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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-501104-10-00 | Registry Identifier | CTIS (EU) | |
| U1111-1289-6825 | Registry Identifier | WHO International Clinical Trials Registry Platform (ICTRP) |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator |
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| Spesolimab | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Spesolimab - solution for infusion | Drug | Solution for infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | At baseline and at Week 16. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mission Dermatology Center | Rancho Santa Margarita | California | 92688 | United States | ||
| Yale University School of Medicine |
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| Label | URL |
|---|---|
| Related Info | View source |
| Related Info | View source |
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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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All subjects were screened for eligibility prior to participation in the trial. Subjects attended a specialist site which ensured that they (the subjects) strictly met all inclusion and none of the exclusion criteria. Subjects were not to be allocated to a treatment group if any of the entry criteria were violated.
This was a placebo-controlled trial to evaluate the efficacy and safety of spesolimab in the treatment of patients with Netherton syndrome.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Randomised period Participants received placebo matching spesolimab from Week 0 to Week 16. Crossover period At Week 16 participants received Spesolimab at higher dose and placebo. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks. Extended treatment Period Non-responding participants receiving medium dose of spesolimab were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomised treatment period |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 24, 2024 | Jan 9, 2026 |
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| Placebo matching to spesolimab - solution for infusion | Drug | Solution for infusion |
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| Spesolimab - solution for injection | Drug | Solution for injection |
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| Placebo matching to spesolimab - solution for injection | Drug | Solution for injection |
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| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | From first study treatment administration until end of randomised period (Week 16) plus 16 weeks of residual effect period, up to 32 weeks. |
| New Haven |
| Connecticut |
| 06519 |
| United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Virginia Clinical Research, Inc. | Norfolk | Virginia | 23502 | United States |
| Westmead Hospital | Westmead | New South Wales | 2145 | Australia |
| ASMC-IPSMC-skin and Veneral Diseases | Sofia | 1407 | Bulgaria |
| Beijing Children's Hospital, Capital Medical University | Beijing | 100045 | China |
| Southern Medical University Dermatology Hospital | Guangzhou | 510091 | China |
| The Children's Hospital Zhejiang University School Of Medicine | Hangzhou | 310000 | China |
| The First Affiliated Hospital, Zhejiang University | Hangzhou | 310003 | China |
| Dermatology Hospital, Chinese Academy of Medical Sciences | Nanjing | 210000 | China |
| Shanghai Skin Disease Hospital | Shanghai | 200000 | China |
| Xinhua Hospital Affiliated to Shanghai Jiaotong University | Shanghai | 200092 | China |
| HOP Saint-Louis | Paris | 75010 | France |
| Universitätsklinikum Heidelberg | Heidelberg | 69120 | Germany |
| Universitätsklinikum Schleswig-Holstein, Campus Kiel | Kiel | 24105 | Germany |
| Klinikum der Universität München AÖR | München | 80337 | Germany |
| Sourasky Medical Center | Tel Aviv | 6423906 | Israel |
| Istituto Dermopatico Dell'Immacolata - IDI - IRCCS | Roma | 00167 | Italy |
| Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino | Torino | 10126 | Italy |
| Nagoya University Hospital | Aichi, Nagoya | 466-8560 | Japan |
| Juntendo University Urayasu Hospital | Chiba, Urayasu | 279-0021 | Japan |
| Okayama University Hospital | Okayama, Okayama | 700-8558 | Japan |
| Hospital Tunku Azizah | Kuala Lumpur | 50300 | Malaysia |
| Erasmus MC Sophia Kinderziekenhuis | Rotterdam | 3015 GD | Netherlands |
| ULS de São José, E.P.E. - Hospital Sto. António Capuchos | Lisbon | 1169-050 | Portugal |
| University Children Hospital Zürich | Zurich | 8032 | Switzerland |
| Queen Elizabeth University Hospital | Glasgow | G51 4TF | United Kingdom |
| FG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| Crossover treatment period |
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| Open label treatment period |
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| Extended treatment period |
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Full analyses set (FAS): This patient set included all patients who were randomised and received at least one study treatment in this trial.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Randomised period Participants received placebo matching spesolimab from Week 0 to Week 16. Crossover period At Week 16 participants received Spesolimab at higher dose and placebo. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks. Extended treatment Period Non-responding participants receiving medium dose of spesolimab were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
| BG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| IASI score at baseline | 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. | Mean | Standard Deviation | score on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | 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. | 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. | Posted | Number | 95% Confidence Interval | proportion of participants | At baseline and at Week 16. |
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| 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) | 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. | 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. | Posted | Number | 95% Confidence Interval | proportion of participants | Baseline and at Week 16 after start of study treatment administration. |
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| Secondary | 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. | 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. | Posted | Number | 95% Confidence Interval | proportion of participants | At Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. |
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| 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) | 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. | 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. | Posted | Number | 95% Confidence Interval | proportion of participants | At baseline and at Week 4, at Week 8, at Week 12 after start of study treatment administration. |
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| 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) | 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. | 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. | Posted | Number | 95% Confidence Interval | proportion of participants | At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. |
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| 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) | 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. | 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. | Posted | Mean | 95% Confidence Interval | proportion of participants | At baseline and at Week 4, at Week 8, at Week 12, at Week 16 after start of study treatment administration. |
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| Secondary | 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. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | 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. |
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| Secondary | 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. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | 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. |
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| Secondary | 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. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | 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. |
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| Secondary | 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. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | 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. |
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| Secondary | 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. | FAS (EH). 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. Only paediatric (>= 12 to < 18 years) participants with data available for the CDLQI score at Week 8 and at Week 16 are reported. | Posted | Mean | Standard Deviation | units on a scale | At baseline and at Week 8 and at Week 16 after study treatment administration. |
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| Secondary | 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. | Safety Analysis Set (SAF): This patient set included all patients who were randomised and received at least one study treatment in this trial. | Posted | Number | percentage of participants | From first study treatment administration until end of randomised period (Week 16) plus 16 weeks of residual effect period, up to 32 weeks. |
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"Randomised period: Placebo" and "Randomised period: Spesolimab": From treatment start until end of Week 16 + 16 weeks residual effect period (REP), up to 32 weeks. "Placebo crossover period" and "Spesolimab crossover period": From Week 16 to Week 20 + 16 weeks REP, up to 20 weeks. "Spesolimab open label": From Week 20 to end of study treatment administration at Week 78 of Extended Treatment period +16 weeks REP, up to 74 weeks.
Safety Analysis Set (SAF): This patient set included all patients who were randomised and received at least one study treatment in this trial.
"Spesolimab open label" arm includes all patients who received open label spesolimab from Week 20 of the Crossover period until end of Extended Treatment Period.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Randomised Period: Placebo | Participants received placebo matching spesolimab from Week 0 to Week 16. | 0 | 15 | 0 | 15 | 11 | 15 |
| EG001 | Randomised Period: Spesolimab | Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. | 0 | 28 | 3 | 28 | 12 | 28 |
| EG002 | Spesolimab Crossover Period | Participants who received spesolimab from Week 0 to Week 16, at Week 16 participants received placebo and a medium or low dose of spesolimab. | 0 | 27 | 1 | 27 | 1 | 27 |
| EG003 | Placebo Crossover Period | Participants who received placebo from Week 0 to Week 16, at Week 16 participants received Spesolimab at higher dose and placebo. | 0 | 15 | 0 | 15 | 4 | 15 |
| EG004 | Spesolimab Open Label | This arm includes all patients who were initially randomised to placebo or Spesolimab, who received from Week 20 of Crossover period to end of the Extended Treatment period either a medium dose of spesolimab or a low dose of spesolimab. | 0 | 42 | 5 | 42 | 25 | 42 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Netherton's syndrome | Congenital, familial and genetic disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Chronic sinusitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Nasal vestibulitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Tenosynovitis | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 28.0 | Systematic Assessment |
| |
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | MedDRA 28.0 | Systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA 28.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Netherton's syndrome | Congenital, familial and genetic disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Injection site hypertrophy | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Herpes simplex | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Periodontitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Pyoderma | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Skin bacterial infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
| |
| Amylase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Blood fibrinogen increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Protein urine present | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Gouty arthritis | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Boehringer Ingelheim, Call Center | Boehringer Ingelheim | 1-800-243-0127 | clintriage.rdg@boehringer-ingelheim.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 23, 2025 | Jan 9, 2026 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D056770 | Netherton Syndrome |
| ID | Term |
|---|---|
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016113 | Ichthyosiform Erythroderma, Congenital |
| D007057 | Ichthyosis |
| D012868 | Skin Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D007232 | Infant, Newborn, Diseases |
| D007642 | Keratosis |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000712973 | spesolimab |
| D007267 | Injections |
| ID | Term |
|---|---|
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
Not provided
Not provided
| Perceived lack of efficacy |
|
| Adverse Event |
|
| Study terminated by sponsor |
|
| Burden of study procedure |
|
| Change of residence |
|
| Perceived lack of efficacy |
|
| Study terminated by sponsor |
|
| Male |
|
| Asian |
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| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Not Hispanic or Latino |
|
| Not Stated |
|
| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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| OG001 | Spesolimab | Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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Randomised period Participants received at Week 0 a high dose of spesolimab. Starting from Week 4 until Week 16 participants received every 4 weeks a medium dose of spesolimab. Crossover period At Week 16 participants received placebo and a medium dose of spesolimab. Open label period After cross-over period, participants entered the open label period to receive spesolimab at medium dose every 4 weeks. At Week 32, patients were evaluated for response to treatment. Responders received a low dose of spesolimab every 4 weeks, while non-responders continued receiving medium dose of spesolimab every 4 weeks until Week 52. Extended treatment Period Non-responding participants were discontinued from the trial. Participants receiving low dose of spesolimab and experiencing a non-response were not to be discontinued, but up-titrated to medium dose first. Only if the patients still didn't show response to treatment, participants were discontinued. |
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