Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome

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

The goal of this clinical trial (via an expanded access Treatment Protocol) is to learn if QRX003 (an investigational drug) applied topically to the skin (including up to the entire body [except the scalp]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. The main questions the trial aims to answer are:

1. Does QRX003 impact the clinical presentation of NS in adults and minors by improving the clinical symptoms (diseased skin area, itch, and discomfort; based on clinical scoring, subject self-assessment, and other criteria)?
2. What medical problems do participants have when taking QRX003?
3. What percent of subjects will require rescue therapy?

Participants will:

Take drug QRX003 twice daily (applied topically to all affected areas of the body excluding the scalp) for 3 months, visit the clinic once every 4-6 weeks for checkups and tests, and to keep a dosing diary that records the times they applied the drug.

Study Overview

• Status: Recruiting
• Conditions: Netherton Syndrome
• Intervention / Treatment: Drug: QRX003, 4% Lotion

• Study Type: Interventional
• Enrollment (Estimated): 8
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: TI Clinical Research; Phone Number: 147 858-571-1800; Email: clinicalresearch@therapeuticsinc.com
• Study Contact Backup: Name: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Recruiting
      • Northwestern Memorial Hospital
      • Contact: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: QRX003, 4% BID; Subjects will apply test article twice daily (BID) for 12 weeks	Drug: QRX003, 4% Lotion; QRX003 Topical Lotion containing 4% active drug (serine protease inhibitor)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of subjects with 1-point reduction on IGA	Description: 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.	Up to week 16

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of subjects with 2-point reduction on IGA	Proportion of subjects with 2-point reduction in IGA from Baseline.	Up to week 16
NS surface area change	Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area	Up to week 16
WI-NRS score change	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".	Up to week 16
Safety Assessment-AEs	Any local and systemic AEs (Adverse Events)/serious AEs	Up to week 16

Collaborators and Investigators

• Sponsor: Quoin Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2025
• Primary Completion (Estimated): July 30, 2026
• Study Completion (Estimated): July 30, 2026

Study Registration Dates

• First Submitted: April 7, 2025
• First Submitted That Met QC Criteria: April 23, 2025
• First Posted (Actual): May 1, 2025

Study Record Updates

• Last Update Posted (Actual): July 20, 2025
• Last Update Submitted That Met QC Criteria: July 16, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Genetic Diseases, Inborn; Disease; Infant, Newborn, Diseases; Skin Diseases; Congenital Abnormalities; Abnormalities, Multiple; Skin Diseases, Genetic; Skin Abnormalities; Keratosis; Ichthyosiform Erythroderma, Congenital; Ichthyosis; Syndrome; Netherton Syndrome
• Other Study ID Numbers: CL-QRX003-003; Sponsor Funded (Other Identifier: Quoin Pharmaceuticals, Ltd.)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No