QRX003 Lotion for Netherton Syndrome

Overseen By Oleg G Khatsenko

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Quoin Pharmaceuticals

Must be taking: Topical therapy

Must not be taking: Topical steroids

Disqualifiers: Pregnancy, Active cancer, Diabetes, 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: 4

Duration: 12 weeks

8 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 determine its effectiveness for people with Netherton syndrome, a genetic skin condition. Researchers aim to assess whether this lotion can reduce symptoms such as itchy and uncomfortable skin and identify any side effects. Participants will apply the lotion twice daily for three months and visit the clinic every 4-6 weeks. The trial seeks individuals diagnosed with Netherton syndrome who are on a stable treatment plan but still experience symptoms like itchy skin. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to potentially benefit from an innovative therapy.

Will I have to stop taking my current medications?

The trial requires that you stay on a stable treatment regimen, including any topical therapy for Netherton Syndrome, before and during the study. However, you must not have used certain treatments like topical steroids or ultraviolet phototherapy in the treatment area within 10 weeks before starting the trial.

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

Research has shown that QRX003 lotion appears safe for individuals with Netherton Syndrome, a genetic skin condition. In earlier studies, most participants tolerated QRX003 well when applied to the skin, with no reports of serious side effects. Some experienced mild skin irritation, a common occurrence with skin treatments, but it was not serious.

The lotion is currently undergoing further testing to confirm these results. As QRX003 is in the later stages of testing, substantial safety information is already available. This suggests the treatment is generally safe, but ongoing trials will offer more detailed safety insights. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

QRX003 Lotion is unique because it targets Netherton Syndrome by delivering a novel active ingredient directly to the skin. Unlike other treatments that might focus on systemic approaches, QRX003 uses a topical delivery method, potentially reducing side effects. Researchers are excited about QRX003 because it promises targeted relief by addressing the underlying skin barrier dysfunction, which could lead to better outcomes for patients suffering from this rare condition.

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

Research has shown that QRX003 lotion may help treat Netherton Syndrome. In earlier studies, patients using QRX003 experienced significant improvement in their condition. After 9 months, some patients had completely healed skin, demonstrating the treatment's lasting effects. Initial results also indicated a clear reduction in symptoms after just 12 weeks. These findings suggest QRX003 may effectively reduce skin problems associated with Netherton Syndrome. ^{3 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with Netherton Syndrome, a genetic skin condition. Participants will apply QRX003 lotion twice daily to affected areas (not the scalp) for three months and attend clinic visits every 4-6 weeks.

Inclusion Criteria

I am at least 14 years old and not pregnant.

I am a woman not able to have children, using birth control, or post-menopausal.

I am on a steady treatment plan for my skin condition that will not change during the study.

Exclusion Criteria

I have diabetes, but it's not the type that's controlled without insulin.

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

I don't have skin conditions in the treatment area that need other treatments.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants apply QRX003 lotion twice daily to affected areas for 3 months

12 weeks

Clinic visits every 4-6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• QRX003

Trial Overview The study tests if QRX003 lotion can improve symptoms of Netherton Syndrome such as diseased skin area, itchiness, and discomfort. It also evaluates safety and whether participants need additional rescue therapy.

How Is the Trial Designed?

Group I: 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

A case of Netherton syndrome (NS), a rare genetic disorder caused by mutations in the SPINK5 gene, was confirmed through whole exome sequencing, identifying two new mutations from the patient's parents.

The patient showed significant clinical improvement after treatment with dupilumab, a medication typically used for other conditions, with no reported adverse effects, suggesting potential for dupilumab as a safe therapeutic option for NS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Successful treatment of Netherton syndrome with dupilumab: A case report and review of the literature. Wang, J., Yu, L., Zhang, S., et al.[2022]

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]

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]

Citations

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

News Release As the table below illustrates all of the positive clinical benefits observed after 12 weeks of testing with QRX003 were completely reversed by 4 weeks after ...

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

Clinical Study of QRX003 Lotion in Subjects With ... 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 ( ...

3. dermatologytimes.com dermatologytimes.com/view/quoin-pharmaceuticals-announces-positive-interim-qrx003-data-for-netherton-syndrome

Quoin Pharmaceuticals' Announces Positive Interim ... QRX003 showed significant improvement in Netherton Syndrome severity, with positive interim results in an open-label study. The study ...

4. investors.quoinpharma.com investors.quoinpharma.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-recruitment-three-additional

News Release After 9 months of continued whole body application of QRX003, the subject's skin remains completely healed demonstrating the durability of ...

5. clinicaltrialsarena.com clinicaltrialsarena.com/news/quoin-dosing-half-patients-lotion/

Quoin completes dosing half patients in study of QRX003 ... Quoin Pharmaceuticals has completed dosing half of the patients in an open-label study of QRX003 topical lotion to treat Netherton Syndrome.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05521438

Safety, Tolerability and Efficacy of QRX003 Lotion in ... Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome. ClinicalTrials.gov ID NCT05521438. Sponsor Quoin Pharmaceuticals.

7. quoinpharma.com quoinpharma.com/product-candidates/qrx003/

QRX003 This leads to the increased risk of infections, warts, skin cancer and irritation by allergens and other environmental microorganisms.

8. quoinpharma.gcs-web.com quoinpharma.gcs-web.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-further-clinical-evidence-qrx003

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