Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
brief summary
The goal of this clinical trial (via an expanded access Treatment Protocol) is to learn if QRX003 (an investigational drug) applied topically to the skin (including up to the entire body \[except the scalp\]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. The main questions the trial aims to answer are: 1. Does QRX003 impact the clinical presentation of NS in adults and minors by improving the clinical symptoms (diseased skin area, itch, and discomfort; based on clinical scoring, subject self-assessment, and other criteria)? 2. What medical problems do participants have when taking QRX003? 3. What percent of subjects will require rescue therapy? Participants will: Take drug QRX003 twice daily (applied topically to all affected areas of the body excluding the scalp) for 3 months, visit the clinic once every 4-6 weeks for checkups and tests, and to keep a dosing diary that records the times they applied the drug.
official title
A Single Center, Open Label, Expanded Access Study of QRX003 Lotion in Subjects With Netherton Syndrome