Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
- Conditions
- Gorlin SyndromeMedDRA version: 20.1Level: LLTClassification code: 10004151Term: Basal cell nevus syndrome Class: 10010331MedDRA version: 20.0Level: LLTClassification code: 10078877Term: Gorlin-Goltz syndrome Class: 10010331Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2023-507528-21-00
- Lead Sponsor
- Sol-Gel Technologies Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 156
The subject must be at least 18 years old at the Screening Visit, The subject must be confirmed to have a PTCH1 mutation, The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1), The subject must be willing to abstain from application of any non-study topical medication (prescription or over the counter) to their facial skin for the duration of the trial, except as prescribed by the investigator
The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP), The subject is known to have hypersensitivity to any of the ingredients in the IP formulation, The subject has uncontrolled systemic disease, The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0 and non-melanoma skin cancer, Stage I cervical cancer, or ductal carcinoma in situ of the breast, Previous Hedgehog inhibitor therapy was considered to have failed for reasons other than unfavorable side effect(s)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the efficacy of Patidegib Gel 2% when applied twice daily to the face of subjects with Gorlin syndrome;Secondary Objective: To assess the safety and tolerability of Patidegib Gel 2% in subjects treated twice daily for 12 months;Primary end point(s): To assess the number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and Vehicle) when applied twice daily to the face of subjects with Gorlin syndrome
- Secondary Outcome Measures
Name Time Method