What side effects are associated with this type of intervention?

Common treatments for Netherton Syndrome include topical corticosteroids, emollients, and keratolytic agents. Topical corticosteroids can lead to skin thinning, stretch marks, and increased infection risk with long-term use. Emollients are generally safe but may cause mild irritation or allergic reactions. Keratolytic agents can result in skin irritation, redness, and peeling. QRX003 lotion, being studied for its safety and efficacy, is likely evaluated for similar side effects, focusing on its tolerability in managing the condition.

What prior FDA approvals exist?

There is limited information on prior FDA approvals specifically for the treatment of Netherton Syndrome. The condition is rare, and treatments are often focused on managing symptoms rather than targeting the underlying cause. QRX003 lotion, currently under study, aims to address Netherton Syndrome by targeting its specific pathophysiology. Other treatments for similar skin conditions often include topical corticosteroids, emollients, and immunomodulatory agents, but these are not specifically approved for Netherton Syndrome.

What other types of research exist?

Promising novel alternatives to QRX003 lotion for Netherton Syndrome patients in 2024 include biologic therapies such as dupilumab, which targets the IL-4 receptor and has shown efficacy in treating severe atopic dermatitis, a condition with overlapping symptoms. Additionally, emerging topical treatments like ruxolitinib cream, a JAK inhibitor, may offer benefits due to their anti-inflammatory properties. These alternatives are based on recent advancements and ongoing research in dermatological treatments.

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Topical Agent

QRX003 Lotion for Netherton Syndrome

Phase 2 & 3

Recruiting

Research Sponsored by Quoin Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject has NS lesions in the Treatment Area (i.e., arms or lower legs).

Subject is a male or non-pregnant female at least 18 years of age.

Must not have

Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to week 16

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medicated cream called QRX003 lotion on patients with Netherton Syndrome, a rare skin disorder. The study will check if different doses and application schedules of the lotion are safe and effective. The goal is to see if the lotion can improve skin symptoms like redness and irritation.

Who is the study for?

Adults with Netherton Syndrome (NS) who are in good health and have NS lesions on their arms or lower legs can join this trial. Women must not be pregnant, should use birth control, and have a negative pregnancy test. Participants shouldn't have used certain treatments for NS or any investigational drugs recently.

What is being tested?

The study is testing QRX003 lotion at two different strengths (2% and 4%) against a placebo lotion to see if they're safe, tolerable, and effective for treating Netherton Syndrome.

What are the potential side effects?

While the side effects of QRX003 lotions aren't detailed here, typical topical treatment risks include skin irritation, redness, itching or allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have skin lesions on my arms or lower legs.

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I am at least 18 years old and not pregnant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have active cancer, not including non-melanoma skin cancer, outside the treatment area.

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I have a skin condition in the treatment area that could affect the study's results.

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I have used prescription skin treatment recently.

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I have HIV, hepatitis B or C, or tuberculosis.

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I have used systemic biologic therapy for my condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to week 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to week 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 16 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Efficacy Endpoints-1-point IGA

Efficacy Endpoints-2-point IGA

Efficacy Endpoints-BSA

+6 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Vehicle LotionExperimental Treatment1 Intervention

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

Group II: QRX003-4% QAMExperimental Treatment1 Intervention

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

Group III: QRX003-4% BIDExperimental Treatment1 Intervention

Subjects will apply test article twice daily BID for 12 weeks

Group IV: QRX003-2% QAMExperimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vehicle

2013

Completed Phase 3

~5070

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Netherton Syndrome is a genetic disorder that affects the skin barrier, leading to chronic skin inflammation and increased susceptibility to infections. Treatments like QRX003 lotion, which is being studied for its efficacy and safety, typically contain active ingredients that aim to restore the skin barrier and reduce inflammation. These treatments often include keratolytic agents, which help to remove the thickened outer layer of the skin, and anti-inflammatory agents that reduce the chronic inflammation characteristic of the condition. Restoring the skin barrier is crucial for Netherton Syndrome patients as it helps to prevent infections, reduce inflammation, and improve overall skin health, thereby enhancing the quality of life.

Systemic glucocorticosteroid therapy of skin disease in children.A systematic review of clinical trials of treatments for the congenital ichthyoses, excluding ichthyosis vulgaris.