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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Experimental |
| |
| Vehicle | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PTX-022 | Drug | QTORIN 3.9 % Sirolimus Topical Gel |
| |
| Vehicle comparator |
| 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 |
| 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 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David Bickers, MD | Columbia University | Study Chair |
| Joyce Teng, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Scottsdale | Arizona | 85259 | United States | ||
| Burke Pharmaceuticals Research |
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| Label | URL |
|---|---|
| Sign-up to be contacted about participation in the clinical trial | View source |
| Gorlin Syndrome Alliance | View source |
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| Drug |
Vehicle comparator topical gel |
|
| Hot Springs |
| Arkansas |
| 71913 |
| United States |
| Stanford Univeristy | Palo Alto | California | 94304 | United States |
| Park Avenue Dermatology | Orange Park | Florida | 32073 | United States |
| Ameriderm Research | Ormond Beach | Florida | 32174 | United States |
| Gwinett Clinical Research Center | Snellville | Georgia | 30078 | United States |
| Clinical Coordinating Center (Minnesota) | New Brighton | Minnesota | 55112 | United States |
| Mayo Clinic - Minnesota | Rochester | Minnesota | 55905 | United States |
| St. Louis University | St Louis | Missouri | 63110 | United States |
| Duke University | Durham | North Carolina | 27705 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| DermResearch Inc | Austin | Texas | 78759 | United States |
| University of Utah | Murray | Utah | 84107 | United States |
| Virginia Clinical Research, Inc. | Norfolk | Virginia | 23502 | United States |
| St. Pancras Clinical Research | London | United Kingdom |
| Salford Foundation Trust | Salford | M6 8HD | United Kingdom |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Dec 23, 2024 | Jan 15, 2025 | 6 |
| ID | Term |
|---|---|
| D001478 | Basal Cell Nevus Syndrome |
| D002280 | Carcinoma, Basal Cell |
| ID | Term |
|---|---|
| D009807 | Odontogenic Cysts |
| D007570 | Jaw Cysts |
| D001845 | Bone Cysts |
| D003560 | Cysts |
| D009369 | Neoplasms |
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009370 | Neoplasms by Histologic Type |
| D018295 | Neoplasms, Basal Cell |
| D009386 | Neoplastic Syndromes, Hereditary |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D007571 | Jaw Diseases |
| D009057 | Stomatognathic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
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