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

Netherton Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05521438
• Other study ID #: CL-QRX003-001
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: June 23, 2022
• Est. completion date: January 31, 2025

Study information

• Verified date: April 2024
• Source: Quoin Pharmaceuticals
• Contact: TI Clinical Research
• Phone: 858-571-1800
• Email: clinicalresearch[@]therapeuticsinc.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: January 31, 2025
• Est. primary completion date: January 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Netherton Syndrome
• Syndrome

Intervention

Drug:
• QRX003-2% Lotion
QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)
• QRX003-4% Lotion QAM
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
• Vehicle
Vehicle Lotion
• QRX003-4% Lotion BID
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)

Locations

Country	Name	City	State
United States	Site #3	Charleston	South Carolina
United States	Site #4	Indianapolis	Indiana
United States	Site #5	Quincy	Massachusetts
United States	Site #2	San Antonio	Texas
United States	Site #1	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Quoin Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Efficacy Endpoints-2-point IGA	Proportion of subjects with 2-point reduction in IGA from Baseline.	Up to week 16
Primary	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
Primary	Efficacy Endpoints-BSA	Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area	Up to week 16
Primary	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
Primary	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
Primary	Efficacy Endpoints-Rescue Therapy	Proportion of subjects requiring rescue therapy	Up to week 16
Primary	Safety Endpoints-AEs	Any local and systemic AEs (Adverse Events)/serious AEs	Up to week 16
Primary	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