Actively Recruiting
Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
Led by Quoin Pharmaceuticals · Updated on 2025-07-20
8
Participants Needed
1
Research Sites
56 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject is a male or non-pregnant female at least 14 years of age.
- Females must be post-menopausal, surgically sterile, or use an effective method of birth control during the study and for 3 months after treatment.
- Women of childbearing potential must have a negative urine pregnancy test at screening and baseline visits.
- Subject has a clinical diagnosis of Netherton syndrome and agrees to genetic testing if no prior confirmation of SPINK5 mutation.
- Subject is in good general health without conditions that impair evaluation or increase risk.
- Subject is on a stable treatment regimen including topical therapy for Netherton syndrome expected to remain stable during the study.
You will not qualify if you...
- Subject is pregnant, lactating, or planning pregnancy during the study.
- Subject has skin conditions in the treatment area that interfere with evaluation or require conflicting therapies.
- Subject has active cancer except non-melanoma skin cancer outside the treatment area.
- Subject has diabetes except reasonably controlled non-insulin dependent diabetes mellitus.
- Subject has active infection at screening or serious infection within 30 days before baseline.
- Subject has HIV, hepatitis B or C, or active or latent tuberculosis.
- Subject used ultraviolet phototherapy in treatment area within 10 weeks before baseline.
- Subject used topical prescription treatments, including steroids, in treatment area within 10 weeks before baseline.
- Subject is enrolled in another investigational drug, biologic, or device study.
- Subject used investigational treatments within 30 days before baseline.
AI-Screening
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Trial Site Locations
Total: 1 location
1
Northwestern Memorial Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
Research Team
T
TI Clinical Research
CONTACT
O
Oleg G Khatsenko
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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