4% QRX003 lotion for Netherton syndrome skin

A Multicenter, Open-label Study of QRX003 Lotion in Participants With Netherton Syndrome

This trial will test whether applying 4% QRX003 lotion twice daily for 12 weeks improves skin symptoms in people aged 4 and older with Netherton syndrome.

Quick facts

• Phase: PHASE2; PHASE3
• Study type: Interventional
• Enrollment: 16 (estimated)
• Ages: 4 Years and up
• Sex: All
• Sponsor: Quoin Pharmaceuticals (industry)
• Locations: 5 sites (Fremont, California and 4 other locations)
• Trial ID: NCT07538583 on ClinicalTrials.gov

What this trial studies

This phase 2/3, multicenter, open-label study administers 4% QRX003 lotion twice daily for 12 weeks to Netherton syndrome–affected skin across approximately 50% or more body surface area, excluding the scalp. Participants aged 4 years and older will be enrolled at U.S. clinical sites and monitored for safety, tolerability, and signs of skin improvement. There is no placebo arm, and outcomes will include investigator- and patient-reported measures of skin signs and symptoms plus adverse event monitoring. Women of childbearing potential must use effective contraception and have negative pregnancy tests per protocol.

Who should consider this trial

Good fit: People aged 4 years or older with clinically diagnosed Netherton syndrome who have affected skin covering roughly half the body or more (excluding the scalp) and who can comply with twice-daily topical application and study visits.

Not a fit: Patients with disease limited to the scalp, those unable to apply a topical twice daily or attend study visits, and pregnant women or others with contraindications are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, QRX003 could reduce scaling and inflammation, strengthen the skin barrier, and improve quality of life for people with Netherton syndrome.

How similar studies have performed: Topical approaches for Netherton syndrome are relatively novel with limited published clinical data, so this specific lotion represents a largely untested therapeutic strategy in larger trials.

Eligibility criteria

Inclusion Criteria:

1. Participant is male or female and at least 4 years of age.
2. 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.
3. 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.
4. 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:

1. Participant is pregnant, lactating, or is planning to become pregnant during the study.
2. Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.
3. Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.
4. Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2/Baseline.
5. Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
6. 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.
7. Participant has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
8. 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.
9. 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.
10. 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.
11. Participant has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
12. Participant is currently enrolled in an investigational drug, biologic, or device study.

Where this trial is running

Fremont, California and 4 other locations

• Center for Dermatology Clinical Research — Fremont, California, United States (RECRUITING)
• Yale University School of Medicine — New Haven, Connecticut, United States (NOT_YET_RECRUITING)
• Dawes Fretzin Clinical Reseacrh Group — Indianapolis, Indiana, United States (RECRUITING)
• Beacon Clinical Research — Quincy, Massachusetts, United States (RECRUITING)
• Texas Dermatology and Laser Specialists — San Antonio, Texas, United States (RECRUITING)

Study contacts

• Study coordinator: TI Clinical Research

Conditions: Netherton Syndrome