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Trial registered on ANZCTR


Registration number
ACTRN12618001351202p
Ethics application status
Submitted, not yet approved
Date submitted
17/06/2018
Date registered
10/08/2018
Date last updated
10/08/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
Pre-operative Vismodegib for Basal Cell Carcinoma surrounding the eye
Scientific title
A prospective case series evaluating neo-adjuvant use of Vismodegib in patients with periocular basal cell carcinoma to evaluate reduction in clinical and surgical tumour margins
Secondary ID [1] 295075 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Basal cell carcinoma 308375 0
Periocular malignancy 308376 0
Condition category
Condition code
Cancer 307372 307372 0 0
Non melanoma skin cancer
Cancer 308050 308050 0 0
Head and neck

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Vismodegib 150 milligram Capsules
Orally
Once Daily for 6 months
Adherence monitored at 1, 3 and 6 monthly clinical appointments

Drug adherence will be monitored by audit of participant diary and 3 month prescribing follow-up
Intervention code [1] 301560 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 306344 0
Change in tumour clinical margins
Timepoint [1] 306344 0
This endpoint will be monitored at 1, 3 and 6 months of treatment with the trial drug
Final measurements will be made at 6 months post intervention commencement
This will be measured using a measuring tape with 1mm increments placed against the longest and shortest axis and taking clinical photographs
Primary outcome [2] 307042 0
Change in tumour surgical margins
Timepoint [2] 307042 0
This endpoint will be monitored at 1, 3 and 6 months of treatment with the trial drug
Final measurements will be made at 6 months post intervention commencement
This will be measured using a measuring tape with 1mm increments placed against the longest and shortest axis and taking clinical photographs
Primary outcome [3] 307043 0
Change in tumour histopathological margins
Timepoint [3] 307043 0
This will be measured at 6 months post-intervention commencement
This will be measured by pathologist report on biopsy of tissue
Secondary outcome [1] 350458 0
Tumour characteristics
Timepoint [1] 350458 0
Morphological characteristics of the tumour will be evaluated and recorded at the time of enrolment then again at 6 months following treatment commencement
These will be described using standard descriptors of tumours and monitored/recorded using clinical photographs
Secondary outcome [2] 350459 0
Response to treatment
Timepoint [2] 350459 0
This will be monitored at 1, 3 and 6 months following commencement of the treatment drug
Definitions are Partial Response: At least 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum of longest diameter
Complete Response: Disappearance of all target lesions
Stable Disease: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the sum longest diameter since the treatment started
Disease Progression: At least 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or the appearance of one or more new lesions

This will be evaluated my measuring target lesions with a 1mm increment ruler and comparing to baseline measurements
Secondary outcome [3] 350460 0
Drug Related Side Effects including:
Nausea, Diarrhoea, Constipation, Vomiting, Fatigue, Loss of Appetite, Muscle Spasms, Taste Loss/Distortion, Hair Loss, Amenorrhoea, Abdominal Pain, Dehydration
Timepoint [3] 350460 0
This will be monitored at 1, 3 and 6 months following treatment commencement by taking a complete medical history specifically screening for these side effects and performing relevant physical examinations at these visits

Eligibility
Key inclusion criteria
1. Male or female aged greater than or equal to 18 years of age with histopathologically confirmed basal cell carcinoma of the periocular skin
2. Able to participate and willing to provide written consent including consent for photographs prior to performance of study-related procedures and to comply with the study protocol
3. Willingness to delay excision of the target tumour site until the time mandated in the protocol, unless evidence of disease progression or lack of drug tolerability
4. Patients must be naïve to Vismodegib and other hedgehog pathway inhibitors
5. Willingness to undergo histopathologic confirmation by with 3mm punch biopsy
6. Female patients of child-bearing age must agree to use 2 reliable forms of contraception including one highly effective method and a barrier method during treatment and for 27months after final dose
7. Breast feeding is not allowed during the treatment and for at least 7 months after completion of study therapy
8. Negative serum pregnancy test within 7 days prior to commencement of dosing in women of child-bearing potential
9. Male patients with partners of child-bearing potential must always use a condom even after vasectomy, during treatment with study medication and for at least 2 months after the final dose
10. Men must agree not to donate sperm during treatment and for 2 months after discontinuation of therapy
11. Agreement not to donate blood or blood products during the study period and for at least 7 months after discontinuation
12. Absence of psychological, familial, sociological, or geographical condition that potentially hampers compliance with the study protocol and follow-up as defined by the treatment discontinuation schedule
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Age <18 years
2. Inability or unwillingness to comply with study and follow-up procedures
3. History of prior treatment with Vismodegib or any hedgehog pathway inhibitor
4. Known hypersensitivity to Vismodegib
5. Inability or unwillingness to swallow capsules
6. History of malabsorption or other condition that would interfere with absorption of orally delivered study drug
7. Pregnant, lactating or breast-feeding women
8. Participation in another clinical study within 28 days before registration
9. Uncontrollable medical illness, including advanced malignancies
10. History of other condition that would affect interpretation of study results
11. Current severe, uncontrolled systemic illness
12. Any medical or psychological illness preventing adequate consent of ability to comply with the study protocol
13. Patients with one of the following rate hereditary conditions: galactose intolerance, primary hypolactasia, or glucose-galactose malabsorption
14. Known or suspected alcohol or drug abuse
15. Unfit for surgical intervention

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)
None
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
None
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint(s)
Efficacy
Statistical methods / analysis
For a power of 0.8 and alpha of 0.05 and a single-sided t-test comparing baseline and follow-up parameters, a minimum sample size of 12 is required.

Recruitment
Recruitment status
Not yet 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)
SA
Recruitment hospital [1] 11170 0
The Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 22997 0
5000 - Adelaide

Funding & Sponsors
Funding source category [1] 299656 0
Hospital
Name [1] 299656 0
Royal Adelaide Hospital
Address [1] 299656 0
1 Port Road,
Adelaide, SA 5000
Country [1] 299656 0
Australia
Primary sponsor type
Hospital
Name
Royal Adelaide Hospital
Address
1 Port Road,
Adelaide, SA 5000
Country
Australia
Secondary sponsor category [1] 299165 0
None
Name [1] 299165 0
Address [1] 299165 0
Country [1] 299165 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 300555 0
Central Adelaide Local Health Network HREC [EC00192]
Ethics committee address [1] 300555 0
1 Port Road,
Adelaide, SA 5000
Ethics committee country [1] 300555 0
Australia
Date submitted for ethics approval [1] 300555 0
05/06/2018
Approval date [1] 300555 0
Ethics approval number [1] 300555 0

Summary
Brief summary
The purpose of this study is to determine the effects of oral Vismodegib as an adjuvant treatment in periocular basal cell carcinoma.

Who is it for?

You may be eligible for this study if you are an adult who has been diagnosed with periocular basal cell carcinoma.

Study details

All participants in this study will receive the treatment of Vismodegib once daily. This medication will be subsidised by PBS for participants. Prior to commencing treatment, female participants will require a pregnancy test and a punch biopsy of the tumour will be taken in all cases.

You may require additional blood tests prior to commencing the treatment medication, no additional scans or investigations will be required outside of standard practice for this trial.

It is hoped that this research will demonstrated a reduction in the surface area of skin removed by wide surgical excision following neo-adjuvant use of Vismodegib for periocular basal cell carcinoma and improved surgical outcomes for patients.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 84046 0
Prof Dinesh Selva
Address 84046 0
Room 6G-462
Royal Adelaide Hospital
1 Port Road
Adelaide SA 5000
Country 84046 0
Australia
Phone 84046 0
+61 8 7074 2391
Fax 84046 0
+61 8 7074 6215
Email 84046 0
saioph@gmail.com
Contact person for public queries
Name 84047 0
Ms Melanie Willoughby
Address 84047 0
Room 6G-462
Royal Adelaide Hospital
1 Port Road
Adelaide SA 5000
Country 84047 0
Australia
Phone 84047 0
+61 8 7074 2391
Fax 84047 0
Email 84047 0
melanie.willoughby@sa.gov.au
Contact person for scientific queries
Name 84048 0
Prof Dinesh Selva
Address 84048 0
Room 6G-462
Royal Adelaide Hospital
1 Port Road
Adelaide SA 5000
Country 84048 0
Australia
Phone 84048 0
+61 8 7074 2391
Fax 84048 0
+61 8 7074 6215
Email 84048 0
saioph@gmail.com

No information has been provided regarding IPD availability
Summary results
No Results