A Multicenter, Randomized, Double blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects with Gorlin Syndrome

Gorlin Syndrome

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 as assessed by the CPRB

Number of New Surgically Eligible BCCs (nSEBs) at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome, Change from Baseline to Month 12 in Advanced Basal Cell Carcinoma Index (aBCCdex) lesion symptoms scale score, Change from Baseline to Month 12 in confirmed BCCs lesion diameter, Proportion of Surgically Eligible BCCs (SEBs) and nSEBs assessed as resolved by Month 12, Proportion of SEBs and nSEBs per subject that have regressed by Month 12, Proportion of subjects developing ≥2 facial new BCCs at Month 12, Change in total number of confirmed BCCs from Baseline to Month 12, Change from Baseline to Month 12 in the Skin Cancer Index quality of life questionnaire score, Adverse events (AEs), including serious adverse events (SAEs) occurring at any time during the study, Investigator Dermal Safety and Local Tolerability Assessments score at any time during the study, Change from Baseline to Month 12 in Patient Global Impression in disease Severity (PGI-S), Change in Patient Global Impression of Change in clinical status (PGI C) at Month 12, 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, Number of nSEBs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome, Time to first new BCC, Skin pain and peak pruritus scores as reported by the subject on a numeric rating scale

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