Clinical Trial: Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas (BCC) in Gorlin Syndrome Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal (SEB) Cell Carcinomas in

Brief Summary: Multicenter, double-blind, randomized, vehicle-controlled evaluates the efficacy and safety of patidegib gel, 2% and 4% in comparison with vehicle in subjects at least 18 years of age that meet the diagnostic criteria for basal cell nevus syndrome (BCNS). Subjects will be randomized to receive patidegib gel 4%, patidegib gel 2%, or the vehicle gel for a 26 week treatment period.

Detailed Summary:

This is a multicenter, double-blind, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel, 2% and 4% in comparison with that of vehicle. To be eligible for the study, subjects must be at least 18 years of age and must meet the diagnostic criteria for basal cell nevus syndrome BCNS.

Approximately 18 subjects who meet the study entry criteria will be randomized in a 1:1:1 ratio to receive patidegib gel 2%, patidegib gel 4%, vehicle gel. One or two tubes of the assigned study drug will be dispensed to the subject at the Baseline visit. Additional tubes will be dispensed at subsequent visits through Week 22. The study drug will be applied topically to the entire face as well as to treatment-targeted surgically eligible basal cell carcinomas (SEBs) at other anatomical sites twice daily for 26 weeks of treatment.

Information on reported and observed adverse events will be obtained at each visit. An abbreviated physical examination will be performed at Baseline, Week 14, and Week 26.

At Baseline and Weeks 2, 6, 10, 14, 18, 22, and 26, all visible BCCs (excluding areas below the knees) will be identified by the Investigator, circled in ink, photographed, measured, and recorded on a body diagram. Treatment targeted SEBs are defined as the five SEBs on the face and/or other anatomical areas identified at Baseline as SEBs will be treated during the 26 week treatment phase. If a subject has 5 eligible previously untreated facial SEBs (excluding tumors on nose and eyelids) these tumors will be the subject's 5 baseline treatment targeted SEBs and non-facial baseline SEBs will not be treated with study medication.


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, 10, 14, 18, 22, and 26 ]
    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 26 weeks of treatment.
  • Molecular efficacy: change from baseline of reduction in the the hedgehog (HH) signaling pathway target gene GLI1 [ Time Frame: Baseline and 6 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 twice daily for 6 weeks to treatment-targeted SEBs.
  • Change in safety and tolerability assessment of treatment with patidegib gel [ Time Frame: Screening, Baseline, Weeks 2, 6, 10, 14, 18, 22, and 26 ]
    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, Weeks 6, 14, and 26.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Clinical efficacy: change from baseline in central facial SEBs [ Time Frame: Baseline, Weeks 2, 6, 10, 14, 18, 22, and 26 ]
    To evaluate the clinical efficacy of treatment as defined by percent decrease in greatest diameter of baseline central facial SEBs.
  • Change from baseline of frequency of new SEBs on the face [ Time Frame: Baseline, Weeks 2, 6, 10, 14, 18, 22, and 26 ]
    To evaluate the frequency of new SEBs on the face.
  • Change from baseline in the proportion of non-central facial BCCs [ Time Frame: Baseline and Weeks 6, 10, 14, 18, 22, and 26 ]
    To evaluate the proportion of non-central facial BCCs that at Baseline and/or Week 2 visit were less than 5 mm in greatest diameter but by the Week 6, 10, 14, 18, 22, or 26 visit have become greater than 5 mm in greatest diameter.
  • Change from baseline in the proportion of baseline treatment targeted SEBs [ Time Frame: Baseline, Weeks 2, 6, 10, 14, 18, 22, and 26 ]
    To evaluate the proportion of baseline treatment targeted SEBs that at the end of 26 weeks of treatment are no longer large enough to be classified as SEBs


Original Secondary Outcome: Same as current

Information By: PellePharm, Inc.

Dates:
Date Received: April 27, 2016
Date Started: June 6, 2016
Date Completion:
Last Updated: May 5, 2017
Last Verified: May 2017