Trial Outcomes & Findings for A Study of Recombinant Vaccinia Virus to Treat Malignant Melanoma (NCT NCT00429312)
NCT ID: NCT00429312
Last Updated: 2026-07-27
Results Overview
Response was evaluated at the participant level based on the sum of the longest diameters of the injected target tumors. Tumor assessments were performed using RECIST criteria. Complete response (CR): all lesions disappear; Partial response (PR): maximum diameter decrease of \> 30% of the longest diameter (LD) of target lesions using the baseline LD; Progressive disease (PD): increase of \>20% in the sum of LD target lesions using the smallest sum LD recorded since treatment started; Stable disease (SD): unable to categorize as PR or PD using as reference the lobe with the smallest diameter at baseline.
COMPLETED
PHASE1/PHASE2
10 participants
Initial response assessment after six weeks (Day 43)
2026-07-27
Participant Flow
Ten evaluable patients with metastatic or locally invasive, unresectable Stage 3 or Stage 4 malignant melanoma were enrolled. Patients were enrolled and treated at the Billings Clinic, the Cancer Center of the Carolinas, or University of California, Los Angeles (UCLA).
This study was a Phase 1/2, open-label, non-comparative trial, so there are no specific pre-assignment, run-in, or washout periods mentioned.
Participant milestones
| Measure |
Recombinant Vaccinia Virus JX-594
All patients were treated with a dose 1 x 10\^8 plaque forming units (pfu) of Recombinant Vaccinia virus JX-594 administered by intratumoral (IT) injection weekly for a total of 6 treatments given over 6 weeks.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
5
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Recombinant Vaccinia Virus JX-594
All patients were treated with a dose 1 x 10\^8 plaque forming units (pfu) of Recombinant Vaccinia virus JX-594 administered by intratumoral (IT) injection weekly for a total of 6 treatments given over 6 weeks.
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|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Incomplete Efficacy Evaluation
|
3
|
Baseline Characteristics
A Study of Recombinant Vaccinia Virus to Treat Malignant Melanoma
Baseline characteristics by cohort
| Measure |
Recombinant Vaccinia Virus JX-594
n=10 Participants
Intratumoral injection(s) of Recombinant Vaccinia GM-CSF, JX-594
JX-594: Thymidine kinase-deleted vaccinia virus plus GM-CSF
|
|---|---|
|
Age, Continuous
|
65.1 years
STANDARD_DEVIATION 11.2 • n=9 Participants
|
|
Race/Ethnicity, Customized
White, Not Hispanic/Latino
|
8 participants
n=9 Participants
|
|
Race/Ethnicity, Customized
Hispanic/Latino
|
2 participants
n=9 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=9 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=9 Participants
|
PRIMARY outcome
Timeframe: Initial response assessment after six weeks (Day 43)Population: Patients who received JX-594 injections according to protocol and were evaluated one week after final injection.
Response was evaluated at the participant level based on the sum of the longest diameters of the injected target tumors. Tumor assessments were performed using RECIST criteria. Complete response (CR): all lesions disappear; Partial response (PR): maximum diameter decrease of \> 30% of the longest diameter (LD) of target lesions using the baseline LD; Progressive disease (PD): increase of \>20% in the sum of LD target lesions using the smallest sum LD recorded since treatment started; Stable disease (SD): unable to categorize as PR or PD using as reference the lobe with the smallest diameter at baseline.
Outcome measures
| Measure |
Recombinant Vaccinia Virus JX-594
n=5 Participants
Intratumoral injection(s) of Recombinant Vaccinia virus JX-594 (Thymidine kinase-inactivated vaccinia virus expressing human GM-CSF)
|
|---|---|
|
Number of Participants With Objective Response in Injected Tumor(s)
|
5 Participants with Stable disease
|
SECONDARY outcome
Timeframe: Up to Day 64Safety was determined by the incidence of treatment-related adverse events (AEs), treatment-related serious adverse events (SAEs), and clinically-significant changes from baseline in routine laboratory parameters. Severity was determined by NCI-CTCAE version 3.0.
Outcome measures
| Measure |
Recombinant Vaccinia Virus JX-594
n=10 Participants
Intratumoral injection(s) of Recombinant Vaccinia virus JX-594 (Thymidine kinase-inactivated vaccinia virus expressing human GM-CSF)
|
|---|---|
|
Incidence of Treatment-related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Treatment-related Adverse Events (AEs)
|
7 Participants
|
|
Incidence of Treatment-related Adverse Events (AEs) and Serious Adverse Events (SAEs)
Treatment-related Serious Adverse Events (SAEs)
|
0 Participants
|
SECONDARY outcome
Timeframe: Initial response assessment after six weeks (Day 43)Overall response was defined as the best response recorded from the start of the treatment until disease progression or recurrence across the entire disease burden (both injected and non-injected tumors), evaluated using RECIST criteria (Complete Response \[CR\], Partial Response \[PR\], Stable Disease \[SD\], or Progressive Disease \[PD\]).
Outcome measures
| Measure |
Recombinant Vaccinia Virus JX-594
n=5 Participants
Intratumoral injection(s) of Recombinant Vaccinia virus JX-594 (Thymidine kinase-inactivated vaccinia virus expressing human GM-CSF)
|
|---|---|
|
Best Overall Response for Entire Disease Burden (RECIST Criteria)
Completed Response (CR)
|
0 Participants
|
|
Best Overall Response for Entire Disease Burden (RECIST Criteria)
Partial Response (PR)
|
0 Participants
|
|
Best Overall Response for Entire Disease Burden (RECIST Criteria)
Stable Disease (SD)
|
3 Participants
|
|
Best Overall Response for Entire Disease Burden (RECIST Criteria)
Progressive Disease (PD)
|
2 Participants
|
SECONDARY outcome
Timeframe: From first study treatment until objective evidence of disease progression or death, up to 20.5 monthsPopulation: The Progression-Free Survival (PFS) analysis was conducted on the efficacy evaluable population, which consisted of 5 out of the 10 enrolled patients. The remaining 5 patients were excluded from the efficacy evaluable population due to protocol-specified reasons: incomplete efficacy evaluation (no CT scan) (n=1); efficacy assessments performed \>28 days beyond the protocol window (n=2); and early study withdrawal prior to the Day 43 efficacy evaluation (n=2).
Progression-free survival was defined as the time from the first study treatment until objective evidence of disease progression or death.
Outcome measures
| Measure |
Recombinant Vaccinia Virus JX-594
n=5 Participants
Intratumoral injection(s) of Recombinant Vaccinia virus JX-594 (Thymidine kinase-inactivated vaccinia virus expressing human GM-CSF)
|
|---|---|
|
Progression-free Survival
|
NA months
Median PFS and 95% confidence intervals could not be calculated because only 5 of the 10 enrolled patients were evaluable for efficacy assessments. Furthermore, for these 5 evaluable patients, post-treatment follow-up tumor measurements were conducted as standard of care and not systematically collected under the study protocol. Consequently, there were insufficient systematic disease progression events and follow-up data points to calculate a median progression-free survival time or confidence
|
SECONDARY outcome
Timeframe: Initial response assessment after six weeks (Day 43)Response was evaluated at the participant level for non-injected tumors.Response rate was evaluated specifically in non-injected tumors to assess systemic anti-tumoral efficacy, evaluated using RECIST criteria (Complete Response \[CR\], Partial Response \[PR\], Stable Disease \[SD\], or Progressive Disease \[PD\]).
Outcome measures
| Measure |
Recombinant Vaccinia Virus JX-594
n=5 Participants
Intratumoral injection(s) of Recombinant Vaccinia virus JX-594 (Thymidine kinase-inactivated vaccinia virus expressing human GM-CSF)
|
|---|---|
|
Number of Participants With Objective Response in Non-injected Tumor(s)
Complete Response (CR)
|
0 Participants
|
|
Number of Participants With Objective Response in Non-injected Tumor(s)
Partial Response (PR)
|
0 Participants
|
|
Number of Participants With Objective Response in Non-injected Tumor(s)
Stable Disease (SD)
|
3 Participants
|
|
Number of Participants With Objective Response in Non-injected Tumor(s)
Progressive Disease (PD)
|
2 Participants
|
Adverse Events
Recombinant Vaccinia Virus JX-594
Serious adverse events
| Measure |
Recombinant Vaccinia Virus JX-594
n=10 participants at risk
Intratumoral injection(s) of Recombinant Vaccinia GM-CSF, JX-594
JX-594: Thymidine kinase-deleted vaccinia virus plus GM-CSF
|
|---|---|
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Gastrointestinal disorders
Acute Gastrointestinal Bleeding
|
10.0%
1/10
|
|
Nervous system disorders
Spinal Cord Compression
|
10.0%
1/10
|
Other adverse events
| Measure |
Recombinant Vaccinia Virus JX-594
n=10 participants at risk
Intratumoral injection(s) of Recombinant Vaccinia GM-CSF, JX-594
JX-594: Thymidine kinase-deleted vaccinia virus plus GM-CSF
|
|---|---|
|
Metabolism and nutrition disorders
Hypokalaemia
|
10.0%
1/10
|
|
Metabolism and nutrition disorders
Weight loss poor
|
20.0%
2/10
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.0%
1/10
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
10.0%
1/10
|
|
Musculoskeletal and connective tissue disorders
Chills
|
20.0%
2/10
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal discomfort
|
10.0%
1/10
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
30.0%
3/10
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.0%
1/10
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour pain
|
30.0%
3/10
|
|
Nervous system disorders
Dysgeusia
|
10.0%
1/10
|
|
Nervous system disorders
Headache
|
20.0%
2/10
|
|
Nervous system disorders
Paraesthesia
|
10.0%
1/10
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
10.0%
1/10
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.0%
1/10
|
|
Vascular disorders
Hypotension
|
10.0%
1/10
|
|
Blood and lymphatic system disorders
Anaemia
|
40.0%
4/10
|
|
Cardiac disorders
Dizziness
|
30.0%
3/10
|
|
Cardiac disorders
Dyspnoea
|
20.0%
2/10
|
|
Gastrointestinal disorders
Constipation
|
10.0%
1/10
|
|
Gastrointestinal disorders
Diverticulum
|
10.0%
1/10
|
|
Gastrointestinal disorders
Dry mouth
|
10.0%
1/10
|
|
Gastrointestinal disorders
Oesophagitis
|
10.0%
1/10
|
|
Gastrointestinal disorders
Dyspepsia
|
10.0%
1/10
|
|
Gastrointestinal disorders
Nausea
|
20.0%
2/10
|
|
Gastrointestinal disorders
Abdominal discomfort
|
10.0%
1/10
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
2/10
|
|
General disorders
Oedema
|
30.0%
3/10
|
|
General disorders
Facial pain
|
10.0%
1/10
|
|
General disorders
Fatigue
|
70.0%
7/10
|
|
General disorders
Pyrexia
|
30.0%
3/10
|
|
General disorders
Injection site erythema
|
40.0%
4/10
|
|
General disorders
Injection site pain
|
30.0%
3/10
|
|
General disorders
Injection site rash
|
10.0%
1/10
|
|
General disorders
Injection site pruritus
|
20.0%
2/10
|
|
General disorders
Local swelling
|
10.0%
1/10
|
|
General disorders
Night sweats
|
20.0%
2/10
|
|
General disorders
Pain in extremity
|
20.0%
2/10
|
|
General disorders
Tenderness
|
10.0%
1/10
|
|
Infections and infestations
Upper respiratory tract infection
|
10.0%
1/10
|
|
Investigations
Breath sounds abnormal
|
20.0%
2/10
|
|
Investigations
Bicarbonate abnormal
|
10.0%
1/10
|
|
Metabolism and nutrition disorders
Anorexia
|
40.0%
4/10
|
|
Metabolism and nutrition disorders
Dyslipidaemia
|
10.0%
1/10
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
10.0%
1/10
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
10.0%
1/10
|
Additional Information
James Burke, MD, Principal Investigator of trial
SillaJen Biotherapeutics, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60