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Pilot Study Sorafenib as Bridge to Orthotopic Liver Transplantation (OLT)

Early Phase 1
Terminated
Conditions
Carcinoma, Hepatocellular
Interventions
Registration Number
NCT01337492
Lead Sponsor
Case Comprehensive Cancer Center
Brief Summary

Patients with either histological confirmation or clinical diagnosis who are candidate for orthotopic liver transplant(OLT) will receive Sorafenib 400mg 2 x day (BID) as a neoadjuvant (bridging) treatment until transplant. If patient progresses or falls outside of the Milan criteria on sorafenib patient can be treated per physician's discretion using local therapy. Patient will be followed until 30 days after the orthotopic liver transplant (OLT).

Detailed Description

Primary objective:

• To evaluate safety (including possible surgical complication) and feasibility of using sorafenib as neo-adjuvant (bridging therapy) prior to orthotopic liver transplant(OLT).

The primary goal of this pilot study is to assess the safety and feasibility of treating HCC patients with sorafenib prior to OLT, as determined by the number of patients experiencing grade 4 hemorrhaging or bleeding post-operatively.

To achieve these goals a 2-stage accrual design will be used with a maximum accrual goal of 10 patients. Initially 5 patients will be entered on-study. If more than one patient experiences grade 4 hemorrhaging/bleeding following OLT the trial will be closed and sorafenib will not be considered safe and/or feasible. If no patient experiences this event 5 additional patients will be enrolled. At the end of the study sorafenib will be considered safe and feasible in this setting if no patient experience grade 4 hemorrhaging/bleeding following transplant. With this design, and assuming at least 3 of the 5 patients in each cohort undergo OLT, the likelihood of stopping early is \<.04 if the underlying risk hemorrhages/bleeding are low (\<5% each) whereas there is a \>.42 chance of early termination if the risks are substantial (e.g. \>.20 likelihood of dose limiting toxicity and \>.30 likelihood of \>grade 4 hemorrhages/bleeding). Overall the likelihood of accepting or not accepting sorafenib as safe and feasible are \>.84, and \<.16, respectively under these conditions.

Secondary objectives:

* Drop out rate

* Time to progression prior to orthotopic liver transplant (OLT)

* Waiting time.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
2
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
SorafenibNexavar-
Primary Outcome Measures
NameTimeMethod
Adverse events associated with technical aspects of the operationpost-transplant

To assess the safety and feasibility of treating HCC patients with sorafenib prior to OLT, as determined by the number of patients experiencing grade 4 hemorrhaging needing blood trnasfusion, thrombosis of vascular graft structures, stenosis of vascular anastomosis or biliary leaks or anastomotic biliary stenosis.

Secondary Outcome Measures
NameTimeMethod
Time to progression prior to OLT10 - 12 months

A measure of time after being put on transplant list until the disease is beyond Milan's criteria prior to OLT.

Drop Out Rate10-12 months

This is the rate of subjects who were eligible for liver transplant but drop out secondary to disease progression while awaiting donor organ.

Waiting Time10-12 months

This is number of months from the time patient get listed on the transplant list to the time patient under goes actual liver transplant.

Trial Locations

Locations (1)

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

🇺🇸

Cleveland, Ohio, United States

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