QRX003 Lotion for Netherton Syndrome

Recruiting at 4 trial locations

Overseen By Oleg G Khatsenko, Ph.D

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Quoin Pharmaceuticals

Must not be taking: Antibiotics, Retinoids, Steroids, others

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

16 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a lotion called QRX003 for individuals with Netherton syndrome, a rare genetic skin condition affecting many body areas. The study aims to determine if applying the lotion twice daily for 12 weeks is safe and effective. Individuals diagnosed with Netherton syndrome, experiencing its effects on at least half of their skin (excluding the scalp), may qualify for this trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to significant advancements in care.

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

Research has shown that QRX003 lotion is generally safe for people with Netherton Syndrome, a genetic skin condition. In earlier studies, most participants handled the treatment well, with few reports of major side effects, suggesting that the lotion is well-tolerated. The FDA granted QRX003 a Fast Track designation, indicating its promise in treating this condition. This designation reflects positive results in terms of safety and effectiveness in past trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Netherton syndrome?

Most treatments for Netherton Syndrome involve managing symptoms with emollients, topical steroids, or immunosuppressive agents. But QRX003 lotion stands out because it targets the root cause of the condition. Unlike standard treatments, QRX003 uses a unique formulation that helps restore the skin barrier function by directly addressing the genetic abnormalities associated with Netherton Syndrome. Researchers are excited about this approach because it has the potential to provide more effective and longer-lasting relief for patients, reducing the dependency on steroids and other symptom-based treatments.

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. Studies have found that it can greatly reduce the severity of the condition, with some patients achieving completely healed skin after nine months. Early trials noted quick skin healing and relief from itching. These positive early results confirm that regular use of the lotion can improve skin conditions. This trial will evaluate the effectiveness of QRX003 lotion, applied twice daily for 12 weeks, as a potential treatment option for people with Netherton Syndrome. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

Inclusion Criteria

I have a confirmed genetic diagnosis of Noonan syndrome.

I am male or female and I am at least 4 years old.

Participant has provided written informed consent/assent. Participants under legal age of consent must provide written informed assent and be accompanied by the parent or legal guardian at the time of assent/consent signing.

Exclusion Criteria

Participant is pregnant, lactating, or planning to become pregnant during the study.

I have another active cancer outside the treatment area, except for melanoma skin cancer.

I do not have serious diabetes, infections, or other major health issues that could exclude me.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 4% QRX003 lotion applied twice daily for 12 weeks to affected areas of the body

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

How Is the Trial Designed?

Group I: QRX003 lotion, 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+

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/NCT05789056

Open Label, Safety and Efficacy Study of QRX003 Lotion in ...

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

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.

clinicaltrialvanguard.com

clinicaltrialvanguard.com/news/quoin-netherton-syndrome-study-shows-positive-9-month-data/

Quoin Netherton Syndrome Study Shows Positive 9-Month ...

In an investigator-led pediatric study of QRX003 for Netherton syndrome, the first enrolled child achieved complete skin clearance by ...

5.

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

6.

dermatologytimes.com

dermatologytimes.com/view/fda-grants-fast-track-designation-to-qrx003-lotion-for-rare-netherton-syndrome

FDA Grants Fast Track Designation to QRX003 Lotion for ...

FDA Fast Track boosts hopes for QRX003 lotion in Netherton Syndrome, with early trials showing rapid skin healing and itch relief.

7.

thedermdigest.com

thedermdigest.com/u-s-fda-oks-quoins-second-netherton-syndrome-clinical-study/

U.S. FDA OKs Quoin's Second Netherton Syndrome ...

Clinical data from Quoin's ongoing studies has demonstrated clear evidence of rapid, prolonged and almost complete skin healing following twice-daily ...