Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas
Condition: Basal Cell Carcinomas
MeSH terms: Carcinoma; Carcinoma, Basal Cell
Study type: Interventional
Phase: Phase 2

Summary: 
      This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to
      compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in
      comparison with that of vehicle in patients with Basal Cell Carcinoma. One investigational
      center (metasite) in the United States will participate in this study. Approximately 36
      subjects who meet the study entry criteria will be enrolled into one of four sequential
      cohorts. Within each cohort subjects will be randomized in a 2:1 ratio to receive active or
      vehicle gel.
    

Detailed description: 
      This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to
      compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in
      comparison with that of vehicle. Approximately 36 subjects who meet the study entry criteria
      will be enrolled into one of four sequential cohorts. As soon as one cohort has been
      completely enrolled, the next cohort will be enrolled. Each subject will treat no more than
      two previously untreated biopsy confirmed treatment-targeted nodular BCCs. If the subject has
      additional non-treatment targeted BCCs they can be treated surgically prior to or during the
      trial. Within each cohort subjects will be randomized in a 2:1 ratio to receive active or
      vehicle gel. The sequential cohorts will be:

        -  Cohort 1: patidegib gel 2% or vehicle, once daily

        -  Cohort 2: patidegib gel 4% or vehicle, once daily

        -  Cohort 3: patidegib gel 2% or vehicle, twice daily

        -  Cohort 4: patidegib gel 4% or vehicle, twice daily

      The study drug will be applied topically to the treatment-targeted BCCs and a rim of adjacent
      skin for 12 weeks. Information on reported and observed adverse events (AEs) will be obtained
      at each visit. An abbreviated physical examination (PE) will be performed at Baseline and
      Week 12. The treatment-targeted BCCs will be identified by the Investigator at the Baseline
      visit and will be circled in ink at Baseline, Weeks 6 and 12 and photographed, and measured
      at all study visits (Baseline, Weeks 2, 6, 8, 10, and 12). Blood samples for complete blood
      count and serum chemistry and urine for urinalysis will be collected from subjects at
      Screening, Week 6, and Week 12. Subjects who terminate study participation early will be
      asked to complete all Week 12 assessments, as appropriate, prior to commencement of any
      alternative therapy for BCCs (if possible). Subjects who discontinue from the study during
      the treatment period will not be replaced.
    

Intervention name(s): Patidegib; Vehicle gel
Intervention type(s): Drug; Drug
Other intervention name(s): Study drug; Placebo
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
Keywords: Gorlin Syndrome; Basal Cell Nevus Syndrome BCNS; nevoid basal cell carcinoma syndrome; Basal cell carcinoma; Hedgehog inhibitor; Surgically Eligible Basal Cell Carcinomas; High Frequency Basal Cell Carcinomas