Crohn Disease
Conditions
Brief summary
The main objectives of this study are to evaluate the long-term safety of spesolimab in patients with perianal fistulising Crohn's disease who have completed treatment in parent trials and to evaluate the long-term efficacy of spesolimab in patients with perianal fistulising Crohn's disease, who have completed treatment in parent trials
Interventions
DRUGSpesolimab
Spesolimab
Sponsors
Boehringer Ingelheim
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patient older than 18 years 2. Has completed all treatments (placebo or active treatment) and the end of treatment (EOT) visit in the parent induction trial in fistulising Crohn's Disease (CD) and is willing and able to continue treatment in 1368-0007 3. Has obtained an individual health benefit, per investigator judgement (such as fistula response or remission or other clinical improvement), from treatment in the parent trial 4. Signed and dated written informed consent for 1368-0007 in accordance with GCP and local legislation prior to admission into the trial 5. Women of childbearing potential (WOCBP) must be ready to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the patient information
Exclusion criteria
1. Have experienced treatment-limiting adverse events during induction treatment with study drug 2. Have developed any condition which meets the
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Exposure Adjusted Rate of Patients Reporting a Treatment Emergent Adverse Event (TEAE) During Maintenance Treatment | First dose of Spesolimab in this trial through to the last dose of spesolimab + 16 weeks, approximately 104 weeks. | Exposure adjusted rate of patients reporting a treatment emergent adverse event (TEAE) during maintenance treatment. The incidence rate was calculated as Incidence rate = 100 \* number of patients with TEAE / Total TEAE-specific time at risk. Where the Time at risk (for patients who experienced a TEAE) was calculated as Time at Risk (in subject years) = ((date of onset of AE - study drug start date) +1 day) / 365.25 and Time at risk (for patients who did not experience a TEAE) Time at Risk (in subject years) = ((date of the end of time at risk - study drug start date) +1 day) / 365.25. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Patients With Perianal Fistula Remission | Baseline, week 48 and 96 of treatment. | Proportion of patients with perianal fistula remission at weeks 48 and 96. Perianal fistula remission was defined as closure of all external openings, i.e. no drainage and discharge despite gentle finger compression, that were open and draining at baseline of the parent trial and closure of all external openings that were newly emerged during the parent trial or this trial. |
| Proportion of Patients With Perianal Fistula Response | Baseline, week 48 and 96 of treatment. | Proportion of patients with perianal fistula response at weeks 48 and 96. Perianal fistula response was defined as closure and no drainage and discharge despite gentle finger compression of at least 50% in number of external openings regardless of the onset time, compared with the number of open and drainage fistulas at baseline of the parent trial. |
Countries
Austria, Belgium, Germany, Netherlands, South Korea
Participant flow
Recruitment details
This was an open-label, single group, long-term extension trial of approximately 89 weeks duration, which investigated the long-term safety and efficacy of spesolimab in patients with perianal fistulas due to Crohn's disease (CD) who had completed treatment in a parent spesolimab trial.
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Spesolimab During the maintenance treatment period 300 milligram (mg) Spesolimab was given by subcutaneous injection at Week 0 and then every 4 weeks for a total duration of 89 weeks.
Patient with a confirmed fistula relapse received a single intravenous infusion of 1200 mg Spesolimab followed by an intensified subcutaneous spesolimab maintenance dosing of 600 mg Spesolimab every 4 weeks. | 12 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Due to early termination of the trial | 10 |
| Overall Study | Protocol Violation | 1 |
Baseline characteristics
| Characteristic | Spesolimab |
|---|---|
| Age, Continuous | 37.1 years STANDARD_DEVIATION 10.2 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 12 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 10 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 12 |
| other Total, other adverse events | 11 / 12 |
| serious Total, serious adverse events | 1 / 12 |
Outcome results
Primary
Exposure Adjusted Rate of Patients Reporting a Treatment Emergent Adverse Event (TEAE) During Maintenance Treatment
Exposure adjusted rate of patients reporting a treatment emergent adverse event (TEAE) during maintenance treatment. The incidence rate was calculated as Incidence rate = 100 \* number of patients with TEAE / Total TEAE-specific time at risk. Where the Time at risk (for patients who experienced a TEAE) was calculated as Time at Risk (in subject years) = ((date of onset of AE - study drug start date) +1 day) / 365.25 and Time at risk (for patients who did not experience a TEAE) Time at Risk (in subject years) = ((date of the end of time at risk - study drug start date) +1 day) / 365.25.
Time frame: First dose of Spesolimab in this trial through to the last dose of spesolimab + 16 weeks, approximately 104 weeks.
Population: The safety set (SAF) included all patients who received at least one dose of trial drug in the overall maintenance period.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Spesolimab | Exposure Adjusted Rate of Patients Reporting a Treatment Emergent Adverse Event (TEAE) During Maintenance Treatment | 816.6 Patients with TEAE per 100 patient years |
Secondary
Proportion of Patients With Perianal Fistula Remission
Proportion of patients with perianal fistula remission at weeks 48 and 96. Perianal fistula remission was defined as closure of all external openings, i.e. no drainage and discharge despite gentle finger compression, that were open and draining at baseline of the parent trial and closure of all external openings that were newly emerged during the parent trial or this trial.
Time frame: Baseline, week 48 and 96 of treatment.
Population: The data was not collected as intended and the remission cannot be reported.
Secondary
Proportion of Patients With Perianal Fistula Response
Proportion of patients with perianal fistula response at weeks 48 and 96. Perianal fistula response was defined as closure and no drainage and discharge despite gentle finger compression of at least 50% in number of external openings regardless of the onset time, compared with the number of open and drainage fistulas at baseline of the parent trial.
Time frame: Baseline, week 48 and 96 of treatment.
Population: The data was not collected as intended and the response cannot be reported.