Clinical Trial: Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the Size of Sporadic Nodular Basal Cell Carcinomas

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Double-Blind, Dose Escalating, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the Size of Sporadic Nodular

Brief 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 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. 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 or 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 t
Sponsor: PellePharm, Inc.

Current Primary Outcome:

  • Clinical efficacy: change from baseline in greatest diameter treatment-targeted surgically eligible basal cell carcinomas [ Time Frame: Baseline and Weeks 2, 6, 8, 10, and 12 ]
    To evaluate the clinical efficacy of patidegib as defined by the percent decrease in greatest diameter of baseline treatment targeted surgically eligible basal cell carcinomas after 12 weeks of treatment
  • Change in safety and tolerability assessment of treatment with patidegib gel [ Time Frame: Screening, Baseline, Weeks 2, 6, 8, 10 and 12 ]
    From baseline, subjects will be assessed for the occurrence of new and ongoing adverse events. AEs present at any visit will be followed to resolution or until clinically stable as determined by the Investigator. Routine safety laboratory tests will be performed at Screening, Baseline and Weeks 2, 6, 8, 10 and 12


Original Primary Outcome: Same as current

Current Secondary Outcome: Molecular efficacy: change from baseline of reduction in the the hedgehog (HH) signaling pathway target gene GLI1 [ Time Frame: Baseline and 12 weeks ]

To evaluate the molecular efficacy of treatment as defined by reduction in the HH signaling pathway target gene GLI1 after treatment with patidegib gel 2% or 4% or vehicle applied once or twice daily for 12 weeks to treatment-targeted SEBs


Original Secondary Outcome: Same as current

Information By: PellePharm, Inc.

Dates:
Date Received: July 1, 2016
Date Started: October 2016
Date Completion: September 2017
Last Updated: May 11, 2017
Last Verified: May 2017