Long-Term Safety and Drug Behavior Study of QTORIN Rapamycin 3.9% Gel for Adults with Pachyonychia Congenita Previously in PALV-05 Trial

What this Trial Is About

Researchers are evaluating the safety and pharmacokinetics of QTORIN rapamycin 3.9% anhydrous gel, called PTX-022, in adults with Pachyonychia Congenita PC who have specific keratin gene mutations and who previously participated in the PALV-05 VAPAUS trial. This open-label treatment study is conducted at multiple centers to better understand long-term exposure effects of the gel in this patient group. Participants receive the study drug PTX-022 applied as a gel, with the research focusing on monitoring safety and how the drug moves through and is processed by the body over time. The study does not have a placebo group or masking, and treatment details are based on prior enrollment and dosing established in earlier trials. Participants are monitored for treatment-emergent adverse events over six months, with pharmacokinetic measurements taken before dosing and at 12 and 24 hours after application. The trial assesses drug safety and how the body absorbs and metabolizes the gel during this period, helping researchers understand long-term use implications.

A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita