Expanded Access Study of QRX003 Lotion for Treating Netherton Syndrome in People Aged 14 and Older

What this Trial Is About

Researchers are studying whether QRX003, an investigational topical lotion, can help treat Netherton syndrome, a genetic skin condition, in both adults and minors aged 14 years and older. The trial also aims to understand the safety of QRX003 and how it affects symptoms like diseased skin area, itching, and discomfort through clinical scores and patient self-assessments. This is an open-label expanded access study sponsored by Quoin Pharmaceuticals. Participants will apply QRX003 lotion containing 4% active drug twice daily to all affected skin areas except the scalp for a period of 12 weeks. The study involves only one treatment group receiving QRX003, with no placebo or comparator. The lotion is applied topically, and the participant follows a stable treatment regimen prior to starting the study. Participants will visit the clinic every 4 to 6 weeks during the 3-month treatment to undergo checkups and tests. They will keep a dosing diary to record each application of the lotion. The main outcome measured is the proportion of subjects who achieve at least a 1-point improvement on the Investigators Global Assessment IGA scale by week 16. Safety monitoring and assessment of any need for rescue therapy are also part of the study. Total participation duration is approximately 3 months.

Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome