A Study to Estimate Safety and Efficacy of Sorafenib (BAY43-9006) in the Treatment of Hepatocellular Carcinoma

Study Overview
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Brief Summary
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Evaluate anti-cancer activity (e.g. proportion of patients with confirmed complete response or partial response) in patients with advanced, inoperable biopsy-proven hepatocellular carcinoma.

Detailed Description
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In addition to the key secondary outcome parameters the following exploratory parameters were evaluated in subpopulations: Pharmacokinetics (PK) profile of Sorafenib Plasma and tissue tumor biomarkers

Official Title
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A Phase II Multicenter Uncontrolled Trial of Sorafenib (BAY43-9006) in Patients With Advanced Hepatocellular Carcinoma

Conditions
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Carcinoma, Hepatocellular

Intervention / Treatment
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* Drug: Sorafenib (Nexavar, BAY43-9006)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Histologically or cytologically confirmed primary hepatocellular carcinoma (HCC) Inoperable disease (T2-T4, any N, M0 or M1) or refused surgery Measurable disease At least 1 bidimensionally measurable lesion of at least 2 cm by computed tomography (CT) scan or magnetic resonance imaging (MRI) Presence of at least 1 of the following: Alpha-fetoprotein greater than the upper limit of normal (ULN) Hepatitis C antibody positive Hepatitis B surface antigen positive Child's Pugh class A or B Candidate for systemic therapy Exclusion Criteria: Fibrolamellar disease mixed histology Metastatic brain or meningeal tumors

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sorafenib 400 mg b.i.d.<br>Sorafenib (Nexavar, BAY43-9006) 400 mg administered bis in die (bid, twice a day) | Drug: Sorafenib (Nexavar, BAY43-9006)<br>* Sorafenib (Nexavar, BAY43-9006) 400 mg administered bis in die (bid, twice a day)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants for Each Type of Response | Objective response rate of sorafenib assessed as the proportion of subjects with confirmed complete or partial response as per modified World Health Organization (WHO) criteria. | Until 30 days after termination of active therapy | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Duration of Response | Duration of response was calculated from the first drug treatment date until documented progressive disease (PD). PD was 1) 25% or more increase in the sum of all target lesion areas taking as reference the smallest sum recorded at or following baseline, 2) unequivocal progression of an existing non-target lesion, or 3) appearance of a new lesion. | up to 3 years later | 
| Time to Response | Time from the first day of receiving study drug to the date the CR or PR was documented (with confirmation). | up to 3 years later | 
| Time to Progression | Time from the first date of receiving study drug until the first documented PD. | up to 3 years later | 
| Duration of Stable Disease | Time from the first day of receiving study drug until there was a documented PD or response. | up to 3 years later | 
| Time to Minor Response | Time from the first day of receiving study drug to the date the MR was first documented (with confirmation). Minor response = >25% regression. | up to 3 years later | 
| Duration of Minor Response | Time from the date that MR was first documented to the date that PD was first documented. | Time from MR to PD | 
| Overall Survival | Time from the first date of receiving study medication to death. | Start of treatment to death | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cancer, Liver Cancer, Hepatocellular carcinoma (HCC)