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A Study of Recombinant Vaccinia Virus Prior to Sorafenib to Treat Unresectable Primary Hepatocellular Carcinoma

This study has been completed.
Information provided by (Responsible Party):
SillaJen, Inc. ( Jennerex Biotherapeutics ) Identifier:
First received: July 26, 2010
Last updated: January 19, 2016
Last verified: June 2011
The purpose of this pilot safety study is to evaluate the safety and tolerability of JX-594 (Pexa-Vec) administered intravenously and intratumorally prior to standard sorafenib therapy.

Condition Intervention Phase
Carcinoma, Hepatocellular Drug: JX-594 followed by sorafenib Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-Label Pilot Safety Study of JX-594 (Vaccinia GM-CSF/Thymidine Kinase-Deactivated Virus) Administered by IV Infusion Followed by Intratumoral Injection Prior to Standard Sorafenib Treatment in Patients With Unresectable Primary Hepatocellular Carcinoma

Resource links provided by NLM:

Further study details as provided by SillaJen, Inc. ( Jennerex Biotherapeutics ):

Primary Outcome Measures:
  • Determine safety and tolerability of intravenous infusion of JX-594 followed by intratumoral injections with JX-594 prior to standard sorafenib therapy [ Time Frame: Safety evaluations through 28 days after last dose of JX-594 ]
    Adverse events will be collected and assessed to assess safety and tolerability through 28 days after last dose of JX-594 (or until all events considered probably or possibly related to JX-594 have resolved, stabilized, or returned to baseline status).

Secondary Outcome Measures:
  • Determine Disease Control Rate (DCR) at 12 weeks [ Time Frame: Disease control and response assessment at 12 weeks from first JX-594 dose ]
    DCR: confirmed complete response, partial response or stable disease based on modified RECIST and/or Choi response criteria

  • Determine radiographic response rate [ Time Frame: Periodically throughout study participation (average of up to 1 year) ]
    Response rate evaluation based on modified RECIST and/or Choi response criteria

  • Determine overall survival time [ Time Frame: Ongoing (average of 1 year) ]

Enrollment: 25
Study Start Date: August 2009
Study Completion Date: December 2015
Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: JX-594 followed by sorafenib
1e9 pfu (plaque-forming units) total JX-594 dose on each of up to four (4) JX-594 treatment days. Sorafenib is initiated after 3 JX-594 treatments and briefly interrupted if an optional 4th JX-594 treatment is given.
Drug: JX-594 followed by sorafenib
Patients will receive a total dose of 1e9 per treatment starting with one IV dose on Day 1 and injected intratumorally in 1-5 intrahepatic tumors on Day 8 and 22. Starting on Day 25 (3 days after the final JX-594 dose) patients will initiate oral sorafenib therapy twice daily according to standard approved guidelines. An optional maintenance JX-594 dose may be given intratumorally at Week 12 (sorafenib briefly interrupted).


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

Inclusion Criteria:

  • Histological confirmation or clinical/laboratory diagnosis of primary hepatocellular carcinoma (HCC)
  • Cancer is not surgically resectable for cure
  • Child Pugh A or B
  • Performance Score: KPS score of ≥ 70
  • Platelet count ≥ 50,000 plts/mm3
  • Total bilirubin ≤ 2.5 x ULN
  • AST, ALT < 5.0 x ULN
  • Acceptable coagulation status: INR ≤ 1.5 x ULN
  • Acceptable kidney function: Serum creatinine < 2.0 mg/dL
  • Sorafenib naive or refractory to sorafenib therapy Tumor Status: At least one intrahepatic tumor, and at least ≥50% of the total intrahepatic viable tumor mass, measurable by CT and injectable under imaging-guidance (note: injected and/or viable tumors must be previously untreated or ≥20% increase in size since preceding local-regional treatment).

Exclusion Criteria:

  • Known contraindications to sorafenib
  • Pregnant or nursing an infant
  • Significant immunodeficiency due to underlying illness (e.g. hematological malignancies, congenital immunodeficiencies and/or HIV infection/AIDS) and/or medication (e.g. high-dose systemic corticosteroids)
  • History of exfoliative skin condition (e.g. severe eczema, ectopic dermatitis, or similar skin disorder) that at some stage has required systemic therapy
  • Clinically significant and/or rapidly accumulating ascites, peri-cardial and/or pleural effusions
  • Severe or unstable cardiac disease
  • Current, known CNS malignancy
  • Use of anti-platelet or anti-coagulation medication
  • Use of the following anti-viral agents: ribavirin, adefovir, cidofovir (within 7 days prior to the first treatment), and PEG-IFN (within 14 days prior to the first treatment).
  • Patients with household contacts who meet any of these criteria unless alternate living arrangements can be made during the patient's active dosing period and for 7 days following the last dose of study medication:
  • Pregnant or nursing an infant
  • Children < 12 months old
  • History of exfoliative skin condition that at some stage has required systemic therapy
  • Significant immunodeficiency due to underlying illness (e.g. HIV/AIDS) and/or medication
  Contacts and Locations
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Please refer to this study by its identifier: NCT01171651

Korea, Republic of
Pusan National University Hospital
Busan, Korea, Republic of
Pusan National University Yangsan Hospital
Yangsan, Korea, Republic of
Sponsors and Collaborators
Jennerex Biotherapeutics
Study Director: David H Kirn, MD Jennerex Biotherapeutics
  More Information

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Jennerex Biotherapeutics Identifier: NCT01171651     History of Changes
Other Study ID Numbers: JX594-HEP016
Study First Received: July 26, 2010
Last Updated: January 19, 2016

Keywords provided by SillaJen, Inc. ( Jennerex Biotherapeutics ):
Vaccinia Virus
Primary Hepatocellular Carcinoma
Primary Liver Cancer
Liver Cancer

Additional relevant MeSH terms:
Carcinoma, Hepatocellular
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Liver Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Liver Diseases
Poxviridae Infections
DNA Virus Infections
Virus Diseases
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Vitamin B Complex
Growth Substances
Physiological Effects of Drugs processed this record on September 19, 2017