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This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Active Comparator |
| |
| Vehicle | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QTORIN 3.9% rapamycin anhydrous gel | Drug | QTORIN 3.9% rapamycin anhydrous gel |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Patient Global Assessment of Activities Difficulty Scale | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Pain at its worst as assessed by numerical rating scale | 6 months | |
| PROMIS physical function | 6 months | |
| Patient global impression of severity (PGI-S) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Amy Paller, MD | Northwestern University | Study Chair |
| Joyce Teng, MD | Stanford University | Principal Investigator |
| David Hansen, MD | University of Utah | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Palo Alto | California | 94304 | United States | ||
| Yale University |
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| Label | URL |
|---|---|
| PC Project | View source |
| Palvella Therapeutics, Inc. | View source |
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| Vehicle |
| Drug |
Matching vehicle (QTORIN anyhydrous gel) |
|
| 6 months |
| Patient global impression of change (PGI-C) | 6 months |
| Clinician global impression of severity (CGI-S) | 6 months |
| Clinician global impression of change (CGI-C) | 6 months |
| New Haven |
| Connecticut |
| 06519 |
| United States |
| Park Avenue Dermatology | Orange Park | Florida | 32073 | United States |
| Minnesota Clinical Study Center | Fridley | Minnesota | 55432 | United States |
| Oregon Health and Sciences University | Portland | Oregon | 97239 | United States |
| DermResearch, Inc. | Austin | Texas | 78759 | United States |
| University of Utah | Murray | Utah | 84107 | United States |
| Royal London Hospital | London | Whitechapel | E11FR | United Kingdom |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Dec 23, 2024 | Jan 16, 2025 | 4 |
| ID | Term |
|---|---|
| D053549 | Pachyonychia Congenita |
| ID | Term |
|---|---|
| D004476 | Ectodermal Dysplasia |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012868 | Skin Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009260 | Nail Diseases |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
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