Netherton SyndromePhase 2/3NCT05521438
Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome
A Phase 2/3 study testing QRX003-2% Lotion, QRX003-4% Lotion QAM and QRX003-4% Lotion BID in people with Netherton Syndrome, run by Quoin Pharmaceuticals at 5 sites in United States.
Trial summary
This study has been designed to determine the safety, tolerability and efficacy of QRX003 lotion 2%, 4% QAM or 4% BID in subjects with Netherton Syndrome (NS) in comparison to vehicle 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?
- QRX003-2% Lotion, QRX003-4% Lotion QAM and QRX003-4% Lotion BID
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 5 sitesUnited States
- Study dates
- 23 Jun 2022to 31 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 has NS lesions in the Treatment Area (i.e., arms or lower legs).
- 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.
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 Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females
- 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.
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- Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
- Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
- Subject has used systemic prescription treatment for NS within 4 weeks prior to Visit 2/Baseline.
- Subject has used systemic biologic therapy for NS.
- Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
- Subject has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours 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
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Site #3
Charleston, South Carolina, 29425
Site #4
Indianapolis, Indiana, 46250
Site #5
Quincy, Massachusetts, 02169
Site #2
San Antonio, Texas, 72218
Site #1
San Diego, California, 92123
About this study
Official title: A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome
This study has been designed to determine the safety, tolerability and efficacy of QRX003 lotion 2%, 4% QAM or 4% BID in subjects with Netherton Syndrome (NS) in comparison to vehicle
- Conditions
- Netherton Syndrome
- Being tested
- QRX003-2% Lotion, QRX003-4% Lotion QAM, Vehicle, QRX003-4% Lotion BID
- Medical subject headings
- Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QRX003-2% LotionDrug
QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)
QRX003-4% Lotion QAMDrug
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
VehicleDrug
Vehicle Lotion
QRX003-4% Lotion BIDDrug
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
| Study group | Role | What they receive |
|---|---|---|
| QRX003-2% QAM | Experimental | Subjects will apply test article once daily in the morning (QAM) for 12 weeks Receives: Drug: QRX003-2% Lotion |
| QRX003-4% QAM | Experimental | Subjects will apply test article once daily in the morning (QAM) for 12 weeks Receives: Drug: QRX003-4% Lotion QAM |
| Vehicle Lotion | Experimental | Subjects will apply test article once daily in the morning (QAM) for 12 weeks Receives: Drug: Vehicle |
| QRX003-4% BID | Experimental | Subjects will apply test article twice daily BID for 12 weeks Receives: Drug: QRX003-4% Lotion BID |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy Endpoints-1-point IGA
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
Efficacy Endpoints-2-point IGA
Proportion of subjects with 2-point reduction in IGA from Baseline.
Measured: Up to week 16
Efficacy Endpoints-NS Sign
Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.
Measured: Up to week 16
Efficacy Endpoints-BSA
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Measured: Up to week 16
Efficacy Endpoints-WI-NRS score
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".
Measured: Up to week 16
Efficacy Endpoints-TSQM
Assessment of subject satisfaction with treatment based on the Treatment Satisfaction Questionnaire for Medication (TSQM). The TSQM measures a subject's level of satisfaction or dissatisfaction with the treatment. Composite scores can range from 0 to 100; higher scores indicate better satisfaction.
Measured: Up to week 16
Efficacy Endpoints-Rescue Therapy
Proportion of subjects requiring rescue therapy
Measured: Up to week 16
Safety Endpoints-AEs
Any local and systemic AEs (Adverse Events)/serious AEs
Measured: Up to week 16
Safety Endpoints-LSR
Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.
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 posted30 Aug 2022
- Study start23 Jun 2022
- Primary completion30 Dec 2025
- Study completion31 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Quoin Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Tony Andrasfay, Study director, Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05521438
- Sponsor's protocol ID
- CL-QRX003-001
- Status last verified
- Mar 2026
- First submitted
- 17 Aug 2022
- Last updated
- 16 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome
- ClinicalTrials.gov ID
- NCT05521438
- Acronym / sponsor ID
- CL-QRX003-001
- Conditions
- Netherton Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Quoin PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 23 Jun 2022 (actual)
- Primary completion
- 30 Dec 2025 (actual)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 30 Aug 2022 (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 study has been designed to determine the safety, tolerability and efficacy of QRX003 lotion 2%, 4% QAM or 4% BID in subjects with Netherton Syndrome (NS) in comparison to vehicle
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| QRX003-2% QAM | Experimental | Drug: QRX003-2% LotionSubjects will apply test article once daily in the morning (QAM) for 12 weeks |
| QRX003-4% QAM | Experimental | Drug: QRX003-4% Lotion QAMSubjects will apply test article once daily in the morning (QAM) for 12 weeks |
| Vehicle Lotion | Experimental | Drug: VehicleSubjects will apply test article once daily in the morning (QAM) for 12 weeks |
| QRX003-4% BID | Experimental | Drug: QRX003-4% Lotion BIDSubjects 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 has NS lesions in the Treatment Area (i.e., arms or lower legs).
- 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.
Key exclusion criteria (14 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 Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females
- 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.
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 |
|---|---|---|
| QRX003-2% LotionQRX003Topical Lotion containing 2% active drug (serine protease inhibitor) | Drug | QRX003-2% QAM |
| QRX003-4% Lotion QAMQRX003Topical Lotion containing 4% active drug (serine protease inhibitor) | Drug | QRX003-4% QAM |
| VehicleVehicle Lotion | Drug | Vehicle Lotion |
| QRX003-4% Lotion BIDQRX003Topical Lotion containing 4% active drug (serine protease inhibitor) | Drug | QRX003-4% BID |
Primary outcome
Efficacy Endpoints-1-point IGA
Time frame: Up to week 16
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.
Efficacy Endpoints-2-point IGA
Time frame: Up to week 16
Proportion of subjects with 2-point reduction in IGA from Baseline.
Efficacy Endpoints-NS Sign
Time frame: Up to week 16
Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.
Efficacy Endpoints-BSA
Time frame: Up to week 16
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Efficacy Endpoints-WI-NRS score
Time frame: Up to week 16
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".
Efficacy Endpoints-TSQM
Time frame: Up to week 16
Assessment of subject satisfaction with treatment based on the Treatment Satisfaction Questionnaire for Medication (TSQM). The TSQM measures a subject's level of satisfaction or dissatisfaction with the treatment. Composite scores can range from 0 to 100; higher scores indicate better satisfaction.
Efficacy Endpoints-Rescue Therapy
Time frame: Up to week 16
Proportion of subjects requiring rescue therapy
Safety Endpoints-AEs
Time frame: Up to week 16
Any local and systemic AEs (Adverse Events)/serious AEs
Safety Endpoints-LSR
Time frame: Up to week 16
Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.
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 16 Mar 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Tony Andrasfay study director
Therapeutics, Inc.
5 sites 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 QRX003-2% Lotion, QRX003-4% Lotion QAM and QRX003-4% Lotion BID in people with Netherton Syndrome, run by Quoin Pharmaceuticals at 5 sites 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 14 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: under way, not looking for participants, as of the sponsor's last update on 16 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy Endpoints-1-point IGA (time frame: Up to week 16). The study lists 9 primary outcomes in all. 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 16 Mar 2026.