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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02562755
Ethics application status
Date submitted
24/09/2015
Date registered
29/09/2015
Date last updated
12/08/2019

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 Survival
Timepoint [1] 0 0
From the date of randomization to the date of death due to any cause up to study completion (approximately 53 months).
Secondary outcome [1] 0 0
Time to Progression (TTP)
Timepoint [1] 0 0
From date of randomization to the date of first documented radiographic tumor progression up to 53 months.
Secondary outcome [2] 0 0
Progression Free Survival (PFS)
Timepoint [2] 0 0
From date of randomization to the date of first documented radiographic tumor progression or death, whichever occurs first, assessed up to 53 months.
Secondary outcome [3] 0 0
Overall Response Rate (ORR)
Timepoint [3] 0 0
From the date of randomization until disease progression, up to 53 months.
Secondary outcome [4] 0 0
Disease Control Rate (DCR) - Proportion of patients whose best overall response during their participation in the study is either CR, PR, or stable disease (SD).
Timepoint [4] 0 0
From date of randomization to end of participation in the study up to 53 months.
Secondary outcome [5] 0 0
Incidence of Adverse Events (AE) and Serious Adverse Events (SAE) - Assessed by the NCI CTCAE (version 4.03). Incidence of AEs and SAEs will be reported.
Timepoint [5] 0 0
From date of randomization to end of participation in the study, up to 53 months.
Secondary outcome [6] 0 0
Time to Symptomatic Progression (TSP)
Timepoint [6] 0 0
Time from randomization until the first documented event of symptomatic 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
Gender
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
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Site No. 8409 - Adelaide
Recruitment hospital [2] 0 0
Site No. 8412 - Adelaide
Recruitment hospital [3] 0 0
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Recruitment hospital [5] 0 0
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Recruitment hospital [6] 0 0
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Recruitment hospital [7] 0 0
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Recruitment hospital [8] 0 0
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Recruitment hospital [9] 0 0
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Recruitment hospital [10] 0 0
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Recruitment hospital [11] 0 0
Site No. 8402 - Parkville
Recruitment hospital [12] 0 0
Site No. 8415 - Perth
Recruitment hospital [13] 0 0
Site No. 8413 - Sydney
Recruitment postcode(s) [1] 0 0
- Adelaide
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- Camperdown
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- Clayton
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- Concord
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- Fitzroy
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- Footscray
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- Heidelberg
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- Melbourne
Recruitment postcode(s) [10] 0 0
- Parkville
Recruitment postcode(s) [11] 0 0
- Perth
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- Sydney
Recruitment outside Australia
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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/show/NCT02562755
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Hyuk Chan KWON, MD
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
Other publications