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Diseases and Abnormalities at or Before Birth

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Carcinoma, Basal Cell

Basal Cell Nevus Syndrome

Nevus, Pigmented

Nevus

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Neoplasms, Basal Cell

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Nevoid Basal Cell Carcinoma Syndrome

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Sirolimus

PTX-022

Vehicle comparator

Kathy Goin

Study Coordinator

Tim Dugan

Elidia Tafoya

Dea Kemlage

Sandra Warrington

Colleen Ethridge

Patient Navigator

Addison Demer

Rhonda Lebbing

Connor Whatley

Karina Garcia

Sean Wetland

Erin Rivera

Stuart Ratcliff, MD

David Bickers, MD

Joyce Teng, MD

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.

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

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

Palvella Therapeutics, Inc.

Skin and Connective Tissue Diseases

Melanoma

Nevi and Melanomas

Antihypertensive Agents

Veratrum Alkaloids

Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.

Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.

Patidegib Topical Gel, 2%

Patidegib Topical Gel, Vehicle

VP, Clinical Operations

This is a global, 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 Gorlin syndrome. Participants will be required to apply the investigational product for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new BCCs that develop over the 12 month period.

An open-label, extension safety and tolerability study is planned for at least 12 months duration following the end of this study. All participants who complete the Month 12 Exit Visit having demonstrated adequate compliance with application of the Investigational Product (IP) without major Protocol Deviations (PDs) during the study will be eligible for participation in the extension study.

All participants will be contacted by phone approximately 30 days following the Exit or Discontinuation Visit to determine if the participant has experienced any new adverse events (AEs)/serious AEs (SAEs) since discontinuation/completion of study treatment.

Participants will be randomized 1:1 to receive Patidegib Topical Gel, 2%, or Vehicle (IP)

As a double-blinded study, the Investigators, the site staff, Sponsor, and the Clinical Monitor(s) will be blinded to the treatment assigned to individual participants.

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 (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

PellePharm, Inc.

Patidegib gel 2%, applied topically, once daily for 12 weeks (Cohort 1)

Patidegib gel 4%, applied topically, once daily for 12 weeks (Cohort 2)

Vehicle gel, applied topically, once daily for 12 weeks (Cohort 1)

Patidegib gel 2%, applied topically, twice daily for 12 weeks (Cohort 3)

Patidegib gel 4%, applied topically, twice daily for 12 weeks (Cohort 4)

Vehicle gel, applied topically, once daily for 12 weeks (Cohort 2)

Vehicle gel, applied topically, twice daily for 12 weeks (Cohort 3)

Vehicle gel, applied topically, twice daily for 12 weeks (Cohort 4)

Patidegib

Vehicle gel

Ervin Epstein, MD

This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle in patients with Basal Cell Carcinoma. One investigational center (metasite) in the United States will participate in this study. Approximately 36 subjects who meet the study entry criteria will be enrolled into one of four sequential cohorts. Within each cohort subjects will be randomized in a 2:1 ratio to receive active or vehicle gel.

This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle. Approximately 36 subjects who meet the study entry criteria will be enrolled into one of four sequential cohorts. As soon as one cohort has been completely enrolled, the next cohort will be enrolled. Each subject will treat no more than two previously untreated biopsy confirmed treatment-targeted nodular BCCs. If the subject has additional non-treatment targeted BCCs they can be treated surgically prior to or during the trial. Within each cohort subjects will be randomized in a 2:1 ratio to receive active or vehicle gel. The sequential cohorts will be:

* Cohort 1: patidegib gel 2% or vehicle, once daily
* Cohort 2: patidegib gel 4% or vehicle, once daily
* Cohort 3: patidegib gel 2% or vehicle, twice daily
* Cohort 4: patidegib gel 4% or vehicle, twice daily

The study drug will be applied topically to the treatment-targeted BCCs and a rim of adjacent skin for 12 weeks. Information on reported and observed adverse events (AEs) will be obtained at each visit. An abbreviated physical examination (PE) will be performed at Baseline and Week 12. The treatment-targeted BCCs will be identified by the Investigator at the Baseline visit and will be circled in ink at Baseline, Weeks 6 and 12 and photographed, and measured at all study visits (Baseline, Weeks 2, 6, 8, 10, and 12). Blood samples for complete blood count and serum chemistry and urine for urinalysis will be collected from subjects at Screening, Week 6, and Week 12. Subjects who terminate study participation early will be asked to complete all Week 12 assessments, as appropriate, prior to commencement of any alternative therapy for BCCs (if possible). Subjects who discontinue from the study during the treatment period will not be replaced.

Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas

Double-Blind, Dose Escalating, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas

The ISGTA is a scale with scores ranging from 0 (clear), 1 (almost clear), 2 (minimal residual tumor), to 3 (clearly visible tumor). The Investigator assessed each Baseline treatment-targeted SEB at Weeks 2, 6, 8, 10, and 12. SEBs were defined as clinically diagnosed BCC 5 to 20 mm in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. The percentage of Baseline treatment-targeted SEBs evaluated as being clear or almost clear at Week x (Week x = 2, 6, 8, 10, or 12) based on the ISGTA scale was calculated as follows: (Number of baseline treatment-targeted SEBs with ISGTA score of 0 or 1 at Week x) / (Number of Baseline treatment-targeted SEBs) \* 100. Missing data were imputed using LOCF. The percentage of responders achieving clear (0) or almost clear (1) on the ISGTA scale are presented by Week.

SEBs were defined as clinically diagnosed BCC 5 to 20 mm in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. The percent change in greatest diameters of Baseline treatment-targeted SEBs from Baseline to Week 12 was calculated as follows: (sum \[Baseline\] - sum \[Week 12\] / sum \[Baseline\]) \* 100), where sum = the greatest diameters of treatment-targeted SEBs.

All serious adverse events (SAEs) and all other non-serious adverse events (AEs) regardless of causality are located in the Reported AE Module. The number of AEs (including both serious and non-serious) considered related to study drug by the Investigator are presented below. Treatment-emergent AEs are those with an onset after use of study drug.

GLI1 change is a biomarker of the HH signaling pathway. A change in GLI1 mRNA levels reflect a change in the HH pathway. Surgically eligible basal cell carcinomas (SEBs) were defined as clinically diagnosed basal cell carcinoma (BCC) 5 to 20 millimeters (mm) in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. A single baseline BCC designated as a treatment-targeted tumor at Baseline was biopsied first at Baseline and again following 12 weeks of treatment. This was used to assess percent change in GLI1 mRNA levels as follows: (Baseline - Week 12) / Baseline \* 100, with positive numbers to represent increases and negative numbers to represent decreases. Any missing values were not imputed; all available data is summarized.

SEBs were defined as clinically diagnosed BCC 5 to 20 mm in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. The percent change in greatest diameters of treatment-targeted surgically eligible basal cell carcinomas (SEBs) from Baseline to Week 12 was calculated as follows: (sum \[Baseline\] - sum \[Week 12\] / sum \[Baseline\] \* 100), where sum = the greatest diameters of treatment-targeted SEBs and positive numbers to represent increases and negative numbers to represent decreases. Missing values were imputed using Last-Observation Carried Forward (LOCF).

SEBs were defined as clinically diagnosed BCC 5 to 20 mm in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. The percentage of tumors showing a complete or partial response at Week 12 was calculated as follows: (Number of baseline treatment-targeted SEBs showing complete or partial response at Week 12) / (Number of Baseline treatment-targeted SEBs) \* 100. Missing values were not imputed.Complete Response is determined when there is no longer any visible evidence of a lesion consistent with BCC at the site, and Partial Response is determined when although a BCC still remains at this site, it has demonstrated a visible decrease in size compared with baseline.

All enrolled participants who were randomized, received at least 1 confirmed dose of study drug, and had at least 1 post-baseline safety assessment.

Applied topically once daily for 12 weeks (Cohort 1)

Patidegib Gel 2% - Once Daily

Applied topically once daily for 12 weeks (Cohort 2)

Patidegib Gel 4% - Once Daily

Applied topically twice daily for 12 weeks (Cohort 3)

Patidegib Gel 2% - Twice Daily

Applied topically twice daily for 12 weeks (Cohort 4)

Patidegib Gel 4% - Twice Daily

Applied topically once or twice daily for 12 weeks (Cohorts 1, 2, 3, and 4)

Vehicle Gel

Total

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Weight

Participants who received at least 1 dose of study drug

Chief Medical Officer

Number of Treatment-emergent Adverse Events (Including Both Serious and Non-Serious) Causally Related to Study Drug

This is the analysis of combined patidegib gel 2% once daily and twice daily compared with vehicle gel at Week 12.

This is the analysis of combined patidegib gel 4% once daily and twice daily compared with vehicle gel at Week 12.

This is the analysis of all four patidegib gel treatment groups combined compared with vehicle gel at Week 12.

Participants who received at least 1 dose of study drug who had evaluable GLI1 mRNA data at Baseline and Week 12.

Molecular Efficacy: Percent Change From Baseline in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels at Week 12

Patidegib Gel 2% - Cohort 1

Vehicle Gel - Cohort 1

Patidegib Gel 4% - Cohort 2

Vehicle Gel - Cohort 2

Patidegib Gel 2% - Cohort 3

Vehicle Gel - Cohort 3

Patidegib Gel 4% - Cohort 4

Vehicle Gel - Cohort 4

Participants who received at least 1 dose of study drug.

Clinical Efficacy: Percent Change From Baseline in Tumor Size of Treatment-targeted SEBs at Week 12

Clinical Efficacy: Percentage of SEBs Showing Complete or Partial Response at Week 12 as Determined by Blinded Photographic Review

Week 2

Week 6

Week 8

Week 10

Week 12

Percentage of Treatment-targeted SEBs Achieving Clear or Almost Clear on the Investigator Static Global Tumor Assessment (ISGTA) Scale

Percent Change in Treatment-targeted SEBs Tumor Size From Baseline as Determined by Blinded Photographic Review

This is the analysis of all four patidegib gel treatment groups combined compared with combined vehicle gel at Week 12.

The ISGTA is a scale with scores ranging from 0 (clear), 1 (almost clear), 2 (minimal residual tumor), to 3 (clearly visible tumor). The Investigator assessed each Baseline treatment-targeted SEB at Week 12. SEBs were defined as clinically diagnosed BCC 5 to 20 mm in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. The percentage of Baseline treatment-targeted SEBs evaluated as being clear at Week 12 based on the ISGTA scale and BCC not present was calculated as follows: (Number of baseline treatment-targeted SEBs with ISGTA score of 0 and BCC not present at Week 12) / (Number of Baseline treatment-targeted SEBs) \* 100.

Vehicle gel, applied topically once or twice daily for 12 weeks (Cohorts 1, 2, 3, and 4)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
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	NCT04893486	BCCs in Gorlin ...	PTX-022 Vehicle compara...	18 Years -	Palvella Therapeutics, Inc.
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 (Gorlin Syndrome)	NCT03703310	Basal Cell Nevu...	Patidegib Topic... Patidegib Topic...	18 Years -	PellePharm, Inc.
Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas	NCT02828111	Basal Cell Carc...	Patidegib Vehicle gel	18 Years - 85 Years	PellePharm, Inc.
Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas	NCT02828111	Basal Cell Carc...	Patidegib Vehicle gel	18 Years - 85 Years	PellePharm, Inc.
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	NCT04893486	BCCs in Gorlin ...	PTX-022 Vehicle compara...	18 Years -	Palvella Therapeutics, Inc.

Spots Global Cancer Trial Database for basal cell nevus syndrome bcns