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Trial registered on ANZCTR


Registration number
ACTRN12615001017516
Ethics application status
Approved
Date submitted
25/08/2015
Date registered
29/09/2015
Date last updated
26/03/2018
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Phase I/IIa Study of the Efficacy and Safety of ASN-002 Alone and in Combination with 5-Fluorouracil (5-FU) in Adult Patients with Low-Risk Nodular Basal Cell Carcinoma
Scientific title
A Phase I/IIa Study of the Efficacy and Safety of ASN-002 Alone and in Combination with 5-Fluorouracil (5-FU) in Adult Patients with Low-Risk Nodular Basal Cell Carcinoma
Secondary ID [1] 287315 0
NIL
Universal Trial Number (UTN)
Trial acronym
ASN-002-001
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Nodular Basal Cell Carcinoma 295949 0
Basal Cell Carcinoma Nevus Syndrome 302763 0
Condition category
Condition code
Cancer 296224 296224 0 0
Non melanoma skin cancer
Skin 296225 296225 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Injection of ASN-002 into tumour nodules to stimulate an immune response. The dose depends on the study cohort.
Cohort 1: 5 X 10(11) virus particles (vp), weekly injection for 3 weeks.
Cohort 2: 1.5X 10(11) vp weekly injection for 3 weeks
Cohort 4: 3.0 X 10(11) vp weekly injections for 3 weeks,
Cohort 5: 2.25 X 10(11) vp weekly injections for 3 weeks,

Only one combination cohort will enrol patients at dose of Cohort 1: 5 X 10(11) virus particles (vp) & 5-FU (5mg) weekly injection for 3 weeks.

Note: Cohort 3 required 6 injections, which has been removed in amended protocol.
The study drug will be administered by the qualified medical doctor/ dermatologist at the study site. The volume of dose to be administered will depend on the size of the tumor.
Tumor size <6 mm: 0.5 mL
6-10mm: 1 mL
11-20mm: 1.5 mL
The compliance will be monitored through a dose administration log.

Surgical excision of tumor will be performed by a surgeon after 13 weeks follow up from the last study drug dose administered to the study participant.

The duration of surgical excision procedure will depend upon the size and location of the tumor.
Intervention code [1] 292635 0
Treatment: Drugs
Intervention code [2] 292790 0
Treatment: Surgery
Comparator / control treatment
No control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 295889 0
Safety of ASN-002 alone or in combination with 5-FU in treatment of basal cell carcinoma will be assessed by examination of inject site area for pain, erythema, edema, ulcerations etc and by checking the vital signs before and after the study drug administration.
Timepoint [1] 295889 0
All study visits i.e. weekly treatment visits, phone call visits for 2 days after each treatment visit, 4 follow up visits at a frequency of 4 weeks and the day of surgical removal of BCC tumors.
Secondary outcome [1] 316856 0
Microscopic clearance of the injected basal cell carcinoma.
Timepoint [1] 316856 0
Microscopic examinations of sample collected before ASN-002 therapy will be compared with that of the sample collected at surgery after ASN-002 therapy.
Secondary outcome [2] 316857 0
Clinical Changes of nBCC over time after treatment with ASN-002.

This will be assessed by the investigator by measuring any change in the tumor size with help of transparency, ruler and tumor photograph before and after the ASN-002 therapy.
Timepoint [2] 316857 0
Change in nBCC will be assessed at baselines and then every 4 weeks until the surgical excision of BCC after ASN-002 therapy.

Eligibility
Key inclusion criteria
1. Low risk nodular basal cell carcinoma
2. Biopsy of any other skin tumor
3. Willingness to have injection therapy followed by surgery
4. Written informed consent

Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. No or only minimal symptoms
2. Known or suspected metastatic disease.
3. Pregnant or Lactating females
4. Clinically active or uncontrolled skin disease
5. Immunocompromised or receiving immunomodulating agent
6. treatment with psoralen plus UVA or UVB therapy within 6 months
7. Any serious or active medical or psychiatric illness
8. Recreational or therapeutic drug or alcohol use
9. Taking any investigational product within 1 month of first dose of ASN-002.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Each eligible subject will be allocated to the next consecutive cohort available based on safety assessment and tumor response of previously treated subjects.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not a randomized study.
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
There will be 4 cohorts in the study with monotherapy with ASN-002.
Cohort 1: 5 X 10(11) virus particles (vp), weekly injection for 3 weeks.
Cohort 2: 1.5X 10(11) vp weekly injection for 3 weeks
Cohort 4: 3.0X 10(11) vp weekly injection for 3 weeks
Cohort 5: 2.25X 10(11) vp weekly injection for 3 weeks

Only one combination cohort will enrol patients at dose of Cohort 1: 5 X 10(11) virus particles (vp) & 5-FU (5mg) weekly injection for 3 weeks.
Note: Cohort 3 included 6 injections, which has been removed in amended protocol.
In combination cohorts, patients will receive any one of the cohort 2, 4 or 5 dose of ASN-002 in combination with 5-FU (at 1mg/2.5mg/5mg/10mg or 25mg) intratumorally.
Phase
Phase 1 / Phase 2
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
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)
QLD,VIC
Recruitment hospital [1] 4503 0
Veracity Clinical Research - Woolloongabba
Recruitment hospital [2] 4504 0
Sinclair Dermatology - East Melbourne
Recruitment hospital [3] 4505 0
Siller Medical - Central Brisbane Dermatology - Brisbane
Recruitment hospital [4] 7797 0
St George Private Hospital - Kogarah

Funding & Sponsors
Funding source category [1] 291879 0
Commercial sector/Industry
Name [1] 291879 0
Ascend Biopharmaceutical Ltd.
Address [1] 291879 0
Level 1, 159 Dorcas Street
South Melbourne VIC 3205
Country [1] 291879 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Ascend Biopharmaceutical Ltd.
Address
Level 1, 159 Dorcas Street
South Melbourne VIC 3205
Country
Australia
Secondary sponsor category [1] 290544 0
None
Name [1] 290544 0
Address [1] 290544 0
Country [1] 290544 0
Other collaborator category [1] 278684 0
Individual
Name [1] 278684 0
Rodney Sinclair
Address [1] 278684 0
Sinclair Dermatology Pty Ltd
2 Wellington Parade
East Melbourne VIC 3002
Country [1] 278684 0
Australia
Other collaborator category [2] 278685 0
Individual
Name [2] 278685 0
Gregory Siller
Address [2] 278685 0
Siller Medical Pty Ltd
Central Brisbane Dermatology
Level 9, Silverton Place
101 Wickham Terrace, Brisbane QLD 4000
Country [2] 278685 0
Australia
Other collaborator category [3] 278686 0
Commercial sector/Industry
Name [3] 278686 0
Q-pharm Pty Ltd
Address [3] 278686 0
Clive Berghofer Research Centre (CBCRC)
300C Herston Road

Herston QLD 4007

Country [3] 278686 0
Australia
Other collaborator category [4] 279522 0
Individual
Name [4] 279522 0
Stephen Shumack
Address [4] 279522 0
St George Dermatology and Skin Cancer Centre
Address: 3/22 Belgrave St, Kogarah, NSW 2217
Country [4] 279522 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 293388 0
BellBerry Ltd.
Ethics committee address [1] 293388 0
129 Glen Osmond Rd
Eastwood SA 5063
Ethics committee country [1] 293388 0
Australia
Date submitted for ethics approval [1] 293388 0
Approval date [1] 293388 0
20/07/2015
Ethics approval number [1] 293388 0
EC00372

Summary
Brief summary
The primary purpose of this study is to determine whether ASN-002 alone or in combination with 5-FUis safe and effective in the treatment of nodular basal cell carcinoma (nBCC).

Who is it for?
You may be eligible to join this study if you are aged 18 or over, have been diagnosed with nBCC.

Study details
ASN-002 is a vaccine-like product that is injected into the BCC spot to be treated. It is made from modified adenovirus serotype 5 (also called Ad5). Adenoviruses are common in nature worldwide and can cause mild colds and respiratory infections from which people usually recover without treatment. The Ad5 used in this study has been modified so that it cannot grow in the body or cause an infection. The modified Ad5 in this study will deliver artificially made genetic material into the cancerous and surrounding cells. This genetic material will produce human interferon which is normally produced by the body to stimulate the immune system. It is hoped that injected ASN-002 will cause the body’s own cells to produce interferon and stimulate the immune system to attack the cancerous cells and reduce the size of or eliminate the nBCC. 5-FU is a chemotherapeutic agent approved to treat many types of cancer. Participants will attend the study centre weekly for an injection of ASN-002 alone or in combination with 5-FU into the nBCC. The participants recruited will have 3 injections over 3 weeks, and then undergo surgical excision of the tumour.
Patient outcomes will then be assessed using a tumour sample collected during surgery and by the incidence of adverse events which occur throughout the study.

It is hoped that the findings of this trial will provide information on the safety and efficacy of using ASN-002 alone and in combination with 5-FU for nBCC, particularly for patients in whom the standard treatment of surgery is not possible or not recommended.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 59662 0
Prof Lynda Spelman
Address 59662 0
Veracity Clinical Research Pty. Ltd 250 Ipswich Road Woolloongabba QLD 4102
Country 59662 0
Australia
Phone 59662 0
+61 7 3039 1346
Fax 59662 0
+61 7 38453635
Email 59662 0
spelchat@iinet.net.au
Contact person for public queries
Name 59663 0
Dr Clement Leong
Address 59663 0
Ascend Biopharmaceuticals Ltd
Level 1, 159 Dorcas Street
South Melbourne VIC 3205
Country 59663 0
Australia
Phone 59663 0
+61 3 86063400
Fax 59663 0
Email 59663 0
clementleong@ascendbiopharma.com
Contact person for scientific queries
Name 59664 0
Prof Lynda Spelman
Address 59664 0
Veracity Clinical Research Pty. Ltd 250 Ipswich Road Woolloongabba QLD 4102
Country 59664 0
Australia
Phone 59664 0
+61 7 38453635
Fax 59664 0
Email 59664 0
spelchat@iinet.net.au

No data has been provided for results reporting
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary