This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with Gorlin syndrome. Participants will be required to apply the investigational product for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new BCCs that develop over the 12 month period.
An open-label, extension safety and tolerability study is planned for at least 12 months duration following the end of this study. All participants who complete the Month 12 Exit Visit having demonstrated adequate compliance with application of the Investigational Product (IP) without major Protocol Deviations (PDs) during the study will be eligible for participation in the extension study. All participants will be contacted by phone approximately 30 days following the Exit or Discontinuation Visit to determine if the participant has experienced any new adverse events (AEs)/serious AEs (SAEs) since discontinuation/completion of study treatment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
174
Patidegib Topical Gel, 2%
Patidegib Topical Gel, Vehicle
Mayo Clinical Cancer Center
Phoenix, Arizona, United States
Dermatology Center of Newport
Newport Beach, California, United States
Stanford University, Department of Dermatology
Redwood City, California, United States
University of California, San Francisco
San Francisco, California, United States
Yale School of Medicine
New Haven, Connecticut, United States
Number of New BCCs Per Participant
Time frame: Month 12
Number of New Surgically Eligible BCCs (nSEBs) Per Participant
Time frame: Month 12
Percentage of Participants Developing >=2 Facial New BCCs
Time frame: Month 12
Percentage of Participants Developing >=1 Facial New BCCs
Time frame: Month 12
Number of New BCCs Per Participant
Time frame: Month 9
Number of New BCCs Per Participant
Time frame: Month 6
Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Symptom Score Scale Score
The Advanced Basal Cell Carcinoma Index (aBCCdex) measures the impact of basal cell carcinoma on patients' quality of life. The index consists of several subscales, each subscale with its own range of scores for each question Lesion Symptoms Scale: Scores range from 1 to 7 and it includes 6 questions. Therefore the total score range is from 6 (minimum) to 42 (maximum), where a higher score correlates with a poorer quality of life.
Time frame: Month 12
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