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PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial.
The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PTX-022 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PTX-022 | Drug | QTORIN rapamycin 3.9% anhydrous gel |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment emergent adverse events | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic parameters | AUC area under the sirolimus blood concentration-time profile | Prior to dose, 12 hours and 24 hours |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Emily Cook | Contact | 267-738-6366 | emily.cook@palvellatx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Minnesota Clinical Study Center | Recruiting | New Brighton | Minnesota | 55112 | United States |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jan 2, 2025 | Jan 27, 2025 | 1 |
| 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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