Testing QRX003 lotion for treating Netherton Syndrome

A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome

This study is testing a new lotion called QRX003 to see if it can help people with Netherton Syndrome feel better by reducing their skin symptoms.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 14 Years and up
• Sex: All
• Sponsor: Quoin Pharmaceuticals (Industry-sponsored)
• Locations: 5 sites (San Diego, California and 4 other locations)
• Trial ID: NCT05521438 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety, tolerability, and efficacy of QRX003 lotion in individuals diagnosed with Netherton Syndrome. Participants will receive either a 2% or 4% concentration of the lotion applied once or twice daily, compared to a vehicle lotion. The study aims to determine how well these treatments work in alleviating symptoms associated with Netherton Syndrome. Eligible participants must be at least 14 years old and have confirmed lesions in the treatment area.

Who should consider this trial

Good fit: Ideal candidates are males or non-pregnant females aged 14 and older with a clinical diagnosis of Netherton Syndrome.

Not a fit: Patients who are pregnant, lactating, or planning to become pregnant during the study will not benefit from this trial.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life for patients suffering from Netherton Syndrome.

How similar studies have performed: Other studies have shown promise in treating skin conditions with topical therapies, but this specific approach for Netherton Syndrome is novel.

Eligibility criteria

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 has NS lesions in the Treatment Area (i.e., arms or lower legs).
* 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.

Exclusion Criteria:

* 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 Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females
* 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 4 weeks prior to Visit 2/Baseline.
* Subject has used systemic prescription treatment for NS within 4 weeks prior to Visit 2/Baseline.
* Subject has used systemic biologic therapy for NS.
* Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
* Subject has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
* Subject is currently enrolled in an investigational drug, biologic, or device study.
* Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2/Baseline.

Where this trial is running

San Diego, California and 4 other locations

• Site #1 — San Diego, California, United States (Recruiting)
• Site #4 — Indianapolis, Indiana, United States (Recruiting)
• Site #5 — Quincy, Massachusetts, United States (Recruiting)
• Site #3 — Charleston, South Carolina, United States (Recruiting)
• Site #2 — San Antonio, Texas, United States (Recruiting)

Study contacts

• Study coordinator: TI Clinical Research

Conditions Netherton Syndrome