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

NCT ID: NCT05789056

Last Updated: 2025-04-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-14

Study Completion Date

2025-12-30

Brief Summary

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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

Detailed Description

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Conditions

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Netherton Syndrome

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Open Label
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Open Label

Study Groups

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QRX003, 4% QAM

Subjects will apply test article once daily in the morning (QAM) for 12 weeks

Group Type EXPERIMENTAL

QRX003, 4% Lotion

Intervention Type DRUG

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

QRX003, 4% BID

Subjects will apply test article twice daily (BID) for 12 weeks

Group Type EXPERIMENTAL

QRX003, 4% Lotion

Intervention Type DRUG

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

Interventions

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QRX003, 4% Lotion

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

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

14 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Quoin Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Tony Andrasfay

Role: STUDY_DIRECTOR

Therapeutics, Inc.

Locations

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Site #1

San Diego, California, United States

Site Status RECRUITING

Site #4

Indianapolis, Indiana, United States

Site Status RECRUITING

Site #5

Quincy, Massachusetts, United States

Site Status RECRUITING

Site #2

San Antonio, Texas, United States

Site Status NOT_YET_RECRUITING

Countries

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United States

Central Contacts

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TI Clinical Research

Role: CONTACT

858-571-1800 ext. 147

Oleg G Khatsenko

Role: CONTACT

858-571-1800 ext. 166

Facility Contacts

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Oleg G Khatsenko

Role: primary

858-571-1800 ext. 166

Oleg G Khatsenko

Role: primary

858-571-1800 ext. 166

Oleg G Khatsenko

Role: primary

858-571-1800 ext. 166

Oleg G Khatsenko

Role: primary

858-571-1800 ext. 166

Other Identifiers

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CL-QRX003-002

Identifier Type: -

Identifier Source: org_study_id

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