NCT02762084

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

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 Cell Carcinomas in Gorlin Syndrome Patients

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: PellePharm, Inc. · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

Participants 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 participant 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 6, 10, 14, 18, 22, and 26, all visible basal cell carcinomas (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 (defined as the 5 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 participant has 5 eligible previously untreated facial SEBs (excluding tumors on nose and eyelids) these tumors will be the participant's 5 baseline treatment-targeted SEBs and non-facial baseline SEBs will not be treated with study drug. Tumors to be measured and mapped include the 5 baseline treatment-targeted tumors as well as all other facial tumors including those on the eyelids and the nose. In addition, up to 10 non-treatment-targeted non-facial tumors will also be measured and mapped.

Conditions

• Basal Cell Nevus Syndrome

Interventions

Type	Name	Description
DRUG	Patidegib
DRUG	Vehicle gel

Timeline

• Start date: 2016-06-06
• Primary completion: 2017-04-24
• Completion: 2017-04-24
• First posted: 2016-05-04
• Last updated: 2020-07-23
• Results posted: 2019-07-15

Locations

2 sites across 1 country: United Kingdom