Clinical Trial Results:
Multi-center, double-blind, randomized, placebo-controlled, phase IIa trial to evaluate spesolimab (BI 655130) efficacy in patients with fibrostenotic Crohn’s Disease
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Summary
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EudraCT number |
2020-005770-99 |
Trial protocol |
BE IE NL DK SE NO PT IT FR |
Global end of trial date |
31 May 2022
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Results information
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Results version number |
v1(current) |
This version publication date |
15 Jun 2023
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First version publication date |
15 Jun 2023
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
1368-0059
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05013385 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Boehringer Ingelheim
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Sponsor organisation address |
Binger Strasse 173, Ingelheim am Rhein, Germany, 55216
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Public contact |
Boehringer Ingelheim, Call Center, Boehringer Ingelheim, 001 18002430127, clintriage.rdg@boehringer-ingelheim.com
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Scientific contact |
Boehringer Ingelheim, Call Center, Boehringer Ingelheim, 001 18002430127, clintriage.rdg@boehringer-ingelheim.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
26 Jul 2022
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
31 May 2022
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Global end of trial reached? |
Yes
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Global end of trial date |
31 May 2022
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
The main objective of this trial was to demonstrate that spesolimab is effective in maintaining Symptomatic Stenosis Response and / or inducing Radiographic Stenosis Response in patients with symptomatic Crohn’s Disease (CD)-related small bowel stenosis, who have achieved Symptomatic Stenosis Response after standard medical therapy.
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Protection of trial subjects |
Only subjects that met all the study inclusion and none of the exclusion criteria were to be entered in the study. All subjects were free to withdraw from the clinical trial at any time for any reason given. Close monitoring of all subjects was adhered to throughout the trial conduct. Rescue medication was allowed for all subjects as required.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
12 Apr 2022
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Canada: 2
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Country: Number of subjects enrolled |
Japan: 1
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Country: Number of subjects enrolled |
Sweden: 1
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Country: Number of subjects enrolled |
United States: 1
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Worldwide total number of subjects |
5
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EEA total number of subjects |
1
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
5
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
The trial was designed to have a Lead-in Period where background medication is optimized, a Randomized Blinded Treatment Period where patients would have been randomized to spesolimab or placebo and a Follow-up Period. The trial was terminated when 5 patients had entered the Lead-in period. No patient was randomized to spesolimab or placebo. | ||||||||||||
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Pre-assignment
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Screening details |
All subjects who entered the Lead-in period 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. | ||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | ||||||||||||
Roles blinded |
Subject, Investigator, Monitor, Carer, Data analyst, Assessor | ||||||||||||
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Arms
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Arm title
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Enrolled patients | ||||||||||||
Arm description |
Fibrostenotic Crohn’s Disease patients who were eligible to enter the Lead-In period of the trial were planned to receive during the Lead-In Period standard medical treatment which included 2 weeks of corticosteroids to be tapered according to a standardized 8 week regimen and individual optimization of anti-inflammatory biological treatment. After 8 weeks of optimized biological anti-inflammatory therapy would have been completed, only patients who would have achieved a Symptomatic Stenosis Response and an absent or mild-to-moderate colonic endoscopic activity (colonic Simple Endoscopic Score in Crohn’s Disease (SES-CD) ≤12) would have been eligible for randomization into the Blinded Treatment Period. | ||||||||||||
Arm type |
Optimization of background medication | ||||||||||||
Investigational medicinal product name |
Standard of care corticosteroids during the Lead-In period
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Enrolled patients were administered 2 weeks of short-term corticosteroids with subsequent tapering at the beginning of the Lead-in period, i.e. when the patient was enrolled in the acute episode of the disease. No one of the enrolled patients, reached the point of 8-weeks optimized biological treatment during the Lead-In period.
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Baseline characteristics reporting groups
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Reporting group title |
Enrolled patients
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Reporting group description |
Fibrostenotic Crohn’s Disease patients who were eligible to enter the Lead-In period of the trial were planned to receive during the Lead-In Period standard medical treatment which included 2 weeks of corticosteroids to be tapered according to a standardized 8 week regimen and individual optimization of anti-inflammatory biological treatment. After 8 weeks of optimized biological anti-inflammatory therapy would have been completed, only patients who would have achieved a Symptomatic Stenosis Response and an absent or mild-to-moderate colonic endoscopic activity (colonic Simple Endoscopic Score in Crohn’s Disease (SES-CD) ≤12) would have been eligible for randomization into the Blinded Treatment Period. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Enrolled patients
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Reporting group description |
Fibrostenotic Crohn’s Disease patients who were eligible to enter the Lead-In period of the trial were planned to receive during the Lead-In Period standard medical treatment which included 2 weeks of corticosteroids to be tapered according to a standardized 8 week regimen and individual optimization of anti-inflammatory biological treatment. After 8 weeks of optimized biological anti-inflammatory therapy would have been completed, only patients who would have achieved a Symptomatic Stenosis Response and an absent or mild-to-moderate colonic endoscopic activity (colonic Simple Endoscopic Score in Crohn’s Disease (SES-CD) ≤12) would have been eligible for randomization into the Blinded Treatment Period. | ||
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End point title |
Proportion of patients with maintained Symptomatic Stenosis Response at Week 48 [1] | ||||||||
End point description |
Study was terminated early. 5 patients entered the Lead-In Period but no patient entered the Randomized Blinded Treatment Period and received the investigational medical products (spesolimab or placebo). Outcome Measures were planned to be reported for the Randomized Blinded Treatment Period.
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End point type |
Primary
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End point timeframe |
At Week 48.
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No data could be collected for the primary end point to perform the statistical analyses. |
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| Notes [2] - No patient entered the Randomized Blinded Treatment Period, therefore no data could be collected. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Proportion of patients with Radiographic Stenosis Response at Week 48 [3] | ||||||||
End point description |
Study was terminated early. 5 patients entered the Lead-In Period but no patient entered the Randomized Blinded Treatment Period and received the investigational medical products (spesolimab or placebo). Outcome Measures were planned to be reported for the Randomized Blinded Treatment Period.
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End point type |
Primary
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End point timeframe |
At week 48.
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| Notes [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No data could be collected for the primary end point to perform the statistical analyses. |
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| Notes [4] - No patient entered the Randomized Blinded Treatment Period, therefore no data could be collected. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Proportion of patients with Radiographic Stenosis Response at Week 24 | ||||||||
End point description |
Study was terminated early. 5 patients entered the Lead-In Period but no patient entered the Randomized Blinded Treatment Period and received the investigational medical products (spesolimab or placebo). Outcome Measures were planned to be reported for the Randomized Blinded Treatment Period.
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End point type |
Secondary
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End point timeframe |
timeframe.
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| Notes [5] - No patient entered the Randomized Blinded Treatment Period, therefore no data could be collected. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Proportion of patients with maintained Symptomatic Stenosis Response at Week 24 | ||||||||
End point description |
Study was terminated early. 5 patients entered the Lead-In Period but no patient entered the Randomized Blinded Treatment Period and received the investigational medical products (spesolimab or placebo). Outcome Measures were planned to be reported for the Randomized Blinded Treatment Period.
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End point type |
Secondary
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End point timeframe |
At Week 24.
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| Notes [6] - No patient entered the Randomized Blinded Treatment Period, therefore no data could be collected. |
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| No statistical analyses for this end point | |||||||||
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Adverse events information [1]
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Timeframe for reporting adverse events |
Not applicable.
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Adverse event reporting additional description |
Adverse events data were planned to be reported for the Randomized Blinded Treatment Period. Since no patient was randomised to receive the investigational medical products (spesolimab or placebo), adverse events reporting is not applicable for this study.
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Assessment type |
Systematic | ||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
0
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| Frequency threshold for reporting non-serious adverse events: 0% | |||
| Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: Non Serious Adverse Events were planned to be reported for the Randomized Blinded Treatment Period. Since no subject entered the Randomized Blinded Treatment Period, reporting of Non-Serious Adverse Events is not applicable for this study. |
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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07 Jun 2021 |
Information was added for clarification in the inclusion and exclusion criteria, rephrasing of wording. |
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20 Oct 2021 |
Addition of permanent trial treatment discontinuation if patient experiences a moderate or severe opportunistic infection, or if a patient experiences any infection that meets serious adverse events (SAE) reporting criteria. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| Study was terminated early due to sponsor's decision. Outcome measure data could not be collected. | |||
