Trial Outcomes & Findings for An Expanded Access Trial in Japan to Provide Spesolimab to People With a Flare-up in Generalized Pustular Psoriasis Who Have no Other Treatment Options (NCT NCT05200247)
NCT ID: NCT05200247
Last Updated: 2025-02-25
Results Overview
Number of patients with any treatment emergent adverse events (TEAEs) is reported. An adverse events (AE) was defined as any untoward medical occurrence in a patient or clinical investigation subject administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE that started before first drug intake and deteriorated under treatment were also considered as 'treatment-emergent'.
COMPLETED
PHASE3
11 participants
From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
2025-02-25
Participant Flow
This was an open-label, multicentre, single-arm trial designed to provide early access to spesolimab, for patients with generalised pustular psoriasis (GPP) presenting with a flare and for whom no satisfactory authorised alternative therapy existed and who were unable to participate in a clinical trial, as assessed by the investigator.
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 900 mg i.v. SD
Patient with GPP flare received intravenously (i.v.) a single dose (SD) of 900 milligram (mg) of spesolimab on Day 1 of Week 1. A second dose of 900 mg spesolimab was administered one week after the initial infusion if deemed necessary by the investigator.
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|---|---|
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Overall Study
STARTED
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11
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Overall Study
COMPLETED
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11
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
An Expanded Access Trial in Japan to Provide Spesolimab to People With a Flare-up in Generalized Pustular Psoriasis Who Have no Other Treatment Options
Baseline characteristics by cohort
| Measure |
Spesolimab 900 mg i.v. SD
n=11 Participants
Patient with GPP flare received intravenously (i.v.) a single dose (SD) of 900 milligram (mg) of spesolimab on Day 1 of Week 1. A second dose of 900 mg spesolimab was administered one week after the initial infusion if deemed necessary by the investigator.
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|---|---|
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Age, Continuous
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50.5 Years
STANDARD_DEVIATION 12.3 • n=5 Participants
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Sex: Female, Male
Female
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6 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
|
11 Participants
n=5 Participants
|
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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
|
11 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.Population: Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
Number of patients with any treatment emergent adverse events (TEAEs) is reported. An adverse events (AE) was defined as any untoward medical occurrence in a patient or clinical investigation subject administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE that started before first drug intake and deteriorated under treatment were also considered as 'treatment-emergent'.
Outcome measures
| Measure |
Spesolimab 900 mg i.v. SD
n=11 Participants
Patient with GPP flare received intravenously (i.v.) a single dose (SD) of 900 milligram (mg) of spesolimab on Day 1 of Week 1. A second dose of 900 mg spesolimab was administered one week after the initial infusion if deemed necessary by the investigator.
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|---|---|
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Occurrence of Treatment Emergent Adverse Events (TEAEs)
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7 Participants
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SECONDARY outcome
Timeframe: From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.Population: Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
Number of patients with treatment emergent serious adverse events (SAEs) is reported. A serious adverse event (SAE) was defined as any AE which fulfils at least one of the following criteria: * results in death, * is life-threatening, which refers to an event in which the patient was at risk of death at the time of the event; * requires inpatient hospitalisation or prolongation of existing hospitalisation, * results in persistent or significant disability or incapacity, * is a congenital anomaly / birth defect, * is deemed serious for any other reason if it is an important medical event when based on appropriate medical judgement which may jeopardise the patient and may require medical or surgical intervention to prevent one of the other outcomes listed in the above definitions. * An event that possibly leads to disability will be handled as 'deemed serious for any other reason' and, therefore, reported as an SAE.
Outcome measures
| Measure |
Spesolimab 900 mg i.v. SD
n=11 Participants
Patient with GPP flare received intravenously (i.v.) a single dose (SD) of 900 milligram (mg) of spesolimab on Day 1 of Week 1. A second dose of 900 mg spesolimab was administered one week after the initial infusion if deemed necessary by the investigator.
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|---|---|
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Occurrence of Treatment Emergent Serious Adverse Events (SAEs)
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0 Participants
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SECONDARY outcome
Timeframe: From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.Population: Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
Number of patients with treatment emergent adverse events of special interest (AESIs) is reported. The term adverse events of special interest (AESI) relates to any specific adverse event (AE) that has been identified at the project level as being of particular concern for prospective safety monitoring and safety assessment within this trial, e.g. the potential for AEs based on knowledge from other compounds in the same class. The following are considered as AESIs: * Potential Severe Drug Induced Liver Injury (DILI) * Systemic hypersensitivity reactions including infusion reactions and anaphylactic reaction * Severe infections (according to RCTC grading in the ISF) * Opportunistic and mycobacterium tuberculosis infections * Peripheral neuropathy.
Outcome measures
| Measure |
Spesolimab 900 mg i.v. SD
n=11 Participants
Patient with GPP flare received intravenously (i.v.) a single dose (SD) of 900 milligram (mg) of spesolimab on Day 1 of Week 1. A second dose of 900 mg spesolimab was administered one week after the initial infusion if deemed necessary by the investigator.
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|---|---|
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Occurrence of Treatment Emergent Adverse Events of Special Interest (AESIs)
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0 Participants
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Adverse Events
Spesolimab 900 mg i.v. SD
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Spesolimab 900 mg i.v. SD
n=11 participants at risk
Patient with GPP flare received intravenously (i.v.) a single dose (SD) of 900 milligram (mg) of spesolimab on Day 1 of Week 1. A second dose of 900 mg spesolimab was administered one week after the initial infusion if deemed necessary by the investigator.
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|---|---|
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Gastrointestinal disorders
Constipation
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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General disorders
Chest pain
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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General disorders
Face oedema
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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General disorders
Malaise
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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Infections and infestations
Periodontitis
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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Injury, poisoning and procedural complications
Wound
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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Investigations
Coronavirus test positive
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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Investigations
Transaminases increased
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
|
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Nervous system disorders
Headache
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
|
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Renal and urinary disorders
Pollakiuria
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
|
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Skin and subcutaneous tissue disorders
Acne
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9.1%
1/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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Skin and subcutaneous tissue disorders
Pustular psoriasis
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18.2%
2/11 • From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Treated Set (TS): This patient set includes all patients who received at least one dose of study drug.
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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