Netherton SyndromePhase 2/3NCT05856526
A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome
A Phase 2/3 study testing Spesolimab - solution for infusion and Spesolimab - solution for injection in people with Netherton Syndrome, run by Boehringer Ingelheim at 28 sites in 14 countries.
Trial summary
This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years or older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS. Participants are divided into a spesolimab and a placebo group. 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?
- Spesolimab - solution for infusion and Spesolimab - solution for injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 28 sitesin 14 countries
- Study dates
- 12 Jun 2023to 31 Jul 2025
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older (weight minimum is 35kg).
- Confirmed diagnosis of Netherton syndrome (NS) (causative SPINK5 mutations) at baseline (Visit 2).
- At least moderate severity of erythema at baseline (visit 2) (Ichthyosis Area Severity Index (IASI) score ≥ 16 and IASI-Erythema (E) score ≥8) and ≥ 3 on Investigator Global Assessment (IGA) score.
- Signed and dated written informed consent and assent in accordance with International Council on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to admission in the trial
- Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the clinical trial protocol (CTP) as well as in the patient, parent(s) (or patient's legal guardian) information.
Who cannot join
- Patients who have used topical corticosteroids (medium to high, US class I-V), topical retinoids, topical calcineurin inhibitors or keratolytics within 1 week prior to randomisation
- Patients who have used emollient on the area to be biopsied in the previous 24 hours
- Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
- Patients who have used systemic antibiotics within 2 weeks prior to randomisation
- Patients who have received live vaccines within 4 weeks prior to randomisation
- Patients who have received investigational products, biologics or immunoglobulins within 4 weeks or 5 half-lives (whichever is longer) prior to randomisation
Show all 9Show fewer
- Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Limit of Normal (ULN) elevation in Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin
- Patients who have any prior exposure to BI 655130 or another interleukin 36 receptor (IL-36R) inhibitor biologics
- Further exclusion criteria apply
Trial locations and contacts
28 sites in 14 countries.
28 sites in 14 countries
China7 sites
Beijing Children's Hospital, Capital Medical University
Beijing, 100045
Southern Medical University Dermatology Hospital
Guangzhou, 510091
The Children's Hospital Zhejiang University School Of Medicine
Hangzhou, 310000
The First Affiliated Hospital, Zhejiang University
Hangzhou, 310003
Dermatology Hospital, Chinese Academy of Medical Sciences
Nanjing, 210000
Shanghai Skin Disease Hospital
Shanghai, 200000
Xinhua Hospital Affiliated to Shanghai Jiaotong University
Shanghai, 200092
United States4 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Yale University School of Medicine
New Haven, Connecticut, 06519
Virginia Clinical Research, Inc.
Norfolk, Virginia, 23502
Mission Dermatology Center
Rancho Santa Margarita, California, 92688
Germany3 sites
Universitätsklinikum Heidelberg
Heidelberg, 69120
Universitätsklinikum Schleswig-Holstein, Campus Kiel
Kiel, 24105
Klinikum der Universität München AÖR
München, 80337
Japan3 sites
Nagoya University Hospital
Aichi, Nagoya, 466-8560
Juntendo University Urayasu Hospital
Chiba, Urayasu, 279-0021
Okayama University Hospital
Okayama, Okayama, 700-8558
Italy2 sites
Istituto Dermopatico Dell'Immacolata - IDI - IRCCS
Roma, 00167
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Torino, 10126
Portugal1 site
ULS de São José, E.P.E. - Hospital Sto. António Capuchos
Lisbon, 1169-050
United Kingdom1 site
Queen Elizabeth University Hospital
Glasgow, G51 4TF
About this study
Official title: EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome
This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years or older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS.
Participants are divided into a spesolimab and a placebo group. Placebo injections look like spesolimab injections but do not contain any medicine. Every participant has a 2 in 3 chance of being in the spesolimab group. In the beginning, participants get the study medicine as an injection into a vein. Afterwards, they get it as an injection under the skin every month.
Read more
After 4 months, participants in the placebo group switch to spesolimab treatment.
Participants are in the study for up to 3 years. During this time, they visit the study site up to 42 times. The doctors regularly check participants' NS symptoms. The results are compared between the groups to see whether spesolimab works. The doctors also regularly check participants' general health and take note of any unwanted effects.
- Conditions
- Netherton Syndrome
- Being tested
- Spesolimab - solution for infusion, Placebo matching to spesolimab - solution for infusion, Spesolimab - solution for injection, Placebo matching to spesolimab - solution for injection
- Medical subject headings
- Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Spesolimab - solution for infusionDrug
Also called BI 655130
Solution for infusion
Placebo matching to spesolimab - solution for infusionDrug
Solution for infusion
Spesolimab - solution for injectionDrug
Also called BI 655130
Solution for injection
Placebo matching to spesolimab - solution for injectionDrug
Solution for injection
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Spesolimab - solution for infusion; Drug: Placebo matching to spesolimab - solution for infusion; Drug: Spesolimab - solution for injection; Drug: Placebo matching to spesolimab - solution for injection |
| Spesolimab | Experimental | Not described Receives: Drug: Spesolimab - solution for infusion; Drug: Placebo matching to spesolimab - solution for infusion; Drug: Spesolimab - solution for injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No)
Proportion of participants with an Ichthyosis Area Severity Index (IASI) response at Week 16 is reported. IASI response was defined as a decrease of at least 50% absolute change in IASI score from baseline at Week 16. Proportions were rounded to 3 decimal places.
IASI is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for erythema and scaling.
The score ranges from 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity.
95% confidence intervals (CI) are calculated using the method of Wilson.
Measured: At baseline and at Week 16.
Secondary outcomes (11)
Key Secondary Endpoint: IGA Response, Defined as a Decrease of at Least 1-grade Absolute Change in IGA Score From Baseline at Week 16 (Yes/No)
Proportion of participants with an Investigator Global Assessment (IGA) response at Week 16 is reported. Proportions were rounded to 3 decimal places. IGA response was defined as a decrease of at least 1-grade absolute change in IGA score from baseline at Week 16.
IGA for Netherton Syndrome (NS) assessed the global severity of erythema and scaling in NS using a 5-point Likert scale ranging from 0=clear, to 4=severe.
95% confidence intervals (CI) are calculated using the method of Wilson.
Measured: Baseline and at Week 16 after start of study treatment administration.
IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No)
Proportion of participants with an Investigator Global Assessment (IGA) score of 0 or 1 at Week 4, at Week 8, at Week 12 and at Week 16 after first study treatment administration is reported. Proportions were rounded to 3 decimal places.
IGA for Netherton Syndrome (NS) assessed the global severity of erythema and scaling in NS using a 5-point Likert scale ranging from 0=clear, to 4=severe.
95% confidence intervals (CI) are calculated using the method of Wilson.
Measured: At Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.
IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Weeks 4, 8, and 12 (Yes/No)
Proportion of participants with an Ichthyosis Area Severity Index (IASI) response at Weeks 4, 8, 12 is reported. Proportions were rounded to 3 decimal places. IASI response was defined as a decrease of at least 50% absolute change in IASI score from baseline at Week 4, Week 8 and Week 12.
IASI is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for erythema and scaling.
The score ranges from 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity.
95% confidence intervals (CI) are calculated using the method of Wilson.
Measured: At baseline and at Week 4, at Week 8, at Week 12 after start of study treatment administration.
IASI-E Subscore Response, Defined as a Decrease of at Least 50% Absolute Change in IASI-E Subscore at Weeks 4, 8, 12, and 16 (Yes/No)
Proportion of participants with an Ichthyosis Area Severity Index-Erythema (IASI-E) subscore response at Weeks 4, 8, 12 and 16 is reported. Proportions were rounded to 3 decimal places. IASI-E response was defined as a decrease of at least 50% absolute change in IASI-E subscore from baseline at Week 4, at Week 8, at Week 12 and at Week 16.
IASI-E is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for erythema.
The score ranges from 0-24. A higher score means worse clinical severity. 95% confidence intervals (CI) are calculated using the method of Wilson.
Measured: At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.
IASI-S Subscore Response, Defined as a Decrease of at Least 50% Absolute Change in IASI-S Subscore From Baseline at Weeks 4, 8, 12, and 16 (Yes/No)
Proportion of participants with an Ichthyosis Area Severity Index-Scaling (IASI-S) subscore response at Weeks 4, 8, 12 and 16 is reported. Proportions were rounded to 3 decimal places. IASI-S response was defined as a decrease of at least 50% absolute change in IASI-S subscore from baseline at Week 4, at Week 8, at Week 12 and at Week 16.
IASI-S is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for scaling.
The score ranges from 0-24. A higher score means worse clinical severity. 95% confidence intervals (CI) are calculated using the method of Wilson.
Measured: At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.
Percent Change From Baseline in IASI Score at Weeks 4, 8, 12 and 16
Percent change from baseline in IASI score at Weeks 4, 8, 12 and 16 is reported. IASI is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for erythema and scaling. The score ranges from 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity.
Percent change of IASI score= (IASI score at Week X - IASI score at baseline)*100/(IASI score at baseline). Week X=4, 8, 12, 16.
Least Squares Mean (95% Confidence Interval) were calculated using mixed effect model with repeated measures (MMRM) which included the fixed, categorical effects of treatment at each visit, age group and the continuous effect of baseline at each visit as well as random effects of subject.
Measured: MMRM model included measurements from baseline and Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 60, 68 after study treatment administration. MMRM values of percent change from baseline at Week 4, 8, 12, 16 are presented.
Absolute Change From Baseline in NRS Pain at Weeks 4, 8, 12 and 16
Absolute change from baseline in Numeric Pain Rating Scale (NRS) pain at Weeks 4, 8, 12 and 16 is presented. The NRS is a unidimensional measure of pain intensity, including chronic pain. The 11-point numeric scale ranges from '0' representing 'no pain' to '10' representing worst pain imaginable.
Absolute change in NRS at Week X= (NRS pain at Week X) - (NRS pain at baseline). Week X= 4, 8, 12, 16.
Least Squares Mean (95% Confidence Interval) were calculated using mixed effect model with repeated measures (MMRM) which included the fixed, categorical effects of treatment at each visit, age group and the continuous effect of baseline at each visit as well as random effects of subject.
Measured: MMRM model included measurements from baseline and Week 4, 8, 12, 16, 20, 24, 28, 32, 40, 52, 64 after study treatment administration. MMRM values of absolute change from baseline in NRS pain at Week 4, 8, 12, 16 are presented.
Absolute Change From Baseline in NRS Itch at Weeks 4, 8, 12 and 16
The Numeric Pain Rating Scale (NRS) itch measures the intensity of itch. The scale ranges from '0' representing 'no itch' to 10 'worst imaginable itch'.
Absolute change in NRS itch at Week X= (NRS itch at Week X) - (NRS itch at baseline). Week X= 4, 8, 12, 16.
Least Squares Mean (95% Confidence Interval) were calculated using mixed effect model with repeated measures (MMRM) which included the fixed, categorical effects of treatment at each visit, age group and the continuous effect of baseline at each visit as well as random effects of subject.
Measured: MMRM model included measurements from baseline and Week 4, 8, 12, 16, 20, 24, 28, 32, 40, 52, 64 after study treatment administration. MMRM values of absolute change from baseline in NRS itch at Week 4, 8, 12, 16 are presented.
Absolute Change From Baseline in DLQI Score at Weeks 8 and 16
Dermatology Life Quality Index (DLQI) assesses various dermatologic conditions and it measures the quality of life. It contains 10 questions which are score from 0 (not relevant) to 3 (very much). Question 7 is a "yes"/ "no" question where "yes" is scored as 3. Total score ranges from 0 to 30 in total. Scores of 0-1 indicate no effect, scores of 2-5 indicate a small effect, scores of 6-10 indicate moderate effect, score 11-20 indicate very large effect and scores 21-30 indicate an extremely large effect of the syndrome on the participant.
Absolute change in DLQI score at Week X= (DLQI score at Week X) - (DLQI score at baseline). Week X= 8, 16.
Least Squares Mean (95% Confidence Interval) were calculated using mixed effect model with repeated measures (MMRM) which included the fixed, categorical effects of treatment at each visit, age group and the continuous effect of baseline at each visit as well as random effects of subject.
Measured: MMRM model included measurements from baseline and Week 8, 16, 24, 32, 52, after study treatment administration. MMRM values of absolute change from baseline in DLQI score at Week 8, 16 are presented.
Absolute Change From Baseline in CDLQI Score at Weeks 8 and 16
Children's Dermatology Life Quality Index (CDLQI) measures the impact of skin disease on the lives of children and young people. CDLQI total score is calculated by summing the scores of each question resulting in a range of 0 to 30. Scores of 0-1 indicate no effect, scores of 2-6 indicate a small effect, scores of 7-12 indicate moderate effect, score 13-18 indicate very large effect and scores 19-30 indicate an extremely large effect of the syndrome on the participant.
Absolute change in CDLQI score at Week X= (CDLQI score at Week X) - (CDLQI score at baseline). Week X= 8, 16.
Measured: At baseline and at Week 8 and at Week 16 after study treatment administration.
The Occurrence of Treatment Emergent Adverse Events Including Serious and/or Opportunistic Infections
Percentage of patients with treatment emergent adverse events including serious and/or opportunistic infections is presented. Percentages are rounded to one decimal place.
Measured: From first study treatment administration until end of randomised period (Week 16) plus 16 weeks of residual effect period, up to 32 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2023
- Study start12 Jun 2023
- Primary completion13 Jan 2025
- Study completion31 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Netherton SyndromeStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05856526
- Sponsor's protocol ID
- 1368-0104
- Other IDs
- 2022-501104-10-00, U1111-1289-6825
- Status last verified
- Feb 2026
- First submitted
- 26 Apr 2023
- Last updated
- 12 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.mystudywindow.com/msw/datatransparency
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome
- ClinicalTrials.gov ID
- NCT05856526
- Acronym / sponsor ID
- 1368-0104
- Conditions
- Netherton Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- 12 Jun 2023 (actual)
- Primary completion
- 13 Jan 2025 (actual)
- Study completion
- 31 Jul 2025 (actual)
- First posted
- 12 May 2023 (actual)
- Results
- Posted 12 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years or older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS.
Participants are divided into a spesolimab and a placebo group. Placebo injections look like spesolimab injections but do not contain any medicine. Every participant has a 2 in 3 chance of being in the spesolimab group. In the beginning, participants get the study medicine as an injection into a vein. Afterwards, they get it as an injection under the skin every month.
After 4 months, participants in the placebo group switch to spesolimab treatment.
Participants are in the study for up to 3 years. During this time, they visit the study site up to 42 times. The doctors regularly check participants' NS symptoms. The results are compared between the groups to see whether spesolimab works. The doctors also regularly check participants' general health and take note of any unwanted effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Spesolimab - solution for infusion, Drug: Placebo matching to spesolimab - solution for infusion, Drug: Spesolimab - solution for injection, Drug: Placebo matching to spesolimab - solution for injection |
| Spesolimab | Experimental | Drug: Spesolimab - solution for infusion, Drug: Placebo matching to spesolimab - solution for infusion, Drug: Spesolimab - solution for injection |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female patients, aged 12 years and older (weight minimum is 35kg).
- Confirmed diagnosis of Netherton syndrome (NS) (causative SPINK5 mutations) at baseline (Visit 2).
- At least moderate severity of erythema at baseline (visit 2) (Ichthyosis Area Severity Index (IASI) score ≥ 16 and IASI-Erythema (E) score ≥8) and ≥ 3 on Investigator Global Assessment (IGA) score.
- Signed and dated written informed consent and assent in accordance with International Council on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to admission in the trial
- Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the clinical trial protocol (CTP) as well as in the patient, parent(s) (or patient's legal guardian) information.
Key exclusion criteria (9 listed)
- Patients who have used topical corticosteroids (medium to high, US class I-V), topical retinoids, topical calcineurin inhibitors or keratolytics within 1 week prior to randomisation
- Patients who have used emollient on the area to be biopsied in the previous 24 hours
- Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
- Patients who have used systemic antibiotics within 2 weeks prior to randomisation
- Patients who have received live vaccines within 4 weeks prior to randomisation
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 |
|---|---|---|
| Spesolimab - solution for infusionAlso: BI 655130Solution for infusion | Drug | Placebo, Spesolimab |
| Placebo matching to spesolimab - solution for infusionSolution for infusion | Drug | Placebo, Spesolimab |
| Spesolimab - solution for injectionAlso: BI 655130Solution for injection | Drug | Placebo, Spesolimab |
| Placebo matching to spesolimab - solution for injectionSolution for injection | Drug | Placebo |
Primary outcome
IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No)
Time frame: At baseline and at Week 16.
Proportion of participants with an Ichthyosis Area Severity Index (IASI) response at Week 16 is reported. IASI response was defined as a decrease of at least 50% absolute change in IASI score from baseline at Week 16. Proportions were rounded to 3 decimal places. IASI is a composite score based on the global Congenital Ichthyosis Severity Instrument (CISI) score that captures differences in severity in different body regions as a function of their body surface area, and also standardizes the number of choices within the Likert scales for erythema and scaling. The score ranges from 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity. 95% confidence intervals (CI) are calculated using the method of Wilson.
11 secondary outcomes
Key Secondary Endpoint: IGA Response, Defined as a Decrease of at Least 1-grade Absolute Change in IGA Score From Baseline at Week 16 (Yes/No)
Time frame: Baseline and at Week 16 after start of study treatment administration.
IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No)
Time frame: At Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.
IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Weeks 4, 8, and 12 (Yes/No)
Time frame: At baseline and at Week 4, at Week 8, at Week 12 after start of study treatment administration.
IASI-E Subscore Response, Defined as a Decrease of at Least 50% Absolute Change in IASI-E Subscore at Weeks 4, 8, 12, and 16 (Yes/No)
Time frame: At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.
IASI-S Subscore Response, Defined as a Decrease of at Least 50% Absolute Change in IASI-S Subscore From Baseline at Weeks 4, 8, 12, and 16 (Yes/No)
Time frame: At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration.
Percent Change From Baseline in IASI Score at Weeks 4, 8, 12 and 16
Time frame: MMRM model included measurements from baseline and Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 60, 68 after study treatment administration. MMRM values of percent change from baseline at Week 4, 8, 12, 16 are presented.
Absolute Change From Baseline in NRS Pain at Weeks 4, 8, 12 and 16
Time frame: MMRM model included measurements from baseline and Week 4, 8, 12, 16, 20, 24, 28, 32, 40, 52, 64 after study treatment administration. MMRM values of absolute change from baseline in NRS pain at Week 4, 8, 12, 16 are presented.
Absolute Change From Baseline in NRS Itch at Weeks 4, 8, 12 and 16
Time frame: MMRM model included measurements from baseline and Week 4, 8, 12, 16, 20, 24, 28, 32, 40, 52, 64 after study treatment administration. MMRM values of absolute change from baseline in NRS itch at Week 4, 8, 12, 16 are presented.
Absolute Change From Baseline in DLQI Score at Weeks 8 and 16
Time frame: MMRM model included measurements from baseline and Week 8, 16, 24, 32, 52, after study treatment administration. MMRM values of absolute change from baseline in DLQI score at Week 8, 16 are presented.
Absolute Change From Baseline in CDLQI Score at Weeks 8 and 16
Time frame: At baseline and at Week 8 and at Week 16 after study treatment administration.
The Occurrence of Treatment Emergent Adverse Events Including Serious and/or Opportunistic Infections
Time frame: From first study treatment administration until end of randomised period (Week 16) plus 16 weeks of residual effect period, up to 32 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No. Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.mystudywindow.com/msw/datatransparency
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Netherton Syndrome
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.
The record lists no contact. Use the official record to reach the sponsor.
28 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Spesolimab - solution for infusion and Spesolimab - solution for injection in people with Netherton Syndrome, run by Boehringer Ingelheim at 28 sites in 14 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 5 inclusion and 9 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: stopped early, as of the sponsor's last update on 12 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 28 sites in China, United States, Germany, Japan, Italy and 9 more countries. 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 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?
Yes. Results are posted in the registry (first posted 12 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No) (time frame: At baseline and at 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 12 Mar 2026.