A Phase 2a Study of Modified Vaccinia Virus to Treat Sorafenib-naïve Advanced Liver Cancer

Phase 2

Completed

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).

Recruitment & Eligibility

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.

Arm && Interventions

Group	Intervention	Description
JX-594 recombinant vaccina GM-CSF	JX-594 recombinant vaccina GM-CSF	JX-594 recombinant vaccina GM-CSF

Primary Outcome Measures

Name	Time	Method
Number of Participants With Radiographic Response	CT scans every 6 week from Week 6 up to 12 months	Number of Participants with Complete response \[CR\] or partial response \[PR\] per modified Response Evaluation Criteria in Solid Tumors (mRECIST) for target and non-target lesions assessed by enhanced CT scan: CR, disappearance of intratumoral enhancing area; PR, \>=30% decrease in sum of diameters of enhancing area; Radiographic Response = CR + PR

Secondary Outcome Measures

Name	Time	Method
Overall Survival (OS)	CT scans every 6 week from Week 6 up to 12 months	OS was defined as the time from first dose of Pexa-Vec until death from any cause. For patients not known to have died at the time of the analysis, OS was censored on the date they were last known to be alive. OS was summarized and a KM curve was constructed.
Time to Progression (TTP)	CT scans every 6 week from Week 6 up to 12 months.	TTP (in months) was defined as the number of months from the date of first Pexa-Vec infusion to the date of disease progression. If the patient had no progression then TTP was censored at the date of last evaluable tumor assessment. TTP was summarized and a Kaplan Meier (KM) curve was constructed.

Locations (5): University of Texas Health Science Center at San Antonio
🇺🇸
San Antonio, Texas, United States
Pusan National University Hospital
🇰🇷
Pusan, Korea, Republic of
Pusan National University Yangsan Hospital
🇰🇷
Yangsan, Korea, Republic of
University Clinic of Navarra
🇪🇸
Pamplona, Navarra, Spain
Mayo Clinic
🇺🇸
Scottsdale, Arizona, United States