An Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas in Patients With Non-Gorlin High Frequency BCC

A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 2 Efficacy and Safety Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Patients With Non-Gorlin High Frequency BCC

This is a multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with non-Gorlin HF-BCC (high-frequency basal cell carcinoma). Participants will be randomized (1:1) to receive either Patidegib Topical Gel, 2%, or Vehicle for 9 months. Randomization will be stratified by gender. The primary endpoint is the number of nSEB (surgically eligible basal cell carcinoma) that develop on the face over the 9 month period. The primary end point will be assessed by imaging and tracking of BCCs consistently throughout the study in order to identify nSEBs.

Study Overview

• Status: Terminated
• Conditions: Recurrent Basal Cell Carcinoma
• Intervention / Treatment: Drug: Patidegib Topical Gel, 2%; Drug: Patidegib Topical Gel, Vehicle

• Study Type: Interventional
• Enrollment (Actual): 47
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Apple Valley, California, United States, 92307
      • Axiom Research, LLC
    • Colton, California, United States, 92324
      • Axiom Research, LLC
    • Fremont, California, United States, 94538
      • Center For Dermatology Clinical Research, Inc.
    • Los Angeles, California, United States, 90045
      • Dermatology Research Associates
    • Newport Beach, California, United States, 92660
      • The Dermatology Center of Newport
  • Florida
    • Delray Beach, Florida, United States, 33484
      • Palm Beach Dermatology Research
    • Ormond Beach, Florida, United States, 32174
      • Leavitt Medical Associates of Florida
    • Saint Augustine, Florida, United States, 32080
      • PellePharm Investigative Site
  • Indiana
    • Plainfield, Indiana, United States, 46168
      • The Indiana Clinical Trials Center
  • Michigan
    • Warren, Michigan, United States, 48088
      • Grekin Skin Institute
  • Nevada
    • Henderson, Nevada, United States, 89052
      • PellePharm Investigative Site
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Skin Laser and Surgery Specialists of NY&NJ
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Utah
    • Murray, Utah, United States, 84107
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. The subject must have had at least 10 (with at least 3 on the face) clinically typical BCCs present within 24 months prior to Screening and Randomization (Baseline/Day 1). Additionally, the subject must have at least 2 BCCs with longest diameter <5 mm present on the face prior to Screening and Randomization (Baseline/Day 1).
2. The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator.

Exclusion Criteria:

1. The subject has been previously diagnosed with Gorlin syndrome
2. On medical history, family history or clinical examination there is a suspicion that the patient has Gorlin syndrome.
3. Patients with a family history of medulloblastoma
4. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
5. The subject has uncontrolled systemic disease.
6. The subject has been treated for invasive cancer within the past 5 years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) Stage 0.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Patidegib Topical Gel, 2%; Participants will be randomized (1:1) to receive Patidegib Topical Gel, 2% for 9 months	Drug: Patidegib Topical Gel, 2%; Patidegib Topical Gel, 2%; Other Names: IP
Active Comparator: Patidegib Topical Gel, Vehicle; Participants will be randomized (1:1) to receive Patidegib Topical Gel, Vehicle for 9 months	Drug: Patidegib Topical Gel, Vehicle; Patidegib Topical Gel, Vehicle; Other Names: IP, Vehicle

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of new surgically eligible BCCs (nSEBs)	Baseline through Month 9

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of treatment emergent adverse events assessed with means and standard errors or proportions	Baseline through Month 9

Collaborators and Investigators

• Sponsor: Sol-Gel Technologies, Ltd.
• Investigators: Study Director: VP, Clinical Operations, PellePharm, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2019
• Primary Completion (Actual): April 23, 2021
• Study Completion (Actual): April 23, 2021

Study Registration Dates

• First Submitted: November 1, 2019
• First Submitted That Met QC Criteria: November 5, 2019
• First Posted (Actual): November 7, 2019

Study Record Updates

• Last Update Posted (Actual): May 31, 2023
• Last Update Submitted That Met QC Criteria: May 29, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Basal Cell Nevus Syndrome; BCC; Basal Cell Carcinoma; High Frequency Basal Cell Carcinoma; non-gorlin; HF-BCC; Bazex-Dupré-Christol Syndrome; Rombo Syndrome; Oley Syndrome; Xeroderma Pigmentosum (XP) Syndrome; SEB; Hedgehog inhibitor; Surgically Eligible Basal Cell Carcinoma; patidegib; HFBCC; HHI
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Basal Cell; Carcinoma; Carcinoma, Basal Cell; Antihypertensive Agents; Veratrum Alkaloids
• Other Study ID Numbers: Pelle-926-203

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No