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NCT Number: NCT05789056

Open Label, Safety and Efficacy 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

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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

Loading trial locations.

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.
  • 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.

Treatment and study plan

QRX003, 4% Lotion

Drug

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

Primary outcomes

  1. Proportion of subjects with 1-point reduction on 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. Proportion of subjects with 2-point reduction on IGA

    Time frame: Up to week 16

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

  3. NS surface area change

    Time frame: Up to week 16

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

  4. WI-NRS score change

    Time frame: Up to week 16

    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".

  5. Assessment of subject satisfaction with treatment based on 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.

  6. Proportion of subjects requiring rescue therapy

    Time frame: Up to week 16

    Proportion of subjects requiring rescue therapy

  7. Safety Assessment-AEs

    Time frame: Up to week 16

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

  8. Safety Assessment-LSRs

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Oleg G Khatsenko

CONTACT

[email protected]

858-571-1800 ext. 166

TI Clinical Research

CONTACT

[email protected]

858-571-1800 ext. 147

Sponsors and collaborators

Lead sponsor

Quoin Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Mar 29, 2023
Registry last updated
Apr 2, 2025

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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