To Determine the Maximum Tolerated Dose Level (MTD) of PEP005 Topical Gel in Patients With sBCC

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to determine maximum tolerated dose level (MTD) of PEP005 Topical Gel for the treatment of superficial basal cell carcinoma on the trunk

Detailed Description
-----------------
Basal cell carcinomas (BCCs) are a common skin problem largely caused by long term sun exposure. Current treatments include surgery, curettage/desiccation and simple excision, which are often cosmetically disfiguring. Non-invasive alternative therapy for treatment of BCC lesions is thus being researched. Sap from the plant Euphorbia peplus has been used for many years in Australia as a folk remedy to treat a number of skin conditions. The active component of Euphorbia peplus has been isolated and made into a gel applied directly to the skin by Peplin Opeation Pty Ltd. The proposed study follows a previously conducted 12 week study in sBCC, PEP005-003. This was a two dose schedule evaluating doses up to 0.05%. Histological clearance rate at the end of study was 71% for the 0.05% concentration on the Day 1, Day 2 schedule compared with 0% in the vehicle group. The proposed study aims to determine the maximum tolerated dose level (MTD) of PEP005 Topical Gel, in patients, when administered either as a single application or as two applications to a selected superficial basal cell carcinoma lesion with a 3 month follow up.

Official Title
-----------------
An Open-label, Multi-center, Dose-escalation, Cohort Study to Determine the Maximum Tolerated Dose and Safety of PEP005 Topical Gel Given as Either a Single Application (on Day 1) or as Two Applications (on Day 1 and Day 8) to a Superficial Basal Cell Carcinoma (sBCC) on the Trunk.

Conditions
-----------------
Superficial Basal Cell Carcinoma

Intervention / Treatment
-----------------
* Drug: PEP005


Participation Criteria
=================
Eligibility Criteria
-----------------
Male or female patients at least 18 years of age. A primary clinically diagnosed and histologically confirmed sBCC lesion located on the trunk which is suitable for excision the histological diagnosis should be made no more than 60 days prior to the screening visit the biopsy specimen should have removed no more that 20% of the total tumour mass Ability to follow study instructions and likely to complete all study requirements. Written informed consent has been obtained. Agreement from the patient to allow photographs of the selected lesion to be taken and used as part of the study data package.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1<br>One day treatment | Drug: PEP005<br>* 0.25% PEP005 Gel<br>|
| Active Comparator: 2<br>Two day treatment | Drug: PEP005<br>* 0.25% PEP005 Gel<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum Tolerated Dose level (MTD) through incidence of treatment related AEs, local skin reactions and pigmentation and scarring. |  | 85 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy (complete sBCC clearance rate and composite sBCC clearance rate) |  | 85 days | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
sBCC, Topical, Dermatology