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

Trial Summary

What is the purpose of this trial?

To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects with Netherton syndrome

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.

How is QRX003 Lotion different from other treatments for Netherton Syndrome?

QRX003 Lotion is unique because it is specifically formulated as a topical treatment for Netherton Syndrome, which is a condition with no standard treatment. Unlike other treatments that may have systemic side effects due to increased absorption through the damaged skin barrier, QRX003 Lotion is designed to be applied directly to the skin, potentially minimizing such risks. ^{1 2 3 4 5}

Research Team

Tony Andrasfay

Principal Investigator

Therapeutics, Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

References

1. 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]

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

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

3. Australia pubmed.ncbi.nlm.nih.gov

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

4. India pubmed.ncbi.nlm.nih.gov

Netherton syndrome in one Chinese adult with a novel mutation in the SPINK5 gene and immunohistochemical studies of LEKTI. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Narrowband UVB phototherapy as a novel treatment for Netherton syndrome. [2012]