Hepatocellular Carcinoma (HCC)_Torisel_

Study Overview
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Brief Summary
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This is a phase I/II study to evaluate dose limited toxicity and efficacy of Torisel

Official Title
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Phase I/II Study of Temsirolimus (Torisel®) as Novel Therapeutic Drug for Patients With Unresectable Hepatocellular Carcinoma (HCC)- A Correlative Study With Stathmin Over-expression

Conditions
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Inoperable HCC

Intervention / Treatment
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* Drug: Torisel


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Histologically confirmed hepatocellular carcinoma that is not amenable to curative resection measurable disease Age >=18 years. Life expectancy of greater than 12 weeks. ECOG performance status <= 2 Prior systemic therapy for HCC is allowed Adequate haematologic, renal and hepatic function Absence of cirrhosis or Child's A cirrhosis Fasting total cholesterol <9.1 mmol/liter and fasting triglyceride level <4.5 mmol/liter) Exclusion Criteria: Patients who have had systemic therapy or radiotherapy within 3 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 3 weeks earlier. Patients receiving any other investigational agents concurrently. Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events. Uncontrolled intercurrent diseases such as, but not limited to, ongoing or active infection, or psychiatric illness/social situations that would limit compliance with study requirements. Pregnant women.

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Torisel<br> | Drug: Torisel<br>* Torisel will be administered intravenously as an IV infusion over a 30-minute period in adult clinical studies.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| • To establish the maximum tolerated dose (MTD) and a suitable dose for Phase II evaluation of Torisel® given as a weekly dose in patients with advanced hepatocellular carcinoma (HCC) |  | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| • To determine and establish the safety and toxicity profile of Torisel® and to identify any dose limiting toxicities (DLTs) in advanced HCC |  | 2 Years | 
| • To determine progression-free survival (PFS) in patients with advanced hepatocellular carcinoma (HCC) treated with Torisel® |  | 4 Years | 
| • To correlate stathmin expression in pre-treatment HCC tumour biopsies and clinical response to Torisel® |  | 2 years | 
| • To determine the response rate (CR and PR) based on RECIST criteria |  | 2 years | 
| • To determine clinical benefit rate (CBR, percent of patients experiencing CR, PR or SD ≥ 12 weeks) |  | 2 years | 
| • To determine duration of response (DR) |  | 2 years | 
| • To determine overall survival (OS) |  | 4 years |