Hepatocellular Carinoma
Conditions
Keywords
Liver Cancer, HCC, first line HCC, Pexa-Vec, JX-594
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).
Interventions
BIOLOGICALJX-594 recombinant vaccina GM-CSF
Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal.
Sponsors
Jennerex Biotherapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
Countries
South Korea, Spain, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| JX-594 Recombinant Vaccina GM-CSF JX-594 recombinant vaccina GM-CSF
JX-594 recombinant vaccina GM-CSF: Enrolled patients will receive 5 weekly IV infusions on Days 1, 8, 15, 22, and 29. After Day 43, if their disease has improved or remained stable and they have not started other cancer therapy, they may be able to continue to receive JX-594 via IV infusion every three weeks. This treatment extension may continue until radiologic progressive disease, initiation of other cancer therapy, or patient withdrawal. | 16 |
| Total | 16 |
Baseline characteristics
| Characteristic | JX-594 Recombinant Vaccina GM-CSF |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 2 Participants |
| Age, Categorical Between 18 and 65 years | 14 Participants |
| Age, Continuous | 54.3 Years STANDARD_DEVIATION 13.14 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 16 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 14 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 2 Participants |
| Region of Enrollment South Korea | 13 participants |
| Region of Enrollment United States | 3 participants |
| Sex: Female, Male Female | 14 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 3 / 16 |
| other Total, other adverse events | 16 / 16 |
| serious Total, serious adverse events | 8 / 16 |
Outcome results
Primary
Number of Participants With Radiographic Response
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
Time frame: CT scans every 6 week from Week 6 up to 12 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| JX-594 Recombinant Vaccina GM-CSF | Number of Participants With Radiographic Response | 4 Participants |
Secondary
Overall Survival (OS)
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 frame: CT scans every 6 week from Week 6 up to 12 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| JX-594 Recombinant Vaccina GM-CSF | Overall Survival (OS) | 7.47 months |
Secondary
Time to Progression (TTP)
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.
Time frame: CT scans every 6 week from Week 6 up to 12 months.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| JX-594 Recombinant Vaccina GM-CSF | Time to Progression (TTP) | 1.38 months |