Trial · NCT05856526
A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome
NCT05856526, Phase 2, Phase 3, Terminated, Boehringer Ingelheim; results first posted 2026-03-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome. Trials, record NCT05856526. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05856526
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome
- NCT number
- NCT05856526
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 43 participants, actual
- Lead sponsor
- Boehringer Ingelheim
- Conditions
- Netherton Syndrome
- Interventions
- Spesolimab - solution for infusion, Placebo matching to spesolimab - solution for infusion, Spesolimab - solution for injection, Placebo matching to spesolimab - solution for injection
- First posted
- 2023-05-12
- Last updated
- 2026-03-12
- Results first posted
- 2026-03-12
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No) | 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. |
| Secondary | 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) | Baseline and at Week 16 after start of study treatment administration. | 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. |
| Secondary | IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | At Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. | 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. |
| Secondary | 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) | At baseline and at Week 4, at Week 8, at Week 12 after start of study treatment administration. | 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. |
| Secondary | 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) | At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. | 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. |
| Secondary | 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) | At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. | 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. |
| Secondary | Percent Change From Baseline in IASI Score at Weeks 4, 8, 12 and 16 | 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. | 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. |
| Secondary | Absolute Change From Baseline in NRS Pain at Weeks 4, 8, 12 and 16 | 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 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. |
| Secondary | Absolute Change From Baseline in NRS Itch at Weeks 4, 8, 12 and 16 | 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. | 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. |
| Secondary | Absolute Change From Baseline in DLQI Score at Weeks 8 and 16 | 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. | 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. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No) | At baseline and at Week 16. | FAS (EC-NRI). This patient set included all patients who were randomised and received at least one study treatment in this trial. EC= primary estimand where use of restricted medications and treatments for NS is considered as non-response. NRI=Imputes missing data as failure, unless there are successful observations at visits both before and after the missing outcome. | proportion of participants | NUMBER |
| SECONDARY | 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) | Baseline and at Week 16 after start of study treatment administration. | FAS (EC-NRI). This patient set included all patients who were randomised and received at least one study treatment in this trial. EC= primary estimand where use of restricted medications and treatments for NS is considered as non-response. NRI=Imputes missing data as failure, unless there are successful observations at visits both before and after the missing outcome. | proportion of participants | NUMBER |
| SECONDARY | IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | At Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. | FAS (EC-NRI). This patient set included all patients who were randomised and received at least one study treatment in this trial. EC= primary estimand where use of restricted medications and treatments for NS is considered as non-response. NRI=Imputes missing data as failure, unless there are successful observations at visits both before and after the missing outcome. | proportion of participants | NUMBER |
| SECONDARY | 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) | At baseline and at Week 4, at Week 8, at Week 12 after start of study treatment administration. | FAS (EC-NRI). This patient set included all patients who were randomised and received at least one study treatment in this trial. EC= primary estimand where use of restricted medications and treatments for NS is considered as non-response. NRI=Imputes missing data as failure, unless there are successful observations at visits both before and after the missing outcome. | proportion of participants | NUMBER |
| SECONDARY | 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) | At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. | FAS (EC-NRI). This patient set included all patients who were randomised and received at least one study treatment in this trial. EC= primary estimand where use of restricted medications and treatments for NS is considered as non-response. NRI=imputes missing data as failure, unless there are successful observations at visits both before and after the missing outcome. | proportion of participants | NUMBER |
| SECONDARY | 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) | At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. | FAS (EC-NRI). This patient set included all patients who were randomised and received at least one study treatment in this trial. EC= primary estimand where use of restricted medications and treatments for NS is considered as non-response. NRI=imputes missing data as failure, unless there are successful observations at visits both before and after the missing outcome. | proportion of participants | MEAN |
| SECONDARY | Percent Change From Baseline in IASI Score at Weeks 4, 8, 12 and 16 | 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. | FAS (EH-MMRM). This patient set included all patients who were randomised and received at least one study treatment in this trial. EH=estimand where data after any use of restricted medications and treatments for NS and treatment discontinuation is censored. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in NRS Pain at Weeks 4, 8, 12 and 16 | 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. | FAS (EH-MMRM). This patient set included all patients who were randomised and received at least one study treatment in this trial. EH=estimand where data after any use of restricted medications and treatments for NS and treatment discontinuation is censored. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in NRS Itch at Weeks 4, 8, 12 and 16 | 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. | FAS (EH-MMRM). This patient set included all patients who were randomised and received at least one study treatment in this trial. EH=estimand where data after any use of restricted medications and treatments for NS and treatment discontinuation is censored. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in DLQI Score at Weeks 8 and 16 | 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. | FAS (EH-MMRM). This patient set included all patients who were randomised and received at least one study treatment in this trial. EH=estimand where data after any use of restricted medications and treatments for NS and treatment discontinuation is censored. Participants \>= 18 years and with a baseline DLQI score value are reported. | units on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group68
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No) | Placebo | 0.133 | 95% Confidence Interval 0.037 to 0.379 | proportion of participants | |
| IASI Response, Defined as a Decrease of at Least 50% Absolute Change in IASI Score From Baseline at Week 16 (Yes/No) | Spesolimab | 0.286 | 95% Confidence Interval 0.153 to 0.471 | proportion of participants | |
| 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) | Placebo | 0.400 | 95% Confidence Interval 0.198 to 0.643 | proportion of participants | |
| 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) | Spesolimab | 0.357 | 95% Confidence Interval 0.207 to 0.542 | proportion of participants | |
| IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | Placebo | Week 4 | 0.000 | 95% Confidence Interval 0.000 to 0.204 | proportion of participants |
| IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | Spesolimab | Week 4 | 0.036 | 95% Confidence Interval 0.006 to 0.177 | proportion of participants |
| IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | Placebo | Week 8 | 0.000 | 95% Confidence Interval 0.000 to 0.204 | proportion of participants |
| IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | Spesolimab | Week 8 | 0.000 | 95% Confidence Interval 0.000 to 0.121 | proportion of participants |
| IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | Placebo | Week 12 | 0.000 | 95% Confidence Interval 0.000 to 0.204 | proportion of participants |
| IGA Score of 0 or 1 at Weeks 4, 8, 12 and 16 (Yes/No) | Spesolimab | Week 12 | 0.036 | 95% Confidence Interval 0.006 to 0.177 | proportion of participants |
1 to 10 of 68Source ClinicalTrials.gov
See all 68 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Randomised treatment period | STARTED | Placebo | 15 | |
| Randomised treatment period | STARTED | Spesolimab | 28 | |
| Randomised treatment period | COMPLETED | Placebo | 15 | |
| Randomised treatment period | COMPLETED | Spesolimab | 27 | |
| Randomised treatment period | NOT COMPLETED | Placebo | 0 | |
| Randomised treatment period | NOT COMPLETED | Spesolimab | 1 | |
| Crossover treatment period | STARTED | Placebo | 15 | |
| Crossover treatment period | STARTED | Spesolimab | 27 | |
| Crossover treatment period | COMPLETED | Placebo | 13 | |
| Crossover treatment period | COMPLETED | Spesolimab | 20 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events220
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Netherton's syndrome | Congenital, familial and genetic disorders | Randomised Period: Placebo | 1 | 15 | |
| other | Netherton's syndrome | Congenital, familial and genetic disorders | Randomised Period: Spesolimab | 4 | 28 | |
| other | Netherton's syndrome | Congenital, familial and genetic disorders | Spesolimab Crossover Period | 0 | 27 | |
| other | Netherton's syndrome | Congenital, familial and genetic disorders | Placebo Crossover Period | 0 | 15 | |
| other | Netherton's syndrome | Congenital, familial and genetic disorders | Spesolimab Open Label | 7 | 42 | |
| other | Haemorrhoids | Gastrointestinal disorders | Randomised Period: Placebo | 1 | 15 | |
| other | Haemorrhoids | Gastrointestinal disorders | Randomised Period: Spesolimab | 0 | 28 | |
| other | Haemorrhoids | Gastrointestinal disorders | Spesolimab Crossover Period | 0 | 27 | |
| other | Haemorrhoids | Gastrointestinal disorders | Placebo Crossover Period | 0 | 15 | |
| other | Haemorrhoids | Gastrointestinal disorders | Spesolimab Open Label | 0 | 42 |
1 to 10 of 220Source ClinicalTrials.gov
See all 220 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Randomised Period: Placebo | 0 | 15 |
| Total, all-cause mortality | Randomised Period: Spesolimab | 0 | 28 |
| Total, all-cause mortality | Spesolimab Crossover Period | 0 | 27 |
| Total, all-cause mortality | Placebo Crossover Period | 0 | 15 |
| Total, all-cause mortality | Spesolimab Open Label | 0 | 42 |
| Total, other adverse events | Randomised Period: Placebo | 11 | 15 |
| Total, other adverse events | Randomised Period: Spesolimab | 12 | 28 |
| Total, other adverse events | Spesolimab Crossover Period | 1 | 27 |
| Total, other adverse events | Placebo Crossover Period | 4 | 15 |
| Total, other adverse events | Spesolimab Open Label | 25 | 42 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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