A Study Testing How BI 655130 Works in Patients With Fistulizing Crohn's Disease
- Registration Number
- NCT03752970
- Lead Sponsor
- Boehringer Ingelheim
- Brief Summary
This is a study in adults with Crohn's Disease who also have fistulas near the anus. The study has 2 parts. The first part is to find out more about what causes the fistulas. In this part of the study, tissue samples are taken from patients. The second part of the study tests whether a medicine called spesolimab (BI 655130) helps patients with Crohn's Disease.
Participants get study medication for 24 weeks. The participants are put into 2 groups. It is decided by chance who gets into which group. One group gets an intravenous drip that contains spesolimab every 4 weeks. The other group gets a placebo drip every 4 weeks. The placebo drip looks like the spesolimab drip, but contains no medicine.
The doctors regularly examine fistulas of the participants. The results of the fistula examinations are compared between the groups. The doctors also check the general health of the patients.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 27
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Study Cohort - Placebo Placebo Patients with perianal fistulising Crohn's disease received Placebo intravenously every 4 weeks (week 0, 4, 8). At week 12 achievement of combined perianal fistula remission was determined, patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Patients with combined perianal fistula remission remained on Placebo and were treated with placebo intravenously every 4 weeks (week 12, 16 and 20). Study Cohort - Spesolimab Spesolimab Patients with perianal fistulising Crohn's disease received Spesolimab 1200 milligram intravenously every 4 weeks (week 0, 4, 8, 12, 16 and 20). At week 12 Placebo patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Study Cohort - Placebo Spesolimab Patients with perianal fistulising Crohn's disease received Placebo intravenously every 4 weeks (week 0, 4, 8). At week 12 achievement of combined perianal fistula remission was determined, patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Patients with combined perianal fistula remission remained on Placebo and were treated with placebo intravenously every 4 weeks (week 12, 16 and 20).
- Primary Outcome Measures
Name Time Method The Total Number of Deregulated Genes at Week 4 Biopsies taken at screening (Week -3) and at week 4 of treatment. The total number of deregulated genes based on biopsies from the inner fistula orifice at Week 4 comparing changes in gene expression from baseline between the two treatment groups. For each gene, a repeated measures linear regression model was utilized with treatment (BI 655130 or Placebo), visit (baseline, week 4), treatment by visit interaction as fixed effect and patient as a blocking factor.
Changes will be quantified by log2 fold changes (FC) and associated False Discovery Rate (FDR) adjusted p-values.
Genes will be considered deregulated if they fulfil the following criteria:
* FDR adjusted p-value ≤ 0.05
* \|fold change\| ≥ 1.5 (\|log2 fold change\| ≥ 0.58)
- Secondary Outcome Measures
Name Time Method Number of Patients With Perianal Fistula Response at Week 12 At baseline (day 1) and week 12 (day 85) of treatment. Number of patients with perianal fistula response at Week 12 defined as closure of at least 50% of external openings, no drainage/discharge despite gentle finger compression of fistulas that were draining at baseline and without new emerging fistulas.
The no response imputation (NRI) approach is applied: missing visits where imputed whereby all subsequent visits after a patient took rescue medication for the disease under study or died due to any cause were considered to be missing.Number of Patients With Perianal Fistula Remission at Week 12 At baseline (day 1) and week 12 (day 85) of treatment. Proportion of patients with perianal fistula remission at Week 12 defined as closure of all external openings, no drainage/discharge despite gentle finger compression of fistulas that were draining at baseline and without new emerging fistulas.
The no response imputation (NRI) approach is applied: missing visits where imputed whereby all subsequent visits after a patient took rescue medication for the disease under study or died due to any cause were considered to be missing.Number of Patients With Combined Perianal Fistula Remission at Week 12 At baseline (day 1) and week 12 (day 85) of treatment. Number of patients with combined perianal fistula remission at Week 12 defined as closure of all external openings, no drainage/discharge despite gentle finger compression of fistulas that were draining at baseline and without new emerging fistulas, AND absence collections of \>2 centimeter, confirmed by magnetic resonance imaging (MRI) in at least 2 of 3 dimensions - blinded and centrally read.
The no response imputation (NRI) approach is applied: missing visits where imputed whereby all subsequent visits after a patient took rescue medication for the disease under study or died due to any cause were considered to be missing.
Trial Locations
- Locations (10)
AKH - Medical University of Vienna
🇦🇹Wien, Austria
UZ Leuven
🇧🇪Leuven, Belgium
Universitätsklinikum Erlangen
🇩🇪Erlangen, Germany
Amsterdam UMC, Locatie AMC
🇳🇱Amsterdam, Netherlands
Universitätsklinikum Ulm
🇩🇪Ulm, Germany
Semmelweis University
🇭🇺Budapest, Hungary
Hospital Universitari de Girona Doctor Josep Trueta
🇪🇸Girona, Spain
Herlev and Gentofte Hospital
🇩🇰Herlev, Denmark
Inje University Haeundae Paik Hospital
🇰🇷Busan, Korea, Republic of
Centre Hospitalier Universitaire de Liège
🇧🇪Liège, Belgium