Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome

A Multicenter, Open Label Study of QRX003 Lotion in Subjects With Netherton Syndrome

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

Study Overview

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

• Study Type: Interventional
• Enrollment (Estimated): 20
• 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
  • California
    • San Diego, California, United States, 92123
      • Recruiting
      • Site #1
      • Contact: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com
  • Indiana
    • Indianapolis, Indiana, United States, 46250
      • Recruiting
      • Site #4
      • Contact: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com
  • Massachusetts
    • Quincy, Massachusetts, United States, 02169
      • Recruiting
      • Site #5
      • Contact: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com
  • Texas
    • San Antonio, Texas, United States, 72218
      • Not yet recruiting
      • Site #2
      • Contact: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 for the duration of the study

Exclusion Criteria:

• Subject is pregnant, lactating, or is planning to become pregnant during the study.
• 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.
• Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
• Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
• Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
• Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
• Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
• Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
• Subject has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
• Subject is currently enrolled in an investigational drug, biologic, or device study.
• 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: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: QRX003, 4% QAM; Subjects will apply test article once daily in the morning (QAM) for 12 weeks	Drug: QRX003, 4% Lotion; QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Experimental: QRX003, 4% BID; Subjects will apply test article twice daily (BID) for 12 weeks	Drug: QRX003, 4% Lotion; QRX003Topical 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	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
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
Assessment of subject satisfaction with treatment based on TSQM	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.	Up to week 16
Proportion of subjects requiring rescue therapy	Proportion of subjects requiring rescue therapy	Up to week 16
Safety Assessment-AEs	Any local and systemic AEs (Adverse Events)/serious AEs	Up to week 16
Safety Assessment-LSRs	Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.	Up to week 16

Collaborators and Investigators

• Sponsor: Quoin Pharmaceuticals
• Investigators: Study Director: Tony Andrasfay, Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 14, 2023
• Primary Completion (Estimated): December 30, 2025
• Study Completion (Estimated): December 30, 2025

Study Registration Dates

• First Submitted: March 16, 2023
• First Submitted That Met QC Criteria: March 16, 2023
• First Posted (Actual): March 29, 2023

Study Record Updates

• Last Update Posted (Actual): April 2, 2025
• Last Update Submitted That Met QC Criteria: March 28, 2025
• Last Verified: March 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-QRX003-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

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