Netherton SyndromePhase 2/3NCT07538583
Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
A Phase 2/3 study testing QRX003 in people with Netherton Syndrome, run by Quoin Pharmaceuticals at 8 sites in 2 countries.
Trial summary
This is a multicenter, open-label study to evaluate the safety, tolerability, and efficacy of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA)… Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Netherton Syndrome
- What is being tested?
- QRX003
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 19 Mar 2026to 31 Mar 2027
- 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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participant is male or female and at least 4 years of age.
- Participant has provided written informed consent/assent. Based on the participant's age and local regulations , a participant 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. The parent or legal guardian must provide informed consent for the participant. If a participant reaches the age at which consent is required by local regulations during the study, the participant must provide written informed consent at that time to continue study participation.
- Female participants must either not have experienced menarche, be postmenopausal , or be surgically sterile , or use a highly effective method of birth control , for the duration of the study and for 3 months following EOT. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline. Males must use a condom in addition to having their female partner use a highly effective method of birth control.
- Participant has a genetically confirmed diagnosis of NS prior to Visit 2/Baseline. Participants without existing results must undergo genetic testing at Visit 1/Screening, which must confirm the diagnosis of NS in order for the participants to be enrolled in the study.
Who cannot join
- Participant is pregnant, lactating, or is planning to become pregnant during the study.
- Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.
- Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.
- Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2/Baseline.
- Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
- Participant has ongoing or recent history of any other uncontrolled and/or clinically significant systemic disease or condition which, in the opinion of the investigator, should exclude participation in the study.
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- Participant has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
- Participant has used systemic non-biologics prescription treatment for NS or that in the opinion of the investigator may affect the participant's NS (examples include, but are not limited to antibiotics, retinoids, etc.) within 4 weeks prior to Visit 2/Baseline.
- Participant has used systemic biologic therapy for NS or that in the opinion of the investigator may affect the participant's NS within 5 half-lives of the biologic therapy prior to Visit 2/Baseline. Published or documented half-life of the product provided by the commercial supplier or Sponsor should be used to establish this value.
- Participant has used topical prescription treatments, including steroids (examples include, but are not limited to keratolytics, steroids, retinoids, etc.) in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
- Participant has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
- Participant is currently enrolled in an investigational drug, biologic, or device study.
Trial locations and contacts
8 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
8 sites in 2 countries
United States5 sites, 5 recruiting
- Recruiting
Center for Dermatology Clinical Research
Fremont, California, 94538
Lisa Duarte, 858-571-1800 ext. 185, lduarte@therapeuticsinc.com
- Recruiting
Dawes Fretzin Clinical Reseacrh Group
Indianapolis, Indiana, 46250
Lisa Duarte, 858-571-1800 ext. 185, lduarte@therapeuticsinc.com
- Not yet recruiting
Yale University School of Medicine
New Haven, Connecticut, 06520
Lisa Duarte, 858-571-1800 ext. 185, lduarte@therapeuticsinc.com
- Recruiting
Beacon Clinical Research
Quincy, Massachusetts, 02169
Lisa Duarte, 858-571-1800 ext. 185, lduarte@therapeuticsinc.com
- Recruiting
Texas Dermatology and Laser Specialists
San Antonio, Texas, 78218
Lisa Duarte, 8585711800 ext. 185, lduarte@therapeuticsinc.com
United Kingdom3 sites, 3 recruiting
- Recruiting
Great Ormond Street Hospital, NIHR - Clinical Research
London, England, WC1N 3JH
Nigel Jones, +44 (0)7867 362044, njones@quoinpharma.com
- Recruiting
Guy's Hospital, Skin Therapy Research Unit
London, SE1 9RT
Nigel Jones, +44 (0)7867 362044, njones@quoinpharma.com
- Recruiting
Walsall Manor Hospital, Research & Development
Walsall, West Midlands, WS2 9PS
Nigel Jones, +44 (0)7867 362044, njones@quoinpharma.com
About this study
Official title: A Multicenter, Open-label Study of QRX003 Lotion in Participants With Netherton Syndrome
This is a multicenter, open-label study to evaluate the safety, tolerability, and efficacy of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA) or more.
- Conditions
- Netherton Syndrome
- Being tested
- QRX003
- Medical subject headings
- Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QRX003Drug
Serine Protease Inhibitor
| Study group | Role | What they receive |
|---|---|---|
| QRX003 lotion, 4% BID | Experimental | Subjects will apply test article twice daily (BID) for 12 weeks Receives: Drug: QRX003 |
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
The IGA (Investigator Global Assessment) is a 5-point scale used to assess the overall severity of a Subject's NS condition.
Measured: Week 12
Secondary outcomes (3)
Proportion of subjects with 2-point improvement on the WI-NRS
Subjects will rate their itch intensity using the 11-point Worst-Itch-Numeric Rating Scale (WI-NRS)
Measured: Week 12
PGI-C Score
Patient (or Caregiver) Global Impression of Change (PGI-C) is a 7-point, self-assessed scale measuring the patient's (or caregiver's) improvement of their NS condition.
Measured: Week 12
CGI-C Score
Clinical Global Impression of Change (PGI-C) is a 7-point scale used by the clinician to assess improvement of the NS condition.
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2026
- Study start19 Mar 2026
- Primary completion31 Jan 2027 (estimated)
- Study completion31 Mar 2027 (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
- Masking
- None (open label)
- Enrolment
- 16 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
- NCT07538583
- Sponsor's protocol ID
- CL-QRX003-004
- Status last verified
- Apr 2026
- First submitted
- 24 Mar 2026
- Last updated
- 28 Sep 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 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label Study of QRX003 Lotion in Participants With Netherton Syndrome
- ClinicalTrials.gov ID
- NCT07538583
- Acronym / sponsor ID
- CL-QRX003-004
- Conditions
- Netherton Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Quoin PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 19 Mar 2026 (actual)
- Primary completion
- 31 Jan 2027 (estimated)
- Study completion
- 31 Mar 2027 (estimated)
- First posted
- 20 Apr 2026 (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.
This is a multicenter, open-label study to evaluate the safety, tolerability, and efficacy of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA) or more.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| QRX003 lotion, 4% BID | Experimental | Drug: QRX003Subjects will apply test article twice daily (BID) for 12 weeks |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Participant is male or female and at least 4 years of age.
- Participant has provided written informed consent/assent. Based on the participant's age and local regulations , a participant 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. The parent or legal guardian must provide informed consent for the participant. If a participant reaches the age at which consent is required by local regulations during the study, the participant must provide written informed consent at that time to continue study participation.
- Female participants must either not have experienced menarche, be postmenopausal , or be surgically sterile , or use a highly effective method of birth control , for the duration of the study and for 3 months following EOT. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline. Males must use a condom in addition to having their female partner use a highly effective method of birth control.
- Participant has a genetically confirmed diagnosis of NS prior to Visit 2/Baseline. Participants without existing results must undergo genetic testing at Visit 1/Screening, which must confirm the diagnosis of NS in order for the participants to be enrolled in the study.
Key exclusion criteria (12 listed)
- Participant is pregnant, lactating, or is planning to become pregnant during the study.
- Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.
- Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.
- Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2/Baseline.
- Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
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 |
|---|---|---|
| QRX003Serine Protease Inhibitor | Drug | QRX003 lotion, 4% BID |
Primary outcome
Proportion of subjects with 1-point reduction on IGA
Time frame: Week 12
The IGA (Investigator Global Assessment) is a 5-point scale used to assess the overall severity of a Subject's NS condition.
3 secondary outcomes
Proportion of subjects with 2-point improvement on the WI-NRS
Time frame: Week 12
PGI-C Score
Time frame: Week 12
CGI-C Score
Time frame: Week 12
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 28 Sep 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.
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing QRX003 in people with Netherton Syndrome, run by Quoin Pharmaceuticals at 8 sites in 2 countries.
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 4 inclusion and 12 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 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, United Kingdom. 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: Week 12). 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 28 Sep 2026.