Netherton SyndromePhase 2/3NCT06953466
Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
A Phase 2/3 study testing QRX003, 4% Lotion in people with Netherton Syndrome, run by Quoin Pharmaceuticals at 1 site in United States.
Trial summary
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 (including up to the entire body [except the scalp]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Netherton Syndrome
- What is being tested?
- QRX003, 4% Lotion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Oct 2025to 30 Dec 2026
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is a male or non-pregnant female at least 14 years of age.
- Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline.
- Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.
- Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.
- Subject is on a stable treatment regimen including topical therapy for NS prior to baseline that is expected to remain stable for the duration of the study
Who cannot join
- Subject is pregnant, lactating, or is planning to become pregnant during the study.
- Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.
- Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
- Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
- Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
- Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
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- Subject has used ultraviolet phototherapy within the Treatment Area within 10 weeks prior to Visit 2/Baseline.
- Subject has used topical prescription treatment in the Treatment Area within 10 weeks prior to Visit 2/Baseline.
- Subject has used any topical steroid prescription treatments in the Treatment Area within 10 weeks prior to Visit 2/Baseline.
- Subject is currently enrolled in an investigational drug, biologic, or device study.
- Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2/Baseline.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Northwestern Memorial Hospital
Chicago, Illinois, 60611
Oleg G Khatsenko, 858-571-1800 ext. 166, okhatsenko@therapeuticsinc.com
About this study
Official title: A Single Center, Open Label, Expanded Access Study of QRX003 Lotion in Subjects With Netherton Syndrome
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 (including up to the entire body [except the scalp]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. The main questions the trial aims to answer are:
- Does QRX003 impact the clinical presentation of NS in adults and minors by improving the clinical symptoms (diseased skin area, itch, and discomfort; based on clinical scoring, subject self-assessment, and other criteria)?
- What medical problems do participants have when taking QRX003?
- What percent of subjects will require rescue therapy?
Read more
Participants will:
Take drug QRX003 twice daily (applied topically to all affected areas of the body excluding the scalp) for 3 months, visit the clinic once every 4-6 weeks for checkups and tests, and to keep a dosing diary that records the times they applied the drug.
- Conditions
- Netherton Syndrome
- Being tested
- QRX003, 4% Lotion
- Medical subject headings
- Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QRX003, 4% LotionDrug
QRX003 Topical Lotion containing 4% active drug (serine protease inhibitor)
| Study group | Role | What they receive |
|---|---|---|
| QRX003, 4% BID | Experimental | Subjects will apply test article twice daily (BID) for 12 weeks Receives: Drug: QRX003, 4% Lotion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with 1-point reduction on IGA
Description: Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe.
Measured: Up to week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2025
- Study start6 Oct 2025
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Netherton SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Quoin Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06953466
- Sponsor's protocol ID
- CL-QRX003-003
- Other IDs
- Sponsor Funded
- Status last verified
- Aug 2026
- First submitted
- 7 Apr 2025
- Last updated
- 31 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Center, Open Label, Expanded Access Study of QRX003 Lotion in Subjects With Netherton Syndrome
- ClinicalTrials.gov ID
- NCT06953466
- Acronym / sponsor ID
- CL-QRX003-003
- Conditions
- Netherton Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Quoin PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 6 Oct 2025 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Dec 2026 (estimated)
- First posted
- 1 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 (including up to the entire body [except the scalp]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. The main questions the trial aims to answer are:
- Does QRX003 impact the clinical presentation of NS in adults and minors by improving the clinical symptoms (diseased skin area, itch, and discomfort; based on clinical scoring, subject self-assessment, and other criteria)?
- What medical problems do participants have when taking QRX003?
- What percent of subjects will require rescue therapy?
Participants will:
Take drug QRX003 twice daily (applied topically to all affected areas of the body excluding the scalp) for 3 months, visit the clinic once every 4-6 weeks for checkups and tests, and to keep a dosing diary that records the times they applied the drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label
| Arm | Type | Receives |
|---|---|---|
| QRX003, 4% BID | Experimental | Drug: QRX003, 4% LotionSubjects will apply test article twice daily (BID) for 12 weeks |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Subject is a male or non-pregnant female at least 14 years of age.
- Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline.
- Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.
- Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.
- Subject is on a stable treatment regimen including topical therapy for NS prior to baseline that is expected to remain stable for the duration of the study
Key exclusion criteria (11 listed)
- Subject is pregnant, lactating, or is planning to become pregnant during the study.
- Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.
- Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
- Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
- Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QRX003, 4% LotionQRX003 Topical Lotion containing 4% active drug (serine protease inhibitor) | Drug | QRX003, 4% BID |
Primary outcome
Proportion of subjects with 1-point reduction on IGA
Time frame: Up to week 16
Description: Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing QRX003, 4% Lotion in people with Netherton Syndrome, run by Quoin Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 31 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact TI Clinical Research (858-571-1800 ext. 147, clinicalresearch@therapeuticsinc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with 1-point reduction on IGA (time frame: Up to week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Aug 2026.