QRX003 Lotion for Netherton Syndrome

Recruiting at 3 trial locations

Overseen By Oleg G Khatsenko

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Quoin Pharmaceuticals

Must be taking: Systemic therapy

Disqualifiers: Pregnancy, Cancer, Diabetes, Infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new lotion, QRX003, to evaluate its safety and effectiveness for individuals with Netherton syndrome, a rare genetic skin condition causing redness, scaling, and itching. Participants will apply the lotion once or twice daily for 12 weeks, alongside their usual treatment. The trial seeks individuals diagnosed with Netherton syndrome who have skin lesions on their arms or legs and are on a stable treatment plan. As a Phase 2/3 trial, it measures the treatment's effectiveness in a larger group and represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify that you need to stop your current medications. In fact, it requires that you stay on a stable treatment regimen, including systemic therapy, for Netherton syndrome throughout the study.

Is there any evidence suggesting that QRX003 lotion is likely to be safe for humans?

Research has shown that QRX003 lotion may help treat skin conditions. In one study involving a child with Peeling Skin Syndrome, the lotion was used for 12 weeks, resulting in significant skin improvement without any reported side effects.

While specific safety information for QRX003 lotion in Netherton Syndrome is not yet available, it is currently being tested in a combined phase 2 and 3 trial. Some safety data from this trial suggest the lotion is likely safe to use. However, further research is needed to confirm its safety for all individuals. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for Netherton syndrome?

Unlike the standard of care for Netherton Syndrome, which often involves topical steroids and emollients, QRX003 Lotion is unique because it is designed specifically to address the underlying cause of the condition. QRX003 uses a new active ingredient that targets the skin barrier dysfunction, which is central to Netherton Syndrome. Researchers are excited about QRX003 because it offers a targeted approach that could potentially improve symptoms more effectively than current treatments, which mainly focus on symptom management rather than addressing the root cause. Additionally, the option of applying the lotion either twice daily or once in the morning provides flexibility for patients, which could enhance adherence and overall treatment experience.

What evidence suggests that QRX003 lotion might be an effective treatment for Netherton syndrome?

Research has shown that QRX003 lotion may help treat Netherton syndrome. One study reported that a child with the condition experienced significant skin improvement and no adverse side effects after using the lotion twice daily for just two weeks. Another report found that after six weeks of daily use, the child's skin was almost completely healed, demonstrating the lotion's lasting benefits. In this trial, participants will join different treatment arms, with some applying QRX003 lotion twice daily and others once daily. These findings suggest that regular use of QRX003 lotion could lead to positive results for people with Netherton syndrome. ^{1 2 4 6 7}

Who Is on the Research Team?

Tony Andrasfay

Principal Investigator

Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with Netherton Syndrome (NS) who are in good health and on a stable NS treatment regimen. Participants must not be pregnant, have active infections, or other skin conditions that could affect the study. Women must use birth control during the study and agree to genetic testing if they haven't confirmed an NS diagnosis.

Inclusion Criteria

I am at least 18 years old and not pregnant.

I am in good health with no conditions that would risk my safety in the study.

I am on a steady treatment plan for my condition that won't change during the study.

Exclusion Criteria

I have used a basic moisturizer on the treatment area within the last 24 hours.

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

I have not had a serious infection in the last 30 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants apply QRX003 lotion either twice daily or once daily for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• QRX003, 4% Lotion

Trial Overview The trial tests QRX003 lotion (4%) combined with standard care for safety, tolerability, and effectiveness in treating NS. It's open-label, meaning everyone knows they're getting the lotion along with their usual systemic therapy.

How Is the Trial Designed?

Group I: QRX003, 4% QAM Experimental Treatment 1 Intervention

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

Group II: QRX003, 4% BID Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Quoin Pharmaceuticals

Lead Sponsor

Trials

2

Recruited

50+

Published Research Related to This Trial

Two cases of Netherton syndrome, a rare genetic disorder affecting skin, showed significant improvement when treated with secukinumab, a medication that targets IL-17.

This suggests that IL-17 therapy could be a promising treatment option for managing the symptoms of Netherton syndrome, which currently lacks specific treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Secukinumab use in the treatment of Netherton's syndrome. Gan, C., King, E., Orchard, D.[2022]

A new human induced pluripotent stem cell (iPSC) line has been created from urine-derived cells of a patient with Netherton syndrome, which is caused by mutations in the SPINK5 gene.

This iPSC line can be used as a valuable research model to study the disease's pathogenesis and explore potential therapies for the severe skin barrier damage associated with Netherton syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Establishment of an induced pluripotent stem cell line (SAHGMUi001-A) from a patient with Netherton syndrome carrying SPINK5 mutation. Xu, M., Wang, L., Yin, J., et al.[2021]

An 11-year-old boy with Netherton syndrome developed Cushing syndrome after using 1% hydrocortisone ointment over his entire body for more than a year, highlighting the risks of systemic absorption in patients with compromised skin barrier function.

This case emphasizes the need for caution when using even low-potency topical steroids in Netherton syndrome, as long-term use in large quantities can lead to significant systemic side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cushing syndrome from percutaneous absorption of 1% hydrocortisone ointment in Netherton syndrome. Halverstam, CP., Vachharajani, A., Mallory, SB.[2017]

Citations

1. investors.quoinpharma.com investors.quoinpharma.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-further-clinical-evidence-qrx003

News Release These results strongly indicate that ongoing, chronic treatment with QRX003 is necessary for a continued positive clinical outcome in Netherton Syndrome ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06953466

Clinical Study of QRX003 Lotion in Subjects With ... A research study involving human volunteers (also called participants) that is intended to add to medical knowledge. There are two types of clinical studies: ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05521438

Safety, Tolerability and Efficacy of QRX003 Lotion in ... A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may ...

4. dermatologytimes.com dermatologytimes.com/view/quoin-reports-positive-qrx003-whole-body-pediatric-data-for-netherton-syndrome

Quoin Reports Positive QRX003 “Whole Body” Pediatric ... A pediatric patient using QRX003 saw an improved disease state with visually healed skin and no adverse effects after just 2 weeks of twice-daily application.

5. investors.quoinpharma.com investors.quoinpharma.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-additional-positive-whole-body

News Release After 6 weeks of continued whole body application of QRX003, the subject's skin remains almost completely healed demonstrating the durability of ongoing daily ...

6. withpower.com withpower.com/trial/phase-2-and-3-netherton-syndrome-5-2025-416c0

QRX003 Lotion for Netherton Syndrome There is no specific safety data available for QRX003 Lotion, but other treatments for Netherton Syndrome, like biological therapies and dupilumab, have shown ...

7. dermatologytimes.com dermatologytimes.com/view/quoin-announces-positive-qrx003-data-for-pediatric-peeling-skin-syndrome

Quoin Announces Positive QRX003 Data for Pediatric ... QRX003 showed significant skin healing in a pediatric patient with Peeling Skin Syndrome after 12 weeks, with no adverse events reported.