Trial Outcomes & Findings for A Study of Recombinant Vaccinia Virus to Treat Unresectable Primary Hepatocellular Carcinoma (NCT NCT00554372)

Last Updated: 2026-06-16

Results Overview

Proportion of subjects achieving disease control at 8 weeks based on a modified Response Evaluation Criteria in Solid Tumors v1.0 (mRECIST). Per mRECIST for target lesions as assessed by dynamic contrast enhanced dynamic MRI: Complete Response (CR), Disappearance of all tumor(s); Partial Response (PR), \>=30% decrease in the sum of longest diameter (LD) of tumor(s) taking as reference the baseline sum; Stable Disease (SD), any cases that do not qualify for PR or progressive disease (PD); PD, any increase of \>= 20% in the sum of longest diameter (LD) of tumor(s) taking as reference the baseline sum. Disease Control (DC) = CR or PR or SD. For mRECIST criteria, new tumor(s) that developed within the liver were measured (a new tumor was defined as a malignant tumor not present at baseline, was ≥ 1 cm in LD had typical hypervascular features of HCC). Their maximum diameter(s) were included in the sum of the maximum diameter; new tumors were not considered evidence for progression.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

Initial progression status and response assessment at 8 weeks from first dose

Results posted on

2026-06-16

Participant Flow

Subjects were randomized 1:1 to a treatment arm (dosage group). Randomization into the dosing groups was stratified by whether the Hepatocellular carcinoma was virally associated (hepatitis B virus or hepatitis C virus) or not virally associated.

Participant milestones

Participant milestones
Measure	Low Dose 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.
Overall Study STARTED	14	16
Overall Study COMPLETED	14	16
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Low Dose n=14 Participants 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose n=16 Participants 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	Total n=60 Participants Total of all reporting groups
Age, Continuous	67.1 years STANDARD_DEVIATION 11.5 • n=14 Participants	62.9 years STANDARD_DEVIATION 12.7 • n=16 Participants	64.9 years STANDARD_DEVIATION 12.2 • n=30 Participants
Sex: Female, Male Female	4 Participants n=14 Participants	3 Participants n=16 Participants	7 Participants n=30 Participants
Sex: Female, Male Male	10 Participants n=14 Participants	13 Participants n=16 Participants	23 Participants n=30 Participants
Region of Enrollment United States	5 participants n=14 Participants	5 participants n=16 Participants	10 participants n=30 Participants
Region of Enrollment Canada	3 participants n=14 Participants	4 participants n=16 Participants	7 participants n=30 Participants
Region of Enrollment South Korea	6 participants n=14 Participants	7 participants n=16 Participants	13 participants n=30 Participants

PRIMARY outcome

Timeframe: Initial progression status and response assessment at 8 weeks from first dose

Population: Patients having evaluable radiographic imaging, 2 patients in each arm were excluded due to unevaluable images, 1 patient in the low dose arm was excluded due to a protocol deviation

Outcome measures

Outcome measures
Measure	Low Dose n=13 Participants 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose n=15 Participants 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.
Proportion of Subjects Achieving Disease Control (Non-progressive Disease) at 8 Weeks After Initiation of Treatment	0.7273 Proportion of evaluable participants Interval 0.39 to 0.94	0.6429 Proportion of evaluable participants Interval 0.351 to 0.872

SECONDARY outcome

Timeframe: Safety and tolerability were evaluated throughout the 8 week period of study participation

Treatment-related serious adverse events in patients treated at two dose levels

Outcome measures

Outcome measures
Measure	Low Dose n=14 Participants 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose n=16 Participants 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.
Safety and Tolerability of JX-594 Administered at Two Dose Levels	1 serious adverse event	0 serious adverse event

SECONDARY outcome

Timeframe: At week 8

Number of subjects achieving disease control (non-progressive disease) at 8 weeks after treatment was initiated based on modified Response Evaluation Criteria in Solid Tumors for Hepatocellular Carcinoma (mRECIST for HCC). mRECIST for HCC adopted the concept of viable tumor as tumor tissue showing uptake in arterial phase of contrast enhanced radiologic imaging techniques. (see Lencioni and Llovet, Semin. Liver Dis. 2010; 30:52-60). Per mRECIST for HCC, for target lesions as assessed by contrast enhanced dynamic MRI: Complete Response (CR), Disappearance of any intratumoral arterial enhancement in all target (viable) lesions; Partial Response (PR), \>=30% decrease in the sum of diameters of viable target lesions; Stable Disease (SD), any cases that do not qualify for PR or progressive disease (PD); PD, any increase of \>= 20% in viable target lesions. Disease Control (DC) = CR or PR or SD.

Outcome measures

Outcome measures
Measure	Low Dose n=13 Participants 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose n=15 Participants 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.
Number of Subjects Achieving Disease Control as Determined Using Intrahepatic Modified RECIST Criteria	6 participants	7 participants

SECONDARY outcome

Timeframe: To 760 days post treatment

Overall survival after treatment in days

Outcome measures

Outcome measures
Measure	Low Dose n=13 Participants 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose n=16 Participants 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.
Median Overall Survival	202 days Interval 109.0 to 271.0	423 days Interval 265.0 to Upper bound was not achieved. The data analysis was done at a time when subjects in both dose groups were still alive.

Adverse Events

Low Dose

Serious events: 4 serious events

Other events: 14 other events

Deaths: 9 deaths

High Dose

Serious events: 4 serious events

Other events: 16 other events

Deaths: 9 deaths

Serious adverse events

Serious adverse events
Measure	Low Dose n=14 participants at risk 1e8 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.	High Dose n=16 participants at risk 1e9 pfu (plaque forming units) total dose of JX-594 (recombinant vaccinia virus) on each of three (3) treatment days (2 weeks apart). Each treatment dose was divided among 1-5 hepatic tumors; all tumors injected at day 1 were also injected at subsequent treatments.
Hepatobiliary disorders Bile duct Obstruction	0.00% 0/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	12.5% 2/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Hepatobiliary disorders Hyperbilirubinaemia	0.00% 0/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	6.2% 1/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Blood and lymphatic system disorders Anemia	7.1% 1/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	0.00% 0/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Cardiac disorders Angina pectoris	7.1% 1/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	0.00% 0/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Cardiac disorders Myocardial Infarction	7.1% 1/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	0.00% 0/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Gastrointestinal disorders Nausea	0.00% 0/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	6.2% 1/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Gastrointestinal disorders Vomiting	0.00% 0/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	6.2% 1/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Infections and infestations Bacteraemia	7.1% 1/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	0.00% 0/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Metabolism and nutrition disorders Hyponatraemia	0.00% 0/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	6.2% 1/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.
Nervous system disorders Hepatic Encephalopathy	7.1% 1/14 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.	0.00% 0/16 • Up to maximum 31 months (or Through study completion) Represents overall survival over the entire study follow-up period.

Other adverse events

Additional Information

Manager, IP, Contracts, Translational Research

SillaJen Biotherapeutics, Inc.

Phone: 415 281 8886

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60