Netherton SyndromePhase 1/2NCT05211830
A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome
A Phase 1/2 study testing SXR1096 cream in people with Netherton Syndrome, run by Sixera Pharma at 5 sites in 4 countries.
Trial summary
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndroms, a rare inflammatory skin disease. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Netherton Syndrome
- What is being tested?
- SXR1096 cream
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 7 Dec 2021to 20 Aug 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patients aged 18 to 65 years at the screening visit and also adolescents (12-17 years) only after initial cohort of 5 adult patients have been treated for at least three days.
- Patients/legal guardian endpoint must be willing to provide written informed consent.
- Clinical diagnosis of NS including at least 3 out of the 4 following clinical criteria;
- Neonatal erythroderma
- Bamboo hair and/or alopecia
- Chronic atopy specified as food allergy, asthma, rhino conjunctivitis and/or eczema for at least 2 years
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- Ichthyosis linearis circumflexa
- Patients must be willing and able to understand and can comply with study requirements, apply the medication as instructed and be able to complete the study.
- Absent LEKTI on immunohistochemistry of skin biopsy and/or confirmed mutation in SPINK5 gene
- NS involvement ≥ 20% of Body Surface Area (BSA) required at both the screening and baseline visits.
- Investigator Global Assessment (IGA) of two areas to be treated, score ≥3, i.e. moderate or severe for each area required. Each target area approx. 9% of BSA. i.e. equal to one arm.
- Female of childbearing potential must either commit true abstinence when this is in line with the preferred and usual lifestyle or use an adequate and approved method of contraception throughout the study and for 4 weeks after the last study drug application. This criterion also applies to a prepubertal female subject who begins menses during the study.
- Adequate and approved methods of contraception applicable for the subject and/or her partner are defined below:
- Progestogen-only oral hormonal contraception
- Combination of male or female condom with cap, diaphragm, or sponge with spermicide (double barrier methods)
- Combined (estrogen- and progestogen-containing) oral, intravaginal, or transdermal hormonal contraception
- Injectable or implanted hormonal contraception
- Intrauterine devices or intrauterine hormone-releasing system
- Bilateral tubal ligation or tube insert (such as the Essure system) at least 3 months before the study
- Vasectomy of partner at least 3 months before the study
- Female subjects of non-childbearing potential must meet one of the following criteria:
- Absence of menstrual bleeding for 1 year prior to screening without any other medical reason
- Documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy at least 3 months before screening
Who cannot join
- Female patient who is pregnant, nursing an infant or planning a pregnancy throughout the course of the study
- Patient with any uncontrolled systemic disease. A potential patient in whom therapy for a systemic disease is not yet stabile for at least 3 months will not be considered for entry into the study.
- Patient with positive serology tests like HIV, HCV \& HBsAg.
- Patient with presence of any skin disease that might interfere with the diagnosis or evaluation of the test medications. Cutaneous infection within 1 week before the baseline visit or, any infection requiring treatment with oral or parenteral antibiotics, antivirals, antiparasitics, or antifungals within 2 weeks before the baseline visit.
- Patient that has a condition or is in a situation, which in the investigator's opinion may put the patient at significant risk, may confound the study results, or may interfere significantly with the patient's participation in the study.
- Use of topical drugs that might alter the course of NS (e.g., topical corticosteroids and topical calcineurin inhibitors) within two weeks before baseline visit.
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- Patient with a known sensitivity to any of the study treatments and/or their components.
- Patient who anticipates a need to use other topical or systemic therapy that might alter the course of NS. Emollients/creams can be used on remaining skin area but not the test areas. Use of topical prescription treatment within 2 weeks prior to initial dosing of study drug. Recent systemic treatment for NS (e.g. systemic corticosteroids, antibiotics, immunosuppressant, biologic and biosimilars treatments). A washout period of 4 weeks will be required for such patients to be eligible to participate in the trial.
- Patient who anticipates the need for surgery or hospitalization during the study.
- Concurrent involvement in any other clinical study/expanded access program with an investigational drug or device, or participation in a clinical study within 30 days prior to entering the study.
- Suspected or confirmed COVID-19 infection within 4 weeks before the screening or baseline visit. Unresolved COVID-19 infection. Planned vaccination for COVID-19 during screening, treatment period or before the follow-up visit.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
About this study
Official title: A Phase I/II, Multicenter, Randomized, Double-blind, Placebo Within-patient Controlled, First-in-human (FIH) Proof of Concept (PoC) Study to Evaluate the Safety and Efficacy of Topically Applied SXR1096 Cream in Patients With Netherton Syndrome (NS)
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndroms, a rare inflammatory skin disease. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing the disease state in Netherton syndrome patients. Patients will be treated for one month with active cream and placebo cream at different areas of the skin.
Read the detailed description
Netherton syndrome is a ultra-rare and very severe form of ichtyosis. It is a mongenetic disease. There is no specific treatment available for Netherton syndrome.
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndrom. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing the disease state in Netherton syndrome patients, as these patients have a gene defect in the gene spink5 encoding for the protease inhibitor LEKTI - that will normally regulate the activity of KLK5, 7 and 14 in the stratum corneum of the skin,
Patients will be treated for one month with active cream and placebo cream at different areas of the skin. The study will assess the safety and efficacy of the treatment in patients. There will also be pharmacokinetic, pharmacodynamic and biomarker studies conducted.
- Conditions
- Netherton Syndrome
- Being tested
- SXR1096 cream, Placebo cream
- Medical subject headings
- Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SXR1096 creamDrug
Skin cream to be applied twice a day to a predefined area of the skin
Placebo creamDrug
Skin cream to be applied twice a day to a predefined area of the skin
| Study group | Role | What they receive |
|---|---|---|
| SXR1096 cream | Experimental | The active treatment will be the specific small molecule inhibitor of KLK5, 7 and 14 in a proprietary skin cream formulation. Receives: Drug: SXR1096 cream |
| Placebo cream | Placebo comparator | The placebo control will be the proprietary skin cream formulation without the active substance. Receives: Drug: Placebo cream |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of the treatment with SXR1096 compared to placebo in initial cohort of adults
Number of patients with treatment-related adverse events during the treatment period of 4 weeks
Measured: 4 week
Safety of the treatment with SXR1096 compared to placebo in adults and adolescents
Number of patients with treatment-related adverse events during the treatment period of 4 weeks
Measured: 4 weeks
Efficacy of the treatment with SXR1096 compared to placebo in adults and adolescents
The change in Investigator Global Assessment (IGA) score 0-4 at EOT compared to baseline (0 being clear of lesions (erythema and scaling) and 4 being the worst score).
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2022
- Study start7 Dec 2021
- Primary completion10 Jun 2025
- Study completion20 Aug 2025
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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- Patients will act as their own controls as they will all be treating different contralateral areas of the skin with active and placebo creams in a blinded fashion.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sixera Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Maarten de Chateau, MD PhD, Study director, Sixera Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05211830
- Sponsor's protocol ID
- SXR001
- Status last verified
- Dec 2025
- First submitted
- 14 Oct 2021
- Last updated
- 16 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Multicenter, Randomized, Double-blind, Placebo Within-patient Controlled, First-in-human (FIH) Proof of Concept (PoC) Study to Evaluate the Safety and Efficacy of Topically Applied SXR1096 Cream in Patients With Netherton Syndrome (NS)
- ClinicalTrials.gov ID
- NCT05211830
- Acronym / sponsor ID
- SXR001
- Conditions
- Netherton Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Sixera PharmaIndustry (pharma, biotech, devices)
- Study start
- 7 Dec 2021 (actual)
- Primary completion
- 10 Jun 2025 (actual)
- Study completion
- 20 Aug 2025 (actual)
- First posted
- 27 Jan 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.
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndroms, a rare inflammatory skin disease. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing the disease state in Netherton syndrome patients. Patients will be treated for one month with active cream and placebo cream at different areas of the skin.
Detailed description
Netherton syndrome is a ultra-rare and very severe form of ichtyosis. It is a mongenetic disease. There is no specific treatment available for Netherton syndrome.
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndrom. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing the disease state in Netherton syndrome patients, as these patients have a gene defect in the gene spink5 encoding for the protease inhibitor LEKTI - that will normally regulate the activity of KLK5, 7 and 14 in the stratum corneum of the skin,
Patients will be treated for one month with active cream and placebo cream at different areas of the skin. The study will assess the safety and efficacy of the treatment in patients. There will also be pharmacokinetic, pharmacodynamic and biomarker studies conducted.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Patients will act as their own controls as they will all be treating different contralateral areas of the skin with active and placebo creams in a blinded fashion.
| Arm | Type | Receives |
|---|---|---|
| SXR1096 cream | Experimental | Drug: SXR1096 creamThe active treatment will be the specific small molecule inhibitor of KLK5, 7 and 14 in a proprietary skin cream formulation. |
| Placebo cream | Placebo comparator | Drug: Placebo creamThe placebo control will be the proprietary skin cream formulation without the active substance. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (23 listed)
- Male or female patients aged 18 to 65 years at the screening visit and also adolescents (12-17 years) only after initial cohort of 5 adult patients have been treated for at least three days.
- Patients/legal guardian endpoint must be willing to provide written informed consent.
- Clinical diagnosis of NS including at least 3 out of the 4 following clinical criteria;
- Patients must be willing and able to understand and can comply with study requirements, apply the medication as instructed and be able to complete the study.
- Absent LEKTI on immunohistochemistry of skin biopsy and/or confirmed mutation in SPINK5 gene
Key exclusion criteria (11 listed)
- Female patient who is pregnant, nursing an infant or planning a pregnancy throughout the course of the study
- Patient with any uncontrolled systemic disease. A potential patient in whom therapy for a systemic disease is not yet stabile for at least 3 months will not be considered for entry into the study.
- Patient with positive serology tests like HIV, HCV \& HBsAg.
- Patient with presence of any skin disease that might interfere with the diagnosis or evaluation of the test medications. Cutaneous infection within 1 week before the baseline visit or, any infection requiring treatment with oral or parenteral antibiotics, antivirals, antiparasitics, or antifungals within 2 weeks before the baseline visit.
- Patient that has a condition or is in a situation, which in the investigator's opinion may put the patient at significant risk, may confound the study results, or may interfere significantly with the patient's participation in the study.
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 |
|---|---|---|
| SXR1096 creamSkin cream to be applied twice a day to a predefined area of the skin | Drug | SXR1096 cream |
| Placebo creamSkin cream to be applied twice a day to a predefined area of the skin | Drug | Placebo cream |
Primary outcome
Safety of the treatment with SXR1096 compared to placebo in initial cohort of adults
Time frame: 4 week
Number of patients with treatment-related adverse events during the treatment period of 4 weeks
Safety of the treatment with SXR1096 compared to placebo in adults and adolescents
Time frame: 4 weeks
Number of patients with treatment-related adverse events during the treatment period of 4 weeks
Efficacy of the treatment with SXR1096 compared to placebo in adults and adolescents
Time frame: 4 weeks
The change in Investigator Global Assessment (IGA) score 0-4 at EOT compared to baseline (0 being clear of lesions (erythema and scaling) and 4 being the worst score).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Netherton Syndrome
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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.
Maarten de Chateau, MD PhD study director
Sixera Pharma
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SXR1096 cream in people with Netherton Syndrome, run by Sixera Pharma at 5 sites in 4 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 23 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: finished, as of the sponsor's last update on 16 Dec 2025. 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 France, Austria, Germany, Sweden. 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety of the treatment with SXR1096 compared to placebo in initial cohort of adults (time frame: 4 week). The study lists 3 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 Dec 2025.