Trial Outcomes & Findings for This Study Tests How BI 655130 Works in Patients With Active Ulcerative Colitis. The Study Also Tests How Well BI 655130 is Tolerated and Whether it Helps the Patients (NCT NCT03100864)
NCT ID: NCT03100864
Last Updated: 2025-10-16
Results Overview
The total number of deregulated genes comparing baseline to post treatment, analysed by gene expression of mucosal biopsies via RNA sequencing, per time point up to Week 12. A total of 60,675 genes were evaluated, 40,586 genes were included in the differential expression analyses. Based on the raw read count values the DESeq2 method, one of the standard methods to analyse RNAseq data, was used for the gene expression analysis and to identify deregulated genes. A gene was considered deregulated with a FDR (false discovery rate) adjusted p-value \< 0.01 and a fold change ≤ -1.3 or ≥ 1.3.
COMPLETED
PHASE2
8 participants
Measurements done at baseline (day -8 to -6), day 1, day 4, day 15, day 57 and day 85 (week 12).
2025-10-16
Participant Flow
This was an Phase IIa multi-centre, non-randomised, uncontrolled single arm), open-label, exploratory trial to assess biomarker changes in response to Interleukin-36 signalling blockade induced by treatment with spesolimab in patients with moderate to severe active Ulcerative colitis.
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.
Participant milestones
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Overall Study
STARTED
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8
|
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Overall Study
COMPLETED
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8
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
This Study Tests How BI 655130 Works in Patients With Active Ulcerative Colitis. The Study Also Tests How Well BI 655130 is Tolerated and Whether it Helps the Patients
Baseline characteristics by cohort
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=8 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Age, Continuous
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43.1 years
STANDARD_DEVIATION 19.1 • n=5 Participants
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Sex: Female, Male
Female
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3 Participants
n=5 Participants
|
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Sex: Female, Male
Male
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5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Measurements done at baseline (day -8 to -6), day 1, day 4, day 15, day 57 and day 85 (week 12).Population: Completers analysis set: completed the trial medication through to end of trial visit, had a baseline and at least 1 post baseline measurement for any clinical efficacy or biomarker endpoint without any Important protocol deviation flagged for exclusion or rescue use on or after Visit 1b but prior to administration of the first dose of spesolimab.
The total number of deregulated genes comparing baseline to post treatment, analysed by gene expression of mucosal biopsies via RNA sequencing, per time point up to Week 12. A total of 60,675 genes were evaluated, 40,586 genes were included in the differential expression analyses. Based on the raw read count values the DESeq2 method, one of the standard methods to analyse RNAseq data, was used for the gene expression analysis and to identify deregulated genes. A gene was considered deregulated with a FDR (false discovery rate) adjusted p-value \< 0.01 and a fold change ≤ -1.3 or ≥ 1.3.
Outcome measures
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=5 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
|
The Total Number of Deregulated Genes Comparing Baseline to Post Treatment, Analysed by Gene Expression of Mucosal Biopsies Via RNA Sequencing, Per Time Point up to Week 12
Change from baseline to Day 1
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3 deregulated genes
|
|
The Total Number of Deregulated Genes Comparing Baseline to Post Treatment, Analysed by Gene Expression of Mucosal Biopsies Via RNA Sequencing, Per Time Point up to Week 12
Change from baseline to Day 4
|
5 deregulated genes
|
|
The Total Number of Deregulated Genes Comparing Baseline to Post Treatment, Analysed by Gene Expression of Mucosal Biopsies Via RNA Sequencing, Per Time Point up to Week 12
Change from baseline to Day 15 (week 2)
|
2 deregulated genes
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|
The Total Number of Deregulated Genes Comparing Baseline to Post Treatment, Analysed by Gene Expression of Mucosal Biopsies Via RNA Sequencing, Per Time Point up to Week 12
Change from baseline to Day 57 (week 8)
|
7 deregulated genes
|
|
The Total Number of Deregulated Genes Comparing Baseline to Post Treatment, Analysed by Gene Expression of Mucosal Biopsies Via RNA Sequencing, Per Time Point up to Week 12
Change from baseline to Day 85 (week 12)
|
9 deregulated genes
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SECONDARY outcome
Timeframe: Measurements done at baseline (day -8 to -6) and week 12 (day 85).Population: Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
Percent change in C-reactive protein (CRP) from baseline to Week 12 (day 85).
Outcome measures
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=5 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Percent Change in C-reactive Protein (CRP) From Baseline to Week 12
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-79.6 percentage change (%)
Interval -97.9 to 936.7
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SECONDARY outcome
Timeframe: Measurements done at baseline (day -8 to -6) and week 12 (day 85).Population: Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
Percent change in faecal calprotectin from baseline to week 12 (day 85).
Outcome measures
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=5 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Percent Change in Faecal Calprotectin From Baseline to Week 12
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13.0 percentage change (%)
Interval -98.7 to 219.3
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SECONDARY outcome
Timeframe: Measurements done at baseline (day -8 to -6) and week 12 (day 85).Population: Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
Percent change in faecal lactoferrin from baseline to week 12 (day 85).
Outcome measures
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=5 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Percent Change in Faecal Lactoferrin From Baseline to Week 12
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0.4 percentage change (%)
Interval -99.7 to 388.7
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SECONDARY outcome
Timeframe: Week 12 (day 85) following start of treatment.Population: Full analysis set (FAS): patients who received at least 1 dose of study drug, who had a baseline and at least 1 post baseline measurement for any clinical efficacy or biomarker endpoint without any Important protocol deviation flagged for exclusion or rescue use on or after Visit 1b but prior to administration of the first dose of spesolimab.
Number of participants with clinical remission (defined as Mayo score ≤2 points, and all subscores ≤1 point) at Week 12. The Mayo score is a composite disease activity score consisting of 4 items or subscores: stool frequency (relative to normal), rectal bleeding, physician's global assessment (PGA), and endoscopic appearance. The overall range of the Mayo score was 0 to 12 (higher scores being worse) and each subscore had a range of 0 to 3.
Outcome measures
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=5 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Number of Participants With Clinical Remission (Defined as Mayo Score ≤2 Points, and All Subscores ≤1 Point) at Week 12
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0 Participants
Interval 0.0 to 0.434
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SECONDARY outcome
Timeframe: Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.Population: Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
Number of patients with drug related adverse events (AEs) during the on-treatment period.
Outcome measures
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=8 Participants
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Number of Patients With Drug Related Adverse Events (AEs)
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6 Participants
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Adverse Events
Spesolimab 1200 mg Intravenous (i.v.)
Serious adverse events
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=8 participants at risk
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
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|---|---|
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Gastrointestinal disorders
Colitis
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12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Infections and infestations
Diverticulitis
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12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
Other adverse events
| Measure |
Spesolimab 1200 mg Intravenous (i.v.)
n=8 participants at risk
1200 milligram Spesolimab (infusion solution, BI 655130) was given for 12 weeks intravenously with a concentration of 20 milligram/milliliter every four weeks (on Day 1, Week 4, and Week 8).
|
|---|---|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
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12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Eye disorders
Conjunctival haemorrhage
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12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Eye disorders
Eye inflammation
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25.0%
2/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Abdominal pain upper
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12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Colitis ulcerative
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12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Dyspepsia
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
General disorders
Fatigue
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Infections and infestations
Gastrointestinal infection
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Infections and infestations
Nasopharyngitis
|
50.0%
4/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Infections and infestations
Tonsillitis
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Investigations
Amylase increased
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Investigations
Lipase increased
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Nervous system disorders
Headache
|
25.0%
2/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
|
Vascular disorders
Orthostatic hypotension
|
12.5%
1/8 • Date of start of infusion of first study drug (Day 1) till the date of end of infusion of last study drug (day 57) + 140 days at 11:59 p.m., up to 197 days.
Safety analysis set (SAF): This patient set included all entered patients who received at least 1 dose of study drug.
|
Additional Information
Boehringer Ingelheim, Call Center
Boehringer Ingelheim
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER