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

A Multicenter, Randomized, Double blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects with Basal Cell Nevus Syndrome - PATIDEGIB study

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-001462-42-DK

Enrollment

150

Registered

2019-02-15

Start date

2019-05-22

Completion date

Unknown

Last updated

2021-04-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Basal Cell Nevus Syndrome MedDRA version: 20.1 Level: LLT Classification code 10004151 Term: Basal cell nevus syndrome System Organ Class: 100000004850

Interventions

Product Name: Patidegib Topical Gel 2% Pharmaceutical Form: Gel INN or Proposed INN: Patidegib CAS Number: 1169829-40-6 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Conc

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Key Inclusion criteria: 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 =65 years) yes F.1.3.1 Number of subjects for this age range 75

Exclusion criteria

Exclusion criteria: Key Exclusion criteria: 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.

Design outcomes

Primary

Measure	Time frame
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	—

Measure

Time frame

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

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Secondary

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

Measure

Time frame

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

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Countries

Belgium, Canada, Denmark, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

Public ContactKrystyna Grycz

PellePharm, Inc.

kgrycz@pellepharm.com+18443325161

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026