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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02562755




Registration number
NCT02562755
Ethics application status
Date submitted
24/09/2015
Date registered
29/09/2015
Date last updated
16/12/2020

Titles & IDs
Public title
Hepatocellular Carcinoma Study Comparing Vaccinia Virus Based Immunotherapy Plus Sorafenib vs Sorafenib Alone
Scientific title
A Phase 3 Randomized, Open-Label Study Comparing Pexa Vec (Vaccinia GM CSF / Thymidine Kinase-Deactivated Virus) Followed by Sorafenib Versus Sorafenib in Patients With Advanced Hepatocellular Carcinoma (HCC) Without Prior Systemic Therapy
Secondary ID [1] 0 0
JX594-HEP024
Universal Trial Number (UTN)
Trial acronym
PHOCUS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hepatocellular Carcinoma (HCC) 0 0
Condition category
Condition code
Infection 0 0 0 0
Other infectious diseases
Cancer 0 0 0 0
Non melanoma skin cancer
Cancer 0 0 0 0
Kidney
Cancer 0 0 0 0
Liver

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Pexastimogene Devacirepvec (Pexa Vec)
Treatment: Drugs - Sorafenib

Experimental: Pexa-Vec followed by Sorafenib - 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.

Active Comparator: Sorafenib - Sorafenib (400 mg twice daily) begins on Day 1.


Other interventions: Pexastimogene Devacirepvec (Pexa Vec)
Pexa-Vec is a vaccinia virus based oncolytic immunotherapy designed to stimulate the immune system following infection and replication within tumor cells.

Treatment: Drugs: Sorafenib
Sorafenib belongs to the pharmacotherapeutic group of antineoplastic agents, protein kinase inhibitors, ATC code: L01XE05.
Sorafenib is a multi-kinase inhibitor which has demonstrated both anti-proliferative and anti-angiogenic properties in vitro and in vivo.
Sorafenib is approved for the treatment of advanced HCC and is the Standard Of Care for this disease.
Intervention code [1] 0 0
Other interventions
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall Response Rate (ORR)
Timepoint [1] 0 0
From date of randomization to the date of first documented radiographic tumor progression up to 53 months

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/10/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/07/2020
Sample size
Target
Accrual to date
Final
459
Recruitment in Australia
Recruitment state(s)
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Site No. 8409 - Adelaide
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- Melbourne
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- Parkville
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- Perth
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
SillaJen, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02562755
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
SillaJen Medical
Address 0 0
SillaJen, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02562755