Cholangiocarcinoma, IntrahepaticNCT04195503
Liver Transplant for Stable, Advanced Intrahepatic Cholangiocarcinoma
A study testing Liver Transplantation in people with Cholangiocarcinoma, Intrahepatic, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
Diagnosis of intrahepatic cholangiocarcinoma (iCCA) is an increasingly common form of primary liver cancer, but patients with this diagnosis are not currently eligible for LT. This study proposes to offer LT as an option to patients with advanced, unresectable iCCA who demonstrate at least 6 months of disease… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cholangiocarcinoma, Intrahepatic
- What is being tested?
- Liver Transplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteCanada
- Study dates
- 10 Dec 2019to 31 Dec 2034
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, aged 18 years or older, at time of consent
- Eastern Cooperative Oncology Group (ECOG) score : 0 or 1 at all times from study entry to LT
- Able and willing to provide written informed consent.
- Women of child-bearing potential must agree to use adequate birth control
- Histologically-confirmed diagnosis of iCCa
- Disease is deemed unresectable based on tumor location or underlying liver dysfunction
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- No vascular invasion, extra-hepatic disease, or lymph node involvement detected on imaging
- Patients must have ≥ 6 months of disease stability or tumor regression on gemcitabine-based therapy. For patients who have progressed to second-line therapy, disease must also be controlled for ≥ 6 months on that regimen
- At least 1 living donor has stepped forward, is blood group compatible and has completed a "Health History form" which, upon review by the Living Donor team, is deemed acceptable and demonstrates no obvious contraindications to donation.
Who cannot join
- Previous or concurrent cancer (some exceptions permitted)
- 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
- Patients having received prior locoregional therapies or radiation to the tumor
- Renal dysfunction with an estimated creatinine clearance of less than 50 ml/min
- Known history of human immunodeficiency virus (HIV) infection.
- Prior history of solid organ or bone marrow transplantation
Show all 9Show fewer
- Substance abuse, medical, psychological or social conditions that may interfere with the patient's participation in the study or evaluation of the study results
- Any condition that is unstable or which could jeopardize the safety of the patient and his/her compliance in the study
- Pregnant or breast-feeding patients (unless willing to abstain from breast-feeding)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Liver Transplantation in Locally- Advanced, Unresectable, Non-Metastatic Intrahepatic Cholangiocarcinoma Treated With Neoadjuvant Systemic Therapy: A Single-centre, Pilot Study
Diagnosis of intrahepatic cholangiocarcinoma (iCCA) is an increasingly common form of primary liver cancer, but patients with this diagnosis are not currently eligible for LT. This study proposes to offer LT as an option to patients with advanced, unresectable iCCA who demonstrate at least 6 months of disease stability /regression while receiving standard chemotherapy. Patients will be referred and screened for study participation if they have achieved 6 months of disease stability.
They will be worked up for transplant and if eligible, will be listed. Disease must remain controlled while awaiting transplantation. After transplantation, patients will be followed for disease recurrence and overall survival, while receiving standard post-transplantation followup.
Read the detailed description
Patients treated with standard of care chemotherapy for unresectable, biopsy-proven intrahepatic cholangiocarcinoma, and who demonstrate stability/regression of disease for a minimum of 6 months, may be eligible for study. Following informed consent and screening, eligible patients with a potential live liver donor (an interested, ABO-compatible individual, without obvious contraindications for donation) will be evaluated for transplantation. During this period, patients will continue to receive chemotherapy and imaging at the usual intervals. Once a patient completes the pre-transplant assessments and is deemed "suitable for transplantation" , the living donor's assessments may begin. If the donor is found suitable, a tentative date will be set for the surgeries. Systemic chemotherapy will be stopped approximately 4 weeks before the surgery date.
At that point, the potential recipient will be reimaged (CT scan). Test results, and inclusion and exclusion criteria will be reviewed to ensure the participant remains eligible to proceed to transplant. If these scans reveal any suspicion of tumor infiltration to surrounding tissues, an exploratory laparotomy or endoscopic ultrasound procedure will be scheduled to allow for visual examination and sampling of those tissues. Tissue samples, such as perihilar fat and/or lymph nodes, will be sent to the pathology department for urgent review. If extrahepatic disease is identified, the participant will be excluded from the study and the transplant will not be performed. Any excluded participants will continue to be followed for the study as "drop outs".
Otherwise, the donor and recipient surgeries will be performed in the standard fashion and with the preferred technique of the surgeon. The patient and donor will receive standard of care post-operative care.
Participants (recipients) will be monitored closely for evidence of tumor recurrence with imaging and blood tests. Results of these tests will be collected until completion of the study follow-up period (5-years after LT). In addition to tumor surveillance, liver transplant status will be assessed at each study timepoint (including graft loss, rejection, liver function tests, and details of immunosuppression). The study will also collect details of any biopsies performed, re-hospitalizations and surgical complications
- Conditions
- Cholangiocarcinoma, Intrahepatic
- Being tested
- Liver Transplantation
- Medical subject headings
- Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liver TransplantationProcedure or surgery
Surgical removal of liver containing tumor(s) and transplantation of new healthy liver graft
| Study group | Role | What they receive |
|---|---|---|
| liver transplantation | Other | Surgical Intervention - Liver transplantation Receives: Procedure: Liver Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
patient survival
Measured: 5 years
Secondary outcomes (2)
disease-free survival
Measured: 5 years
patient survival
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2019
- Study start10 Dec 2019
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cholangiocarcinoma, IntrahepaticStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gonzalo Sapisochin, MD, Principal investigator, UHN
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04195503
- Sponsor's protocol ID
- 19-5581
- Status last verified
- Sep 2026
- First submitted
- 9 Dec 2019
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lunsford KE, Javle M, Heyne K, Shroff RT, Abdel-Wahab R, Gupta N, Mobley CM, Saharia A, Victor DW, Nguyen DT, Graviss EA, Kaseb AO, McFadden RS, Aloia TA, Conrad C, Li XC, Monsour HP, Gaber AO, Vauthey JN, Ghobrial RM; Methodist-MD Anderson Joint Cholangiocarcinoma Collaborative Committee (MMAJCCC). Liver transplantation for locally advanced intrahepatic cholangiocarcinoma treated with neoadjuvant therapy: a prospective case-series. Lancet Gastroenterol Hepatol. 2018 May;3(5):337-348. doi: 10.1016/S2468-1253(18)30045-1. Epub 2018 Mar 13.
PubMed 29548617Blair AB, Alobuia WM, Palta M, Raman SS, Levine MH, Benson AB 3rd, D'Angelica MI, Cloyd JM. Locoregional Treatment Options for Locally Advanced Intrahepatic Cholangiocarcinoma. J Natl Compr Canc Netw. 2025 Aug 14;23(9):e257085. doi: 10.6004/jnccn.2025.7085.
PubMed 40812353Achurra P, Fernandes E, O'Kane G, Grant R, Cattral M, Sapisochin G. Liver transplantation for intrahepatic cholangiocarcinoma: who, when and how. Curr Opin Organ Transplant. 2024 Apr 1;29(2):161-171. doi: 10.1097/MOT.0000000000001136. Epub 2024 Jan 23.
PubMed 38258823
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Liver Transplantation in Locally- Advanced, Unresectable, Non-Metastatic Intrahepatic Cholangiocarcinoma Treated With Neoadjuvant Systemic Therapy: A Single-centre, Pilot Study
- ClinicalTrials.gov ID
- NCT04195503
- Acronym / sponsor ID
- 19-5581
- Conditions
- Cholangiocarcinoma, Intrahepatic
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- 10 Dec 2019 (actual)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2034 (estimated)
- First posted
- 12 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Diagnosis of intrahepatic cholangiocarcinoma (iCCA) is an increasingly common form of primary liver cancer, but patients with this diagnosis are not currently eligible for LT. This study proposes to offer LT as an option to patients with advanced, unresectable iCCA who demonstrate at least 6 months of disease stability /regression while receiving standard chemotherapy. Patients will be referred and screened for study participation if they have achieved 6 months of disease stability.
They will be worked up for transplant and if eligible, will be listed. Disease must remain controlled while awaiting transplantation. After transplantation, patients will be followed for disease recurrence and overall survival, while receiving standard post-transplantation followup.
Detailed description
Patients treated with standard of care chemotherapy for unresectable, biopsy-proven intrahepatic cholangiocarcinoma, and who demonstrate stability/regression of disease for a minimum of 6 months, may be eligible for study. Following informed consent and screening, eligible patients with a potential live liver donor (an interested, ABO-compatible individual, without obvious contraindications for donation) will be evaluated for transplantation. During this period, patients will continue to receive chemotherapy and imaging at the usual intervals. Once a patient completes the pre-transplant assessments and is deemed "suitable for transplantation" , the living donor's assessments may begin. If the donor is found suitable, a tentative date will be set for the surgeries. Systemic chemotherapy will be stopped approximately 4 weeks before the surgery date.
At that point, the potential recipient will be reimaged (CT scan). Test results, and inclusion and exclusion criteria will be reviewed to ensure the participant remains eligible to proceed to transplant. If these scans reveal any suspicion of tumor infiltration to surrounding tissues, an exploratory laparotomy or endoscopic ultrasound procedure will be scheduled to allow for visual examination and sampling of those tissues. Tissue samples, such as perihilar fat and/or lymph nodes, will be sent to the pathology department for urgent review. If extrahepatic disease is identified, the participant will be excluded from the study and the transplant will not be performed. Any excluded participants will continue to be followed for the study as "drop outs".
Otherwise, the donor and recipient surgeries will be performed in the standard fashion and with the preferred technique of the surgeon. The patient and donor will receive standard of care post-operative care.
Participants (recipients) will be monitored closely for evidence of tumor recurrence with imaging and blood tests. Results of these tests will be collected until completion of the study follow-up period (5-years after LT). In addition to tumor surveillance, liver transplant status will be assessed at each study timepoint (including graft loss, rejection, liver function tests, and details of immunosuppression). The study will also collect details of any biopsies performed, re-hospitalizations and surgical complications
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| liver transplantation | Other | Procedure: Liver TransplantationSurgical Intervention - Liver transplantation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female, aged 18 years or older, at time of consent
- Eastern Cooperative Oncology Group (ECOG) score : 0 or 1 at all times from study entry to LT
- Able and willing to provide written informed consent.
- Women of child-bearing potential must agree to use adequate birth control
- Histologically-confirmed diagnosis of iCCa
Key exclusion criteria (9 listed)
- Previous or concurrent cancer (some exceptions permitted)
- 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
- Patients having received prior locoregional therapies or radiation to the tumor
- Renal dysfunction with an estimated creatinine clearance of less than 50 ml/min
- Known history of human immunodeficiency virus (HIV) infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Liver TransplantationSurgical removal of liver containing tumor(s) and transplantation of new healthy liver graft | Procedure or surgery | liver transplantation |
Primary outcome
patient survival
Time frame: 5 years
2 secondary outcomes
disease-free survival
Time frame: 5 years
patient survival
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
- Background: PubMed 29548617Lunsford KE, Javle M, Heyne K, Shroff RT, Abdel-Wahab R, Gupta N, Mobley CM, Saharia A, Victor DW, Nguyen DT, Graviss EA, Kaseb AO, McFadden RS, Aloia TA, Conrad C, Li XC, Monsour HP, Gaber AO, Vauthey JN, Ghobrial RM; Methodist-MD Anderson Joint Cholangiocarcinoma Collaborative Committee (MMAJCCC). Liver transplantation for locally advanced intrahepatic cholangiocarcinoma treated with neoadjuvant therapy: a prospective case-series. Lancet Gastroenterol Hepatol. 2018 May;3(5):337-348. doi: 10.1016/S2468-1253(18)30045-1. Epub 2018 Mar 13.
- Derived from this study: PubMed 40812353Blair AB, Alobuia WM, Palta M, Raman SS, Levine MH, Benson AB 3rd, D'Angelica MI, Cloyd JM. Locoregional Treatment Options for Locally Advanced Intrahepatic Cholangiocarcinoma. J Natl Compr Canc Netw. 2025 Aug 14;23(9):e257085. doi: 10.6004/jnccn.2025.7085.
- Derived from this study: PubMed 38258823Achurra P, Fernandes E, O'Kane G, Grant R, Cattral M, Sapisochin G. Liver transplantation for intrahepatic cholangiocarcinoma: who, when and how. Curr Opin Organ Transplant. 2024 Apr 1;29(2):161-171. doi: 10.1097/MOT.0000000000001136. Epub 2024 Jan 23.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Gonzalo Sapisochin, MD principal investigator
UHN
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Liver Transplantation in people with Cholangiocarcinoma, Intrahepatic, run by University Health Network, Toronto at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
patient survival (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04195503, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.