Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-07-31

Study Completion Date

2026-07-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The goal of this clinical trial (via an expanded access Treatment Protocol) is to learn if QRX003 (an investigational drug) applied topically to the skin (including up to the entire body \[except the scalp\]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. The main questions the trial aims to answer are:

1. Does QRX003 impact the clinical presentation of NS in adults and minors by improving the clinical symptoms (diseased skin area, itch, and discomfort; based on clinical scoring, subject self-assessment, and other criteria)?
2. What medical problems do participants have when taking QRX003?
3. What percent of subjects will require rescue therapy?

Participants will:

Take drug QRX003 twice daily (applied topically to all affected areas of the body excluding the scalp) for 3 months, visit the clinic once every 4-6 weeks for checkups and tests, and to keep a dosing diary that records the times they applied the drug.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT05789056

Netherton Syndrome

RECRUITING PHASE2/PHASE3

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

NCT05521438

Netherton Syndrome

ACTIVE_NOT_RECRUITING PHASE2/PHASE3

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

NCT03605069

Epidermolysis Bullosa Dystrophica, Recessive Epidermolysis Bullosa Dystrophica, Dominant

TERMINATED PHASE1/PHASE2

Natural History of Netherton Syndrome

NCT05902663

Netherton Syndrome

TERMINATED

Gene Therapy for Severe Crigler Najjar Syndrome

NCT03466463

Crigler-Najjar Syndrome

RECRUITING NA

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Netherton Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open-label

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

QRX003, 4% BID

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

Group Type EXPERIMENTAL

QRX003, 4% Lotion

Intervention Type DRUG

QRX003 Topical Lotion containing 4% active drug (serine protease inhibitor)

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

QRX003, 4% Lotion

QRX003 Topical Lotion containing 4% active drug (serine protease inhibitor)

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Subject is a male or non-pregnant female at least 14 years of age.
2. 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.
3. 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.
4. 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.
5. Subject is on a stable treatment regimen including topical therapy for NS prior to baseline that is expected to remain stable for the duration of the study

Exclusion Criteria

1. Subject is pregnant, lactating, or is planning to become pregnant during the study.
2. 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.
3. Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
4. Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
5. Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
6. Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
7. Subject has used ultraviolet phototherapy within the Treatment Area within 10 weeks prior to Visit 2/Baseline.
8. Subject has used topical prescription treatment in the Treatment Area within 10 weeks prior to Visit 2/Baseline.
9. Subject has used any topical steroid prescription treatments in the Treatment Area within 10 weeks prior to Visit 2/Baseline.
10. Subject is currently enrolled in an investigational drug, biologic, or device study.
11. 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