Clinical trial · Observational
Liver Transplantation in 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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Cholangiocarcinoma, Intrahepatic | Intrahepatic Cholangiocarcinoma | ONTOLOGY_EXACT | 0.98 |
| Intrahepatic Cholangiocarcinoma | Intrahepatic Cholangiocarcinoma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Liver Transplant | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- label
- Intrahepatic cholangiocarcinoma listed for liver transplant
- description
- 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.
- interventionNames
- Procedure: Liver Transplant
Primary outcomes (1)
- measure
- 5-year Overall Survival Rate (ORS)
- timeFrame
- 5 years from the time of transplant or until death of patient, whichever came first
- description
- 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.
Secondary outcomes (3)
- measure
- Recurrence-free survival (RFS)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 89 Years
Show eligibility criteria text
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
References
Publications (30)
- BACKGROUNDBanales JM, Cardinale V, Carpino G, Marzioni M, Andersen JB, Invernizzi P, Lind GE, Folseraas T, Forbes SJ, Fouassier L, Geier A, Calvisi DF, Mertens JC, Trauner M, Benedetti A, Maroni L, Vaquero J, Macias RI, Raggi C, Perugorria MJ, Gaudio E, Boberg KM, Marin JJ, Alvaro D. Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA). Nat Rev Gastroenterol Hepatol. 2016 May;13(5):261-80. doi: 10.1038/nrgastro.2016.51. Epub 2016 Apr 20. PMID 27095655
- BACKGROUNDSpolverato G, Kim Y, Ejaz A, Alexandrescu S, Marques H, Aldrighetti L, Gamblin TC, Pulitano C, Bauer TW, Shen F, Sandroussi C, Poultsides G, Maithel SK, Pawlik TM. Conditional Probability of Long-term Survival After Liver Resection for Intrahepatic Cholangiocarcinoma: A Multi-institutional Analysis of 535 Patients. JAMA Surg. 2015 Jun;150(6):538-45. doi: 10.1001/jamasurg.2015.0219. PMID 25831462
- BACKGROUNDBenson AB 3rd, Abrams TA, Ben-Josef E, Bloomston PM, Botha JF, Clary BM, Covey A, Curley SA, D'Angelica MI, Davila R, Ensminger WD, Gibbs JF, Laheru D, Malafa MP, Marrero J, Meranze SG, Mulvihill SJ, Park JO, Posey JA, Sachdev J, Salem R, Sigurdson ER, Sofocleous C, Vauthey JN, Venook AP, Goff LW, Yen Y, Zhu AX. NCCN clinical practice guidelines in oncology: hepatobiliary cancers. J Natl Compr Canc Netw. 2009 Apr;7(4):350-91. doi: 10.6004/jnccn.2009.0027. No abstract available. PMID 19406039
- BACKGROUNDEndo I, Gonen M, Yopp AC, Dalal KM, Zhou Q, Klimstra D, D'Angelica M, DeMatteo RP, Fong Y, Schwartz L, Kemeny N, O'Reilly E, Abou-Alfa GK, Shimada H, Blumgart LH, Jarnagin WR. Intrahepatic cholangiocarcinoma: rising frequency, improved survival, and determinants of outcome after resection. Ann Surg. 2008 Jul;248(1):84-96. doi: 10.1097/SLA.0b013e318176c4d3. PMID 18580211
- BACKGROUNDHyder O, Hatzaras I, Sotiropoulos GC, Paul A, Alexandrescu S, Marques H, Pulitano C, Barroso E, Clary BM, Aldrighetti L, Ferrone CR, Zhu AX, Bauer TW, Walters DM, Groeschl R, Gamblin TC, Marsh JW, Nguyen KT, Turley R, Popescu I, Hubert C, Meyer S, Choti MA, Gigot JF, Mentha G, Pawlik TM. Recurrence after operative management of intrahepatic cholangiocarcinoma. Surgery. 2013 Jun;153(6):811-8. doi: 10.1016/j.surg.2012.12.005. Epub 2013 Mar 15.