NCT07538583 · Quoin Pharmaceuticals
Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
What this study is about
This is a conducted at multiple hospitals, where both patients and doctors know the treatment given study to evaluate the safety, tolerability, and effectiveness of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA) or more.
View original scientific description
This is a multicenter, open-label study to evaluate the safety, tolerability, and efficacy of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA) or more.
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
- Participant is male or female and at least 4 years of age.
- Participant has provided written informed consent/assent. Based on the participant's age and local regulations , a participant under legal age of consent must provide written informed assent and be accompanied by the parent or legal guardian at the time of assent/consent signing. The parent or legal guardian must provide informed consent for the participant. If a participant reaches the age at which consent is required by local regulations during the study, the participant must provide written informed consent at that time to continue study participation.
- Female participants must either not have experienced menarche, be postmenopausal , or be surgically sterile , or use a highly effective method of birth control , for the duration of the study and for 3 months following EOT. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline. Males must use a condom in addition to having their female partner use a highly effective method of birth control.
- Participant has a genetically confirmed diagnosis of NS prior to Visit 2/Baseline. Participants without existing results must undergo genetic testing at Visit 1/Screening, which must confirm the diagnosis of NS in order for the participants to be enrolled in the study.
Exclusion criteria
- Participant is pregnant, lactating, or is planning to become pregnant during the study.
- Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.
- Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.
- Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2/Baseline.
- Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
- Participant has ongoing or recent history of any other uncontrolled and/or clinically significant systemic disease or condition which, in the opinion of the investigator, should exclude participation in the study.
- Participant has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
- Participant has used systemic non-biologics prescription treatment for NS or that in the opinion of the investigator may affect the participant's NS (examples include, but are not limited to antibiotics, retinoids, etc.) within 4 weeks prior to Visit 2/Baseline.
- Participant has used systemic biologic therapy for NS or that in the opinion of the investigator may affect the participant's NS within 5 half-lives of the biologic therapy prior to Visit 2/Baseline. Published or documented half-life of the product provided by the commercial supplier or Sponsor should be used to establish this value.
- Participant has used topical prescription treatments, including steroids (examples include, but are not limited to keratolytics, steroids, retinoids, etc.) in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
- Participant has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
- Participant is currently enrolled in an investigational drug, biologic, or device study.
Where
- Fremont, California
- New Haven, Connecticut
- Indianapolis, Indiana
- Quincy, Massachusetts
- San Antonio, Texas
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 21, 2026 · Source of record for eligibility and locations