Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome (NCT06953466) | Clinical Trial Compass
RecruitingPhase 2/3
Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
United States8 participantsStarted 2025-07
Plain-language 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.
Who can participate
Age range
14 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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 , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline.
. Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.
. Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.
. Subject is on a stable treatment regimen including topical therapy for NS prior to baseline that is expected to remain stable for the duration of the study
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Proportion of subjects with 1-point reduction on IGA
. Subject is pregnant, lactating, or is planning to become pregnant during the study.
. Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.
. Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
. Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
. Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
. Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
. Subject has used ultraviolet phototherapy within the Treatment Area within 10 weeks prior to Visit 2/Baseline.
. Subject has used topical prescription treatment in the Treatment Area within 10 weeks prior to Visit 2/Baseline.