Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome

A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome

This study has been designed to determine the safety, tolerability and efficacy of QRX003 lotion 2%, 4% QAM or 4% BID in subjects with Netherton Syndrome (NS) in comparison to vehicle

Study Overview

• Status: Recruiting
• Conditions: Netherton Syndrome
• Intervention / Treatment: Drug: QRX003-2% Lotion; Drug: Vehicle; Drug: QRX003-4% Lotion QAM; Drug: QRX003-4% Lotion BID

• Study Type: Interventional
• Enrollment (Estimated): 30
• 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
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Site #3
      • Contact: Oleg G Khatsenko; Phone Number: 166 858-571-1800; Email: okhatsenko@therapeuticsinc.com
  • Texas
    • San Antonio, Texas, United States, 72218
      • 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.

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 Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females
• 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 systemic prescription treatment for NS within 4 weeks prior to Visit 2/Baseline.
• Subject has used systemic biologic therapy for NS.
• 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: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Vehicle Lotion; Subjects will apply test article once daily in the morning (QAM) for 12 weeks	Drug: Vehicle; Vehicle Lotion
Experimental: QRX003-2% QAM; Subjects will apply test article once daily in the morning (QAM) for 12 weeks	Drug: QRX003-2% Lotion; QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)
Experimental: QRX003-4% QAM; Subjects will apply test article once daily in the morning (QAM) for 12 weeks	Drug: QRX003-4% Lotion QAM; 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 BID; QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy Endpoints-1-point 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
Efficacy Endpoints-2-point IGA	Proportion of subjects with 2-point reduction in IGA from Baseline.	Up to week 16
Efficacy Endpoints-NS Sign	Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.	Up to week 16
Efficacy Endpoints-BSA	Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area	Up to week 16
Efficacy Endpoints-WI-NRS score	Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".	Up to week 16
Efficacy Endpoints-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
Efficacy Endpoints-Rescue Therapy	Proportion of subjects requiring rescue therapy	Up to week 16
Safety Endpoints-AEs	Any local and systemic AEs (Adverse Events)/serious AEs	Up to week 16
Safety Endpoints-LSR	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): June 23, 2022
• Primary Completion (Estimated): January 31, 2025
• Study Completion (Estimated): January 31, 2025

Study Registration Dates

• First Submitted: August 17, 2022
• First Submitted That Met QC Criteria: August 26, 2022
• First Posted (Actual): August 30, 2022

Study Record Updates

• Last Update Posted (Estimated): April 15, 2024
• Last Update Submitted That Met QC Criteria: April 11, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Disease; Congenital Abnormalities; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Abnormalities; Abnormalities, Multiple; Keratosis; Ichthyosiform Erythroderma, Congenital; Ichthyosis; Syndrome; Netherton Syndrome
• Other Study ID Numbers: CL-QRX003-001

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