This is a randomized Phase 3 study to determine whether treatment with vaccinia virus based immunotherapy (Pexa-Vec) followed by sorafenib increases survival compared to treatment with sorafenib in patients with advanced hepatocellular carcinoma who have not received prior systemic therapy.
Completed
Phase 3
| Drug | Synonyms | Arms |
|---|---|---|
| Pexastimogene Devacirepvec (Pexa Vec) | JX-594 | Pexa-Vec followed by Sorafenib |
| Sorafenib | Nexavar | Pexa-Vec followed by Sorafenib |
This is a multi-center, randomized, open-label, Phase 3 study comparing Pexa Vec followed by
sorafenib versus sorafenib in patients with advanced HCC without prior systemic therapy.
A total of 459 patients were randomly assigned to 2 treatment arms- 234 patients in the
Pexa-Vec followed by sorafenib treatment group and 225 patients in the sorafenib only
treatment group.
| Name | Type | Description | Interventions |
|---|---|---|---|
| Pexa-Vec followed by Sorafenib | Experimental | Pexa-Vec (pexastimogene devacirepvec) will be administered as 3 bi-weekly intratumoral (IT) injections of 1e9 pfu at day 1 and weeks 2 and 4, followed by sorafenib at Week 6. |
|
| Sorafenib | Active Comparator | Sorafenib (400 mg twice daily) begins on Day 1. |
|
Inclusion Criteria:
- Histological/cytological diagnosis of primary HCC
- Advanced stage HCC (Barcelona Clinic Liver Cancer [BCLC] Stage C or B per American
Association for the Study of Liver Disease [AASLD] guidelines)
- At least one measurable viable tumor in the liver, ≥1 cm longest diameter (LD), using
a dynamic imaging technique (arterial phase of triphasic computerized tomography [CT]
scan, or dynamic contrast-enhanced magnetic resonance imaging [MRI]), and injectable
under imaging-guidance (CT and/or ultrasound)
- Child-Pugh Class A
- Performance status 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) scale
- Adequate hematological, hepatic, and renal function:
- Additional inclusion criteria exist
Exclusion Criteria:
- Histological diagnosis of cholangiocarcinoma, hepatocholangiocarcinoma, fibrolamellar
carcinoma and hepatoblastoma
- Symptomatic cardiovascular disease, including but not limited to significant coronary
artery disease (e.g., requiring angioplasty or stenting) or congestive heart failure
within the preceding 12 months
- Current or past history of cardiovascular disease (e.g.. past history of myocardial
infarction, ischemic cardiomyopathy) unless cardiology consultation and clearance has
been obtained for study participation
- History of moderate or severe ascites, bleeding esophageal varices, hepatic
encephalopathy or pleural effusions related to liver insufficiency within 6 months of
screening
- Bulky disease patients - tumors encompassing >50% of the liver volume and / or
inferior vena cava invasion
- Known significant immunodeficiency due to underlying illness (e.g., HIV/AIDS) and/or
immune-suppressive medication including high-dose corticosteroids
- Ongoing severe inflammatory skin condition (as determined by the Investigator)
requiring medical treatment
- History of severe eczema (as determined by the Investigator) requiring medical
treatment
- Additional exclusion criteria exist
| Maximum Eligible Age: | N/A |
| Minimum Eligible Age: | 18 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
| Measure: | Overall Response Rate (ORR) |
| Time Frame: | From date of randomization to the date of first documented radiographic tumor progression up to 53 months |
| Safety Issue: | |
| Description: | Percentage of participants who showed overall response during their participation in the study. Per Modified Response Evaluation Criteria In Solid Tumors Criteria (mRECIST) and assessed by tri-phasic contrast enhanced CT: Complete Response (CR), Disappearance of intratumoral enhancing area; Partial Response (PR), >=30% decrease in the sum of the diameters of enhancing area; Overall Response (OR) = CR + PR. |
| Phase: | Phase 3 |
| Primary Purpose: | Interventional |
| Overall Status: | Completed |
| Lead Sponsor: | SillaJen, Inc. |
December 16, 2020
