QRX003 Lotion for Netherton Syndrome

Recruiting at 4 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Recruiting

Sponsor: Quoin Pharmaceuticals

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications to join the trial?

The trial requires you to stop certain medications. You must not use systemic prescription treatments for Netherton Syndrome within 4 weeks before starting the trial, systemic biologic therapy for NS, topical prescription treatments in the treatment area within 2 weeks before starting, or any topical moisturizers/emollients in the treatment area within 24 hours before starting.

What data supports the idea that QRX003 Lotion for Netherton Syndrome is an effective treatment?

The available research does not provide specific data on the effectiveness of QRX003 Lotion for Netherton Syndrome. However, it mentions other treatments like secukinumab and acitretin that have shown improvement in patients with this condition. Secukinumab led to marked skin improvement in two cases, while acitretin, combined with a moisturizer, resulted in significant skin and hair improvement in a child over several months. This suggests that while QRX003 Lotion's effectiveness isn't directly supported by the available data, other treatments have shown positive outcomes for Netherton Syndrome. ^{1 2 3 4 5}

What safety data exists for QRX003 Lotion for Netherton Syndrome?

The provided research does not contain any specific safety data for QRX003 Lotion or its variants. The articles focus on allergic reactions to natural rubber, contact dermatitis, and latex allergies, which are unrelated to QRX003 Lotion. Therefore, no relevant safety data for QRX003 Lotion is available in the provided research. ^{6 7 8 9 10}

Is QRX003 Lotion a promising drug for Netherton Syndrome?

QRX003 Lotion is a promising drug for Netherton Syndrome because it is being tested specifically for this rare skin condition, which is caused by a genetic mutation leading to severe skin problems. The focus on this specific syndrome suggests that QRX003 Lotion could offer targeted relief and improvement for patients with Netherton Syndrome. ^{2 11 12 13 14}

Research Team

Tony Andrasfay

Principal Investigator

Therapeutics, Inc.

Eligibility Criteria

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.

Inclusion Criteria

I have Netherton Syndrome and agree to genetic testing if I don't have SPINK5 mutation results.

I am not able to become pregnant, or I will use birth control during and 3 months after the study.

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

Exclusion Criteria

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

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

I have used prescription skin treatment recently.

Treatment Details

Interventions

• QRX003-2% Lotion (Topical Agent)
• QRX003-4% Lotion (Topical Agent)
• Vehicle (Vehicle)

Trial Overview 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.

Participant Groups

Group I: Vehicle Lotion Experimental Treatment 1 Intervention

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

Group II: QRX003-4% QAM Experimental Treatment 1 Intervention

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

Group III: QRX003-4% BID Experimental Treatment 1 Intervention

Subjects will apply test article twice daily BID for 12 weeks

Group IV: QRX003-2% QAM Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Quoin Pharmaceuticals

Lead Sponsor

Trials

2

Recruited

50+

Findings from Research

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]

A pediatric case of a Buschke-Lowenstein tumor in a patient with Netherton syndrome was successfully treated with surgery, demonstrating that surgical intervention can be safe in these patients despite concerns about wound healing complications.

The use of conventional dressings and topical negative-pressure therapy allowed for effective wound management and healing, suggesting that these methods can be beneficial in patients with Netherton syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Considerations in surgical management of a Buschke-Lowenstein tumor in Netherton syndrome: A case report. Ashton, R., Moledina, J., Sivakumar, B., et al.[2018]

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]

An 8-year-old boy with Netherton syndrome was initially misdiagnosed with severe atopic dermatitis, highlighting the importance of accurate and timely diagnosis for effective treatment.

Treatment with a low dose of oral acitretin (0.25 mg/kg) and a topical moisturizer led to significant improvement in the child's skin condition and pruritus within 2 months, with continued improvement observed at the 6-month follow-up.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An 8-Year-Old Child with Delayed Diagnosis of Netherton Syndrome. Leung, AKC., Barankin, B., Leong, KF.[2020]

There has been a significant increase in severe allergic reactions, including anaphylaxis, related to natural rubber exposure, particularly affecting patients with spina bifida and healthcare workers, with an estimated 2% to 10% of medical personnel being latex allergic.

Latex allergy can cause a range of reactions from contact dermatitis to systemic anaphylaxis, and while diagnosis often relies on clinical history, skin prick tests and serologic testing are available but have limitations in specificity and sensitivity. Prophylactic measures to reduce rubber exposure are recommended to prevent sensitization.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Allergic reaction to products made of natural rubber]. Antczak, M., Kuna, P., Cieślewicz, G.[2008]

A retrospective study of 618 patients with suspected allergic contact dermatitis revealed that the most common allergens were nickel sulfate, thimerosal, and potassium bichromate.

The study found a statistically significant decrease in positive patch-test results for lanolin, neomycin, and anthraquinone over the years, suggesting these allergens may have a lower risk of sensitization and could potentially be excluded from standard testing in the future.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patch test standard series recommended by the Brazilian Contact Dermatitis Study Group during the 2006-2011 period. Duarte, IA., Tanaka, GM., Suzuki, NM., et al.[2021]

A high incidence of positive patch test reactions to E45 cream was observed, with 118 out of 362 patients testing positive, indicating potential allergic reactions to its ingredients.

The main allergen identified was triethanolamine, while irritant reactions were linked to the formation of triethanolamine stearate in the cream, leading to a formulation change that will remove triethanolamine to enhance safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Contact dermatitis from the old formula E45 cream. Batten, TL., Wakeel, RA., Douglas, WS., et al.[2019]

Latex allergy is becoming more common due to the widespread use of latex products, with three main types of reactions: irritant contact dermatitis, allergic contact dermatitis, and Type I hypersensitivity, particularly affecting at-risk groups like children with spina bifida.

Diagnosis of latex allergy involves history, skin prick tests, and specific serum IgE tests, but avoidance of latex is the best prevention strategy, which is challenging due to the prevalence of latex products in everyday life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A discussion of natural rubber latex allergy with special reference to children: clinical considerations. Eustachio, N., Cristina, CM., Antonio, F., et al.[2019]

A study analyzing 16 brands of gloves marketed as 'accelerator-free' found that all gloves contained at least one rubber accelerator, contradicting their claims.

Specifically, dipentamethylenethiuram disulfide was detected in all gloves, and half of the samples contained multiple accelerators, highlighting a potential risk for patients with allergic contact dermatitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Are Rubber Gloves Marketed as Accelerator-Free Truly Free of Accelerators? Pillsbury, ME., Ronkainen, S., Goodier, M., et al.[2021]

Netherton syndrome (NS) is a severe genetic disorder linked to chromosome 5q32, identified through linkage analysis in 20 affected families, indicating a strong genetic basis for the condition.

This research marks a significant step towards identifying the NS gene, which could enhance our understanding of skin and immune system development, potentially leading to better treatments for affected individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Localization of the Netherton syndrome gene to chromosome 5q32, by linkage analysis and homozygosity mapping. Chavanas, S., Garner, C., Bodemer, C., et al.[2021]

Netherton's syndrome is a rare congenital condition in children that can present with various skin issues, such as ichthyosis linearis circumflexa and atopic symptoms, as seen in a case of a five-year-old boy with additional neurological complications.

Due to its nonspecific symptoms, Netherton's syndrome can be easily misdiagnosed as other skin disorders, highlighting the importance for pediatricians to include it in their differential diagnosis for generalized erythematous skin conditions in children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A case of Netherton's syndrome with cerebral infarction. Calikoğlu, E., Anadolu, R., Sanli, H., et al.[2004]

Netherton syndrome is a rare genetic disorder characterized by specific symptoms including ichthyosis linearis circumflexa, atopic conditions, and unique hair shaft anomalies.

The diagnosis of Netherton syndrome in the presented patient was confirmed through the identification of 'matchstick' and 'golf tee' hair signs, alongside the classic hair shaft anomaly known as trichorrhexis invaginata.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rapid and easy diagnosis of Netherton syndrome with dermoscopy. Meltem Akkurt, Z., Tuncel, T., Ayhan, E., et al.[2017]

References

1. United States pubmed.ncbi.nlm.nih.gov

Cushing syndrome from percutaneous absorption of 1% hydrocortisone ointment in Netherton syndrome. [2017]

2. England pubmed.ncbi.nlm.nih.gov

Establishment of an induced pluripotent stem cell line (SAHGMUi001-A) from a patient with Netherton syndrome carrying SPINK5 mutation. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Considerations in surgical management of a Buschke-Lowenstein tumor in Netherton syndrome: A case report. [2018]

4. Australia pubmed.ncbi.nlm.nih.gov

Secukinumab use in the treatment of Netherton's syndrome. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

An 8-Year-Old Child with Delayed Diagnosis of Netherton Syndrome. [2020]

6. Poland pubmed.ncbi.nlm.nih.gov

[Allergic reaction to products made of natural rubber]. [2008]

7. Spain pubmed.ncbi.nlm.nih.gov

Patch test standard series recommended by the Brazilian Contact Dermatitis Study Group during the 2006-2011 period. [2021]

8. England pubmed.ncbi.nlm.nih.gov

Contact dermatitis from the old formula E45 cream. [2019]

9. United Arab Emirates pubmed.ncbi.nlm.nih.gov

A discussion of natural rubber latex allergy with special reference to children: clinical considerations. [2019]

10. United States pubmed.ncbi.nlm.nih.gov

Are Rubber Gloves Marketed as Accelerator-Free Truly Free of Accelerators? [2021]

11. United States pubmed.ncbi.nlm.nih.gov

Localization of the Netherton syndrome gene to chromosome 5q32, by linkage analysis and homozygosity mapping. [2021]

12. Portugal pubmed.ncbi.nlm.nih.gov

[Netherton syndrome]. [2013]

13. Turkey pubmed.ncbi.nlm.nih.gov

A case of Netherton's syndrome with cerebral infarction. [2004]

14. United States pubmed.ncbi.nlm.nih.gov

Rapid and easy diagnosis of Netherton syndrome with dermoscopy. [2017]