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NCT Number: NCT05521438

Safety, Tolerability and Efficacy 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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Netherton Syndrome Abnormalities, Multiple Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Ichthyosiform Erythroderma, Congenital Ichthyosis Infant, Newborn, Diseases Keratosis Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

14 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Site #1, San Diego, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

QRX003-2% Lotion

Drug

QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)

QRX003-4% Lotion QAM

Drug

QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)

Vehicle

Drug

Vehicle Lotion

QRX003-4% Lotion BID

Drug

QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)

Primary outcomes

  1. Efficacy Endpoints-1-point IGA

    Time frame: Up to week 16

    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.

  2. Efficacy Endpoints-2-point IGA

    Time frame: Up to week 16

    Proportion of subjects with 2-point reduction in IGA from Baseline.

  3. Efficacy Endpoints-NS Sign

    Time frame: Up to week 16

    Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.

  4. Efficacy Endpoints-BSA

    Time frame: Up to week 16

    Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area

  5. Efficacy Endpoints-WI-NRS score

    Time frame: Up to week 16

    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".

  6. Efficacy Endpoints-TSQM

    Time frame: Up to week 16

    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.

  7. Efficacy Endpoints-Rescue Therapy

    Time frame: Up to week 16

    Proportion of subjects requiring rescue therapy

  8. Safety Endpoints-AEs

    Time frame: Up to week 16

    Any local and systemic AEs (Adverse Events)/serious AEs

  9. Safety Endpoints-LSR

    Time frame: Up to week 16

    Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.

Sponsors and collaborators

Lead sponsor

Quoin Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2025
Study completion
2026
First posted
Aug 30, 2022
Registry last updated
Mar 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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