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Erlotinib in Treating Patients With Liver Cancer That Cannot be Surgically Removed

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00047333
Recruitment Status : Terminated (Administratively complete.)
First Posted : January 27, 2003
Last Update Posted : January 23, 2013
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Brief Summary:
Phase II trial to study the effectiveness of erlotinib in treating patients who have liver cancer that cannot be surgically removed. Erlotinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth

Condition or disease Intervention/treatment Phase
Adult Primary Hepatocellular Carcinoma Advanced Adult Primary Liver Cancer Localized Unresectable Adult Primary Liver Cancer Drug: erlotinib hydrochloride Other: laboratory biomarker analysis Other: pharmacological study Phase 2

Detailed Description:


I. To assess progression-free survival (PFS) measured at 16 weeks following initiation of once daily continuous oral therapy with OSI-774 in patients with unresectable hepatocellular carcinoma.


I. To assess objective response rate, rate and duration of stable disease, time to progression, median and overall survival in this patient population, and any changes in tumor perfusion based on functional CT imaging.

II. To correlate response with patient characteristics including: age, disease stage (TNM, Okuda [6]), viral hepatitis status, pathologic grade of cirrhosis, Childs-Pugh status, Performance Status, serum values of: alpha feto-protein, bilirubin, transaminases, albumin; EGFR expression score by IHC; and development of skin rash during therapy.

III. To determine the pharmacokinetic and pharmacodynamic profile of OSI-774 in this patient population.

IV. To determine the safety and tolerability of OSI-774 in this patient population.

OUTLINE: Patients are stratified according to epidermal growth factor receptor expression (low, 0-1+ vs high, 2-3+).

Patients receive oral erlotinib once daily. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Patients are followed every 3 months.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 80 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-Label Study Of OSI-774 (NSC 718781) In Unresectable Hepatocellular Carcinoma
Study Start Date : August 2002
Actual Primary Completion Date : January 2006

Arm Intervention/treatment
Experimental: Treatment (erlotinib hydrochloride)
Patients receive oral erlotinib once daily. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Drug: erlotinib hydrochloride
Given PO
Other Names:
  • CP-358,774
  • erlotinib
  • OSI-774

Other: laboratory biomarker analysis
Correlative studies

Other: pharmacological study
Correlative studies
Other Name: pharmacological studies

Primary Outcome Measures :
  1. Progression-free survival [ Time Frame: Time from initiation of therapy until documented disease progression, assessed at 16 weeks ]
    Summarized by Kaplan-Meier curves, from which medians and progression-free survival can be attained.

Secondary Outcome Measures :
  1. Objective response rate [ Time Frame: Up to 4 years ]
    Summarized by estimates and standard errors.

  2. Rate of stable disease [ Time Frame: Up to 4 years ]
    Summarized by estimates and standard errors.

  3. Duration of stable disease [ Time Frame: From the start of the treatment until the criteria for progression are met, assessed up to 4 years ]
    Summarized by Kaplan-Meier curves, from which medians and overall duration of stable disease can be attained.

  4. Time to progression [ Time Frame: Up to 4 years ]
    Summarized by Kaplan-Meier curves, from which medians and time to progression can be attained.

  5. Overall survival [ Time Frame: Up to 4 years ]
    Summarized by Kaplan-Meier curves, from which medians and overall survival can be attained.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Histologically confirmed hepatocellular carcinoma (HCC)not amenable to curative resection

    • No fibrolamellar HCC
  • No prior therapy for HCC, including systemic chemotherapy, hepatic arterial infusion of chemotherapeutic agents or irradiated microspheres, and epidermal growth factor receptor-targeting agents

    • The following prior therapies are allowed provided previously treated lesions remain separate from those to be evaluated in present study

      • Surgery
      • Liver-directed therapy (e.g., radiofrequency ablation, transarterial embolization/chemoembolization, or percutaneous ethanol injection)
  • At least 1 unidimensionally measurable lesion

    • At least 20 mm by conventional techniques
  • Must have paraffin tissue block or unstained slides from biopsy or surgical specimen
  • No known brain metastases
  • No ascites that are refractory to conservative management (e.g., sodium restriction to 50 mEq/day dietary sodium and fluid restrictions and/or diuretics)
  • Performance status - ECOG 0-2
  • At least 16 weeks
  • Granulocyte count at least 1,500/mm^3
  • Platelet count at least 60,000/mm^3
  • Hemoglobin at least 10 g/dL
  • Bilirubin no greater than 1.8 mg/dL
  • Albumin at least 2.5 g/dL
  • AST/ALT no greater than 5 times upper limit of normal
  • PT no greater than 1-3 seconds over normal
  • No decompensated liver disease
  • No jaundice
  • No portosystemic encephalopathy (evidenced by confusion, asterixis, significant sleep disturbance, or hypothermia less than 36º Celsius)
  • No hyponatremia with sodium less than 125 mEq/L
  • No portal hypertension with bleeding esophageal or gastric varices within the past 3 months
  • Creatinine no greater than 2 mg/dL
  • No symptomatic congestive heart failure
  • No unstable angina pectoris
  • No cardiac arrhythmia
  • No gastrointestinal tract disease resulting in an inability to take oral medication or requirement for IV alimentation
  • No active peptic ulcer disease
  • No abnormalities of the cornea (e.g., dry eye syndrome or Sjögren's syndrome)
  • No congenital abnormality (e.g., Fuch's dystrophy)
  • No other uncontrolled concurrent illness that would preclude study participation
  • No ongoing or active infection
  • No psychiatric illness or social situation that would preclude study compliance
  • No other malignancy within the past 5 years except nonmelanoma skin cancer
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No prior surgical therapy affecting absorption
  • More than 30 days since prior investigational agents
  • No concurrent commercial or other investigational anticancer agents or therapies
  • No concurrent combination antiretroviral therapy for HIV-positive patients

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00047333

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United States, Texas
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
National Cancer Institute (NCI)
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Principal Investigator: Melanie Thomas M.D. Anderson Cancer Center
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Responsible Party: National Cancer Institute (NCI) Identifier: NCT00047333    
Other Study ID Numbers: NCI-2012-02498
CDR0000257665 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: January 23, 2013
Last Verified: January 2013
Additional relevant MeSH terms:
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Carcinoma, Hepatocellular
Liver Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases
Erlotinib Hydrochloride
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action