Clinical trial NCT01636284: A Phase 2a Study of Modified Vaccinia Virus to Treat Sorafenib-naïve Advanced Liver Cancer
Official title: A Single-Arm, Open-Label Phase 2 Study of JX 594 (Thymidine Kinase-Deactivated Vaccinia Virus Plus GM-CSF) Administered by Weekly Intravenous (IV) Infusions in Sorafenib-naïve Patients With Advanced Hepatocellular Carcinoma (HCC)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hepatocellular Carinoma
Keywords: Liver Cancer
HCC
first line HCC
Pexa-Vec
JX-594
Lead sponsor: SillaJen, Inc.
Sponsor class: INDUSTRY
First posted: 2012-07-10
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is to determine how effectively JX-594 (Pexa-Vec) will prolong life in patients with advanced Hepatocellular Carcinoma (HCC) who have not been previously treated with sorafenib, and the safe administration of JX-594 in five weekly IV infusions.

Detailed description: This was a Phase 2a, two-staged, single-arm, open-label study in sorafenib-naïve patients with advanced HCC.

Patients received 5 weekly IV infusions of Pexa-Vec and could have continued to receive IV infusions of Pexa-Vec every 3 weeks until progressive disease (PD).

Eligibility criteria: KEY Inclusion Criteria:

* Histologic or cytologic confirmation of advanced primary hepatocellular carcinoma (HCC)
* Measurable tumor (at least one tumor with ≥1 cm LD of contrast-enhancement during the arterial phase on CT scanning)
* ECOG performance status 0, 1 or 2
* Child-Pugh Class A; or Child-Pugh Class B7 without clinically significant ascites
* Platelet count ≥50,000 plts/mm3
* WBC count ≥2,000 cells/mm3 and ≤50,000 cells/mm3
* Hemoglobin ≥10 g/dL
* Adequate liver function

KEY Exclusion Criteria:

* Received sorafenib as previous treatment for HCC for more than 14 days
* History of severe exfoliative skin condition (e.g., eczema or atopic dermatitis requiring systemic therapy for \> 4 weeks)
* Prior treatment with JX-594
* Known significant immunodeficiency due to underlying illness (e.g. HIV/AIDS) and/or medication
* Severe or unstable cardiac disease
* Viable CNS malignancy associated with clinical symptoms
* Pregnant or nursing an infant
* Significant bleeding event within the last 12 months.

Primary outcomes:
measure: Number of Participants With Radiographic Response
timeFrame: CT scans every 6 week from Week 6 up to 12 months
Secondary outcomes:
measure: Time to Progression (TTP)
timeFrame: CT scans every 6 week from Week 6 up to 12 months.
measure: Overall Survival (OS)
timeFrame: CT scans every 6 week from Week 6 up to 12 months
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: San Antonio
country: United States
facility: University of Texas Health Science Center at San Antonio
state: Texas
city: Pusan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Yangsan
country: Korea, Republic of
facility: Pusan National University Yangsan Hospital
city: Pamplona
country: Spain
facility: University Clinic of Navarra
state: Navarra
Study officials:
affiliation: Jennerex Biotherapeutics
name: James Burke, MD
role: STUDY_DIRECTOR
References: