QRX003 4% lotion for people with Netherton syndrome

A Single Center, Open Label, Expanded Access Study of QRX003 Lotion in Subjects With Netherton Syndrome

This trial will try QRX003 4% lotion applied twice daily to affected skin to see if it improves symptoms in people aged 14 and older with Netherton syndrome.

Quick facts

• Phase: PHASE2; PHASE3
• Study type: Interventional
• Enrollment: 8 (estimated)
• Ages: 14 Years and up
• Sex: All
• Sponsor: Quoin Pharmaceuticals (industry)
• Locations: 1 site (Chicago, Illinois)
• Trial ID: NCT06953466 on ClinicalTrials.gov

What this trial studies

This open-label expanded access protocol gives QRX003 4% lotion to participants with Netherton syndrome, applied topically to affected skin (excluding the scalp) twice daily for three months. Participants visit the clinic every 4–6 weeks for clinical exams, safety monitoring, and to review dosing diaries. Outcomes include changes in diseased skin area, clinician and patient itch/discomfort scores, safety events, and the proportion needing rescue therapy. Genetic confirmation of SPINK5 is required if not already available, and subjects must be on a stable topical regimen before enrollment.

Who should consider this trial

Good fit: People aged 14 and older with a clinical diagnosis of Netherton syndrome (or confirmed SPINK5 mutation), in generally good health, not pregnant, and on a stable topical treatment regimen are ideal candidates.

Not a fit: People under 14, pregnant individuals, those with unstable medical conditions, or those whose disease is limited to the scalp (which is not treated) may not receive benefit from this topical treatment.

Why it matters

Potential benefit: If successful, QRX003 could reduce diseased skin area, lessen itch and discomfort, and lower the need for rescue therapies in people with Netherton syndrome.

How similar studies have performed: This is a relatively novel investigational topical approach for Netherton syndrome with limited prior clinical data specific to QRX003.

Eligibility criteria

Inclusion Criteria:

1. Subject is a male or non-pregnant female at least 14 years of age.
2. 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.
3. 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.
4. 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.
5. 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

Exclusion Criteria:

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

Where this trial is running

Chicago, Illinois

• Northwestern Memorial Hospital — Chicago, Illinois, United States (RECRUITING)

Study contacts

• Study coordinator: TI Clinical Research

Conditions: Netherton Syndrome