NCT05789056 · Quoin Pharmaceuticals
Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome
What this study is about
To assess the safety, tolerability, and effectiveness of QRX003 lotion (4%) when added to the usual treatment treatment regimen, including systemic therapy in subjects with Netherton syndrome
View original scientific description
To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects with Netherton syndrome
Interventions
DRUG
QRX003, 4% Lotion
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Primary outcome measures
Proportion of subjects with 1-point reduction on IGA
Time frame: Up to week 16
Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe.
Proportion of subjects with 2-point reduction on IGA
Time frame: Up to week 16
Proportion of subjects with 2-point reduction in IGA from Baseline.
NS surface area change
Time frame: Up to week 16
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
WI-NRS score change
Time frame: Up to week 16
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score ≥4. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".
Assessment of subject satisfaction with treatment based on TSQM
Time frame: Up to week 16
Assessment of subject satisfaction with treatment based on the Treatment Satisfaction Questionnaire for Medication (TSQM). The TSQM measures a subject's level of satisfaction or dissatisfaction with the treatment. Composite scores can range from 0 to 100; higher scores indicate better satisfaction.
Proportion of subjects requiring rescue therapy
Time frame: Up to week 16
Proportion of subjects requiring rescue therapy
Safety Assessment-AEs
Time frame: Up to week 16
Any local and systemic AEs (Adverse Events)/serious AEs
Safety Assessment-LSRs
Time frame: Up to week 16
Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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.
- Subject is on a stable treatment regimen including systemic therapy for NS prior to baseline that is expected to remain stable
Where
- San Diego, California
- Indianapolis, Indiana
- Quincy, Massachusetts
- San Antonio, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 2, 2025 · Source of record for eligibility and locations