tumor and non-tumor) and biopsy samples will be collected. Samples will be utilized for extraction of DNA, RNA, protein, and histologic analysis.

MeSH Terms

Conditions

Cholangiocarcinoma

Interventions

Liver Transplantation

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Tissue Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Digestive System Surgical Procedures; Surgical Procedures, Operative; Organ Transplantation; Transplantation

Study Officials

• Keri E Lunsford, MD, PhD

  Rutgers University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Melissa Lugardo

CONTACT

973-972-8412; lugardme@uhnj.org

Angenie Preval, PA

CONTACT

prevalan@uhnj.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Translational and Surgical Science, Department of Surgery/Assistant Professor, Division of Transplant and Hepatobiliary Surgery/Member, Center for Immunity and Inflammation

Study Record Dates

• First Submitted: October 2, 2023
• First Posted: November 18, 2023
• Study Start: December 15, 2023
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2029
• Last Updated: March 20, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Following publication of study results

Locations

US (2)