CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome

Study Overview
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Brief Summary
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A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.

Official Title
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A Multicenter, Phase 2B, Randomized, Double-Blind, Stratified, Vehicle-Controlled Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel In Prevention Of BCCs In Patients With Gorlin Syndrome

Conditions
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BCCs in Gorlin Syndrome Patients

Intervention / Treatment
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* Drug: PTX-022
* Drug: Vehicle comparator


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Adults, 18 years or older Meet diagnostic criteria for Gorlin Syndrome Willing to avoid application of a non-study topical medication to the face (prescription or over the counter) during the study. Willing to forego treatment of BCCs with anything other than the study medication except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. During the trial, the only allowed form of BCC treatment is surgical. Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily Key Exclusion Criteria: Recently participated in a clinical trial evaluating an investigational product for the treatment of BCCs or Gorlin Syndrome within 3 months prior to to starting study medication. Participant can start the study screening period prior to completing the 3 month washout. Recently used topical or systemic (oral) treatment that might interfere with the evaluation of the study medication.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Active<br> | Drug: PTX-022<br>* QTORIN 3.9 % Sirolimus Topical Gel<br>|
| Placebo Comparator: Vehicle<br> | Drug: Vehicle comparator<br>* Vehicle comparator topical gel<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms |  | Month 6 | 
| Incidence of dermatological, treatment emergent adverse events after treatment with active |  | Month 6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face. |  | Month 6 | 
| Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face. |  | Month 6 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gorlin Syndrome, Basal Cell Carcinoma, Nevoid basal cell carcinoma syndrome, Basal cell nevus syndrome BCNS, Gorlin Syndrome Alliance, Prevention, Topical, Sirolimus