A 1-year Study of Topical Application of a Gel Containing the Investigational Active Ingredient Patidegib versus the same Gel Without Patidegib (Placebo) for the Prevention of New Facial Skin Tumors (Basal Cell Carcinomas) in People with Gorlin Syndrome

Phase 1

Conditions: Basal Cell Nevus Syndrome
MedDRA version: 20.1Level: LLTClassification code 10004151Term: Basal cell nevus syndromeSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2018-001462-42-DK

Lead Sponsor: PellePharm, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 150

Inclusion Criteria

Key
1. The subject must be at least 18 years old at the Screening visit.
2. The subject must meet diagnostic criteria for Gorlin syndrome (Inclusion Criteria 3).
3. The subject must have had at least 10 (with at least 3 on the face) clinically typical BCCs present within 24 months prior to Randomization (Baseline/Day 1). Additionally, the subject must have at least 2 BCCs with longest diameter <5 mm present on the face prior to randomization.
4. 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 75
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 75

Exclusion Criteria

Key
1. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
2. The subject is known to have a hypersensitivity to any of the ingredients in the IP.
3. The subject has uncontrolled systemic disease.
4. 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 & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to assess the number of new BCCs in the 2 arms (Patidegib Topical Gel, 2%, and Vehicle) when applied twice daily to the face of subjects with Gorlin syndrome;Secondary Objective: The secondary objectives of the study is to assess the safety and tolerability of Patidegib Topical Gel, 2%, in subjects treated twice daily for 12 months ;Primary end point(s): The primary endpoint is the number of nSEBs at 12 months.;Timepoint(s) of evaluation of this end point: Month 12

Secondary Outcome Measures

Name	Time	Method
Timepoint(s) of evaluation of this end point: 1. Month 12 <br>2. Month 12 <br>3. Month 12 <br>4. Month 9<br>5. Month 6<br>6. Month 12;Secondary end point(s): Secondary Endpoints:<br>1. The number of nSEBs per subject by Month 12.<br>2. The proportion of subjects developing =2 facial new BCCs by Month 12.<br>3. The proportion of subjects developing =1 facial new BCCs by Month 12.<br>4. The number of qualifying new BCCs per subject by Month 9.<br>5. The number of qualifying new BCCs per subject by Month 6.<br>6. aBCCdex change in Lesion Symptoms scale score from Baseline to Month 12

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