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CompletedNCT04399837Updated Oct 20, 2025Results posted

A Study to Test Whether BI 655130 (Spesolimab) Prevents Flare-ups in Patients With Generalized Pustular Psoriasis

A Phase 2 interventional study of Spesolimab and Placebo in Generalized Pustular Psoriasis, sponsored by Boehringer Ingelheim. Completed at 72 sites in 23 countries. Open to participants aged 12 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-10-20.

Sponsored by Boehringer Ingelheim · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
123
Allocation
Randomized
Ages
12 Years to 75 Years
Sex
All
01

Study summary

This is a study in adolescents and adults with Generalized Pustular Psoriasis (GPP). People between 12 and 75 years old can take part in the study. The study is open to people who had GPP flare-ups in the past but whose skin is clear or almost clear when they join the study. The purpose of the study is to test 3 different doses of a medicine called spesolimab and to see whether it helps to prevent GPP flare-ups.

Participants are put into 4 groups by chance. Three groups get different doses of spesolimab. The fourth group gets a placebo. Placebo looks like spesolimab but does not contain any medicine.

Spesolimab and placebo are given as an injection under the skin. Participants are in the study for about 1 year and 4 months. During this time, they visit the study site about 15 times. For the first 11 months, participants get spesolimab or placebo injections every month. At the study visits, the doctors check participants' skin for signs of a new GPP flare-up. The doctors also check the general health of the participants.

If a participant has a GPP flare-up during the study, more visits may be necessary. In case of a flare-up, participants get a dose of spesolimab as an infusion into a vein.

02

Conditions studied

  • Generalized Pustular Psoriasis

Browse trials for

03

In context

Psoriasis

1,899 studies on the registry are indexed under Psoriasis; 233 are open to participants now.

This study's enrollment of 123 is above the median of 70 across 1,447 interventional studies indexed under Psoriasis.

Browse Psoriasis studies →

Lead sponsor

Boehringer Ingelheim is the lead sponsor of 2,245 studies on the registry; 58 are open to participants now.

Of its 162 completed or terminated interventional studies of FDA-regulated products, 116 (72%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Patients with a known and documented history of GPP per ERASPEN criteria (see Section 3.3.1) regardless of IL36RN mutation status, with at least 2 presentations of moderate to severe GPP flares with fresh pustulation (new appearance or worsening) in the past.
  • Patients with a GPPGA score of 0 or 1 at screening and randomization.
  • Patients who are not on concomitant GPP treatment at time of randomization (V2) must have had at least two presentations of moderate to severe GPP flare in the past year, at least one of which had evidence of either fever and/or elevated CRP and/or elevated WBC, and/or asthenia and/or myalgia.
  • Patients who are not on concomitant GPP treatment at time of randomization (V2) but who were on concomitant GPP treatment until shortly before randomization (V2) (≤ 12 weeks before randomization), these patients must have a history of flaring while on concomitant treatment for GPP or in case of dose reduction or discontinuation of their concomitant medication.
  • Patients who are on concomitant treatment regimen with retinoids and/or methotrexate and/or cyclosporine must stop at the day of randomization (V2). These patients must have a history of flaring while on concomitant treatment for GPP or in case of dose reduction or discontinuation of these concomitant medications.
  • Male or female patients, aged 12 to 75 years at screening. For all patients, a minimum weight of 40 kg is required.
  • Signed and dated written informed consent and assent in accordance with ICH-GCP and local legislation prior to admission in the trial.
  • Women of childbearing potential (WOCBP)1 must be ready and able 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 CTP as well as in the patient, parent(s) (or patient's legal guardian) information.

Exclusion criteria

Exclusion Criteria:

  1. Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome.
  2. Patients with primary erythrodermic psoriasis vulgaris.
  3. Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Limit of Normal (ULN) elevation in Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin.

  4. Treatment with:

    1. Any restricted medication as specified in the CTP, or any drug considered likely to interfere with the safe conduct of the study, as assessed by the investigator.
    2. Any prior exposure to BI 655130 or another IL36R inhibitor biologic.
  5. Increased risk of infectious complications (e.g. recent pyogenic infection, any congenital or acquired immunodeficiency (e.g. HIV), past organ or stem cell transplantation), as assessed by the investigator.
  6. Relevant chronic or acute infections including active tuberculosis, human immunodeficiency virus (HIV) infection or viral hepatitis at the time of randomization. A patient can be re-screened if the patient was treated and is cured from the acute infection.

  7. Active or Latent Tuberculosis (TB):

    • Patients with active tuberculosis should be excluded
    • Patients with a positive QuantiFERON® (or if applicable, T-Spot®) TB test during screening are excluded, unless the patient had previous diagnosis of active or latent TB and has completed appropriate treatment per the discretion of the local investigator within the last 3 years and at the latest at the time of screening (i.e. 2 to 4 weeks before study drug administration); patients may be re-screened once to meet this criterion)
    • Patients with suspected false positive or indeterminate QuantiFERON® (or if applicable, T-Spot®) TB result may be re-tested once
    • If QuantiFERON® (or if applicable, T-Spot®) TB testing is not available or provides indeterminate results after repeat testing, a tuberculin skin test (TST) can be performed: A TST reaction of ≥10mm (≥5mm if receiving ≥15mg/d prednisone or its equivalent) is considered positive.
  8. History of allergy/hypersensitivity to the systemically administered trial medication agent or its excipients.

Further exclusion criteria apply.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
123 participants (actual)

Study arms

  • Experimental
    Spesolimab SC low dose

    Drug: Spesolimab

  • Experimental
    Spesolimab SC medium dose

    Drug: Spesolimab

  • Experimental
    Spesolimab SC high dose

    Drug: Spesolimab

  • Placebo comparator
    Placebo

    Drug: Placebo

Interventions

  • DrugSpesolimab

    Solution for injection

  • DrugPlacebo

    Solution for injection

06

What researchers measure

Primary outcomes

  1. Time to First Generalized Pustular Psoriasis (GPP) Flare

    A GPP flare was defined as increase in Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score by ≥ 2 from baseline and the pustular component of GPPGA ≥ 2) up to week 48. Use of rescue medication, or investigator-prescribed Standard of Care (SoC) for GPP worsening, was considered to represent a GPP flare onset. GPPGA relied on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The GPPGA total score was calculated by taking the mean of the erythema subscore, pustules subscore and scaling/crusting subscore. The severity of each subscore was assessed using a 5 point scale score ranging from 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score is assigned as follows: * 0 , if scores for all three subscores are 0, * 1, if 0 \< mean \< 1.5, * 2, if 1.5 ≤ mean \< 2.5, * 3, if 2.5 ≤ mean \< 3.5, * 4, if mean ≥ 3.5.

    Time frame: GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.

Secondary outcomes

  1. Key Secondary Endpoint: The Occurrence of at Least One Generalized Pustular Psoriasis (GPP) Flare up to Week 48

    Proportion of patients with at least one GPP flare up to Week 48 is reported. Proportions were rounded up to three decimal places. A GPP flare was defined as increase in Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score by ≥ 2 from baseline and the pustular component of GPPGA ≥ 2. Any use of rescue medication, or investigator-prescribed SoC for GPP worsening, prior to week 48 was considered to represent the onset of a GPP flare. Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) relied on the clinical assessment of GPP patient's skin presentation. The total score is calculated by taking the mean of the three subscores: 1) erythema; 2) pustules and 3) scaling/crusting which were assessed using a scale score 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score: 0 , if scores for all three subscores are 0, 1. if 0 \< mean \< 1.5; 2. if 1.5 ≤ mean \< 2.5; 3. if 2.5 ≤ mean \< 3.5; 4. if mean ≥ 3.5.

    Time frame: GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.

  2. Time to First Worsening of Psoriasis Symptom Scale (PSS) up to Week 48

    Worsening of Psoriasis Symptom Scale (PSS) was defined as a 4-point increase in total score from baseline. Intake of rescue medication, or investigator-prescribed SoC for GPP worsening, was considered as onset of a worsening. The PSS is a 4-item patient-reported outcome (PRO) instrument that was developed to assess the severity of 4 psoriasis symptoms in patients with moderate to severe psoriasis. The symptoms included are: pain, redness, itching, and burning. Current symptom severity is assessed using a 5-point scale ranging from 0 (none) to 4 (very severe). The symptom scores are added to an unweighted total score (range: 0 (no symptoms) to 16 (severe symptoms)).

    Time frame: PSS assessments were performed at: Baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Visit window was ±7 days.

  3. Time to First Worsening of Dermatology Quality of Life Index (DLQI) up to Week 48

    Worsening of DLQI up to week 48 was defined as a 4-point increase in total score from baseline. Intake of rescue medication, or investigator-prescribed SoC for GPP worsening, was considered as onset of a worsening. The DLQI is a patient-administered, ten-question, quality of life questionnaire that covers six domains including symptoms and feelings, daily activities, leisure, work and school, personal relationships and treatment. Response categories include "not relevant" (score of 0), "not at all" (score of 0), "a little" (score of 1), "a lot" (score of 2) and "very much" (score of 3). Question 7 is a "yes"/ "no" question where "yes" is scored as 3. DLQI total score is calculated by summing the scores of each question resulting in a range of 0 (no effect on patient's life) to 30 (extremely large effect on patient's life).

    Time frame: DLQI assessments were performed at: Baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 24, 36 and 48). Visit window was ±7 days. Time window for Week 48 was from Week 46 to Week 50.

  4. Sustained Remission

    Proportion of patients with sustained remission at all visits up to Week 48. Proportions were rounded up to three decimal places. Remission was defined as a patient with a GPPGA score of 0 or 1 (clear or almost clear) at all visits up to week 48, without intake of rescue medication, or investigator-prescribed SoC for GPP worsening. GPPGA relied on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The GPPGA total score was calculated by taking the mean of the erythema subscore, pustules subscore and scaling/crusting subscore. The severity of each subscore was assessed using a 5 point scale score ranging from 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score is assigned as follows: * 0 , if scores for all three subscores are 0, * 1, if 0 \< mean \< 1.5, * 2, if 1.5 ≤ mean \< 2.5, * 3, if 2.5 ≤ mean \< 3.5, * 4, if mean ≥ 3.5.

    Time frame: GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.

  5. The Occurrence of Treatment Emergent Adverse Events (TEAEs)

    Percentage of patients with treatment emergent adverse events (TEAEs) is reported. Percentages were rounded up to one decimal places. Time Frame: Placebo, Spesolimab (Speso) SC low, medium, high: From randomized study treatment start until the first use of rescue medication with IV spesolimab or until last dose + 16 weeks, up to 62 weeks. Speso IV SD and Speso IV DD: From first use of rescue medication with IV spesolimab until OL maintenance spesolimab SC or until last dose of spesolimab IV + 16 weeks, up to 17 weeks. Speso OL SC: From the first dose of OL spesolimab SC treatment until last dose + 16 weeks, up to 62 weeks.

    Time frame: Up to 62 weeks (for detailed timeframe see description).

07

Results

Posted Dec 14, 2023

Participant flow

This was a randomized multicenter, parallel group, double-blind, placebo-controlled Phase IIb trial comprising of 3 active doses compared to placebo in adolescents from 12 years to less than 18 years of age and adult patients with history of Generalized Pustular Psoriasis (GPP) and presenting (at screening and at randomization) with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 (clear or almost clear).

Participant flow — Overall Study
MilestonePlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High Dose
Started31313130
Completed30272826
Not completed1434
Withdrew: Other than listed0223
Withdrew: Withdrawal by subject1211

Outcome measures

PrimaryTime to First Generalized Pustular Psoriasis (GPP) Flare

A GPP flare was defined as increase in Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score by ≥ 2 from baseline and the pustular component of GPPGA ≥ 2) up to week 48. Use of rescue medication, or investigator-prescribed Standard of Care (SoC) for GPP worsening, was considered to represent a GPP flare onset. GPPGA relied on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The GPPGA total score was calculated by taking the mean of the erythema subscore, pustules subscore and scaling/crusting subscore. The severity of each subscore was assessed using a 5 point scale score ranging from 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score is assigned as follows: * 0 , if scores for all three subscores are 0, * 1, if 0 \< mean \< 1.5, * 2, if 1.5 ≤ mean \< 2.5, * 3, if 2.5 ≤ mean \< 3.5, * 4, if mean ≥ 3.5.

Time frame:
GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.
Reported as:
Median · weeks
Time to First Generalized Pustular Psoriasis (GPP) Flare
weeksPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High Dose
Time to First Generalized Pustular Psoriasis (GPP) Flare37.3 (4.0 to NA)NA (NA to NA)NA (NA to NA)NA (NA to NA)
Statistical analysis
  • Placebo vs Spesolimab SC Low Dose vs Spesolimab SC Medium Dose vs Spesolimab SC High Dose · MCP-Mod linear model fit · p = 0.002 · Multiple contrast test: 3.041Model assumption: Dose effect is linear with the increase of dose.
  • Placebo vs Spesolimab SC Low Dose vs Spesolimab SC Medium Dose vs Spesolimab SC High Dose · MCP-Mod Emax2 model fit · p = 0.002 · Multiple contrast test: 3.033Model assumption: 95% of the maximum effect is achieved at low dose.
  • Placebo vs Spesolimab SC Low Dose vs Spesolimab SC Medium Dose vs Spesolimab SC High Dose · MCP-Mod Emax1 model fit · p = 0.002 · Multiple contrast test: 3.088Model assumption: 70% of the maximum effect is achieved at low dose.
  • Placebo vs Spesolimab SC Low Dose vs Spesolimab SC Medium Dose vs Spesolimab SC High Dose · MCP-Mod exponential model fit · p = 0.003 · Multiple contrast test: 2.977Model assumption: 35% of the maximum effect is achieved at medium dose.
  • Placebo vs Spesolimab SC Medium Dose · Log Rank · p = 0.0269 (One-sided p-value was computed from the log-rank test stratified by use of systemic GPP medication at randomisation. Threshold for statistical significance: one-sided p-value ≤ 0.01875.) · Hazard ratio (hr): 0.468 · 95% CI 0.206 to 1.064Hazard ratio and its 95% Confidence Interval are from Cox regression model stratified by use of systemic GPP medication at randomisation.
  • Placebo vs Spesolimab SC High Dose · Log Rank · p = 0.0005 (One-sided p-value is computed from the log-rank test stratified by use of systemic GPP medication at randomisation. Threshold for statistical significance: One-sided p-value ≤0.0125.) · Hazard ratio (hr): 0.157 · 95% CI 0.046 to 0.541Hazard ratio and its 95% CI (confidence interval) are from Cox regression model stratified by use of systemic GPP medication at randomisation.
SecondaryKey Secondary Endpoint: The Occurrence of at Least One Generalized Pustular Psoriasis (GPP) Flare up to Week 48

Proportion of patients with at least one GPP flare up to Week 48 is reported. Proportions were rounded up to three decimal places. A GPP flare was defined as increase in Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score by ≥ 2 from baseline and the pustular component of GPPGA ≥ 2. Any use of rescue medication, or investigator-prescribed SoC for GPP worsening, prior to week 48 was considered to represent the onset of a GPP flare. Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) relied on the clinical assessment of GPP patient's skin presentation. The total score is calculated by taking the mean of the three subscores: 1) erythema; 2) pustules and 3) scaling/crusting which were assessed using a scale score 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score: 0 , if scores for all three subscores are 0, 1. if 0 \< mean \< 1.5; 2. if 1.5 ≤ mean \< 2.5; 3. if 2.5 ≤ mean \< 3.5; 4. if mean ≥ 3.5.

Time frame:
GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.
Reported as:
Number · proportion of patients
Key Secondary Endpoint: The Occurrence of at Least One Generalized Pustular Psoriasis (GPP) Flare up to Week 48
proportion of patientsPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High Dose
Key Secondary Endpoint: The Occurrence of at Least One Generalized Pustular Psoriasis (GPP) Flare up to Week 480.516 (0.348 to 0.680)0.226 (0.114 to 0.398)0.297 (0.181 to 0.445)0.127 (0.050 to 0.289)
Statistical analysis
  • Placebo vs Spesolimab SC High Dose · Cochran-Mantel-Haenszel · p = 0.0013 (One-sided p-value was computed from the Cochran-Mantel-Haenszel test stratified by use of systemic GPP medication at randomisation. one-sided alpha= 0.00625) · Risk difference (rd): -0.390 · 95% CI -0.621 to -0.159Risk difference=Spesolimab high dose-Placebo.
SecondaryTime to First Worsening of Psoriasis Symptom Scale (PSS) up to Week 48

Worsening of Psoriasis Symptom Scale (PSS) was defined as a 4-point increase in total score from baseline. Intake of rescue medication, or investigator-prescribed SoC for GPP worsening, was considered as onset of a worsening. The PSS is a 4-item patient-reported outcome (PRO) instrument that was developed to assess the severity of 4 psoriasis symptoms in patients with moderate to severe psoriasis. The symptoms included are: pain, redness, itching, and burning. Current symptom severity is assessed using a 5-point scale ranging from 0 (none) to 4 (very severe). The symptom scores are added to an unweighted total score (range: 0 (no symptoms) to 16 (severe symptoms)).

Time frame:
PSS assessments were performed at: Baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Visit window was ±7 days.
Reported as:
Median · weeks
Time to First Worsening of Psoriasis Symptom Scale (PSS) up to Week 48
weeksPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High Dose
Time to First Worsening of Psoriasis Symptom Scale (PSS) up to Week 4816.0 (4.0 to NA)NA (8.1 to NA)NA (8.7 to NA)NA (12.0 to NA)
Statistical analysis
  • Placebo vs Spesolimab SC High Dose · Log Rank · p = 0.0134 (One-sided p-value was computed from the log-rank test stratified by use of systemic GPP medication at randomisation. one-sided alpha= 0.00625) · Hazard ratio (hr): 0.424 · 95% CI 0.197 to 0.914spesolimab high dose vs. Placebo
SecondaryTime to First Worsening of Dermatology Quality of Life Index (DLQI) up to Week 48

Worsening of DLQI up to week 48 was defined as a 4-point increase in total score from baseline. Intake of rescue medication, or investigator-prescribed SoC for GPP worsening, was considered as onset of a worsening. The DLQI is a patient-administered, ten-question, quality of life questionnaire that covers six domains including symptoms and feelings, daily activities, leisure, work and school, personal relationships and treatment. Response categories include "not relevant" (score of 0), "not at all" (score of 0), "a little" (score of 1), "a lot" (score of 2) and "very much" (score of 3). Question 7 is a "yes"/ "no" question where "yes" is scored as 3. DLQI total score is calculated by summing the scores of each question resulting in a range of 0 (no effect on patient's life) to 30 (extremely large effect on patient's life).

Time frame:
DLQI assessments were performed at: Baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 24, 36 and 48). Visit window was ±7 days. Time window for Week 48 was from Week 46 to Week 50.
Reported as:
Median · weeks
Time to First Worsening of Dermatology Quality of Life Index (DLQI) up to Week 48
weeksPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High Dose
Time to First Worsening of Dermatology Quality of Life Index (DLQI) up to Week 4816.0 (4.0 to NA)35.8 (8.1 to NA)49.3 (8.7 to 49.3)NA (NA to NA)
Statistical analysis
  • Placebo vs Spesolimab SC High Dose · Log Rank · p = 0.0010 (One sided p-value was computed from the log-rank test stratified by use of systemic GPP medication at randomisation.) · Hazard ratio (hr): 0.259 · 95% CI 0.109 to 0.620spesolimab high dose vs. Placebo
SecondarySustained Remission

Proportion of patients with sustained remission at all visits up to Week 48. Proportions were rounded up to three decimal places. Remission was defined as a patient with a GPPGA score of 0 or 1 (clear or almost clear) at all visits up to week 48, without intake of rescue medication, or investigator-prescribed SoC for GPP worsening. GPPGA relied on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The GPPGA total score was calculated by taking the mean of the erythema subscore, pustules subscore and scaling/crusting subscore. The severity of each subscore was assessed using a 5 point scale score ranging from 0 to 4 (0=clear, 1=almost clear, 2=mild, 3=moderate, 4=severe). The final GPPGA score is assigned as follows: * 0 , if scores for all three subscores are 0, * 1, if 0 \< mean \< 1.5, * 2, if 1.5 ≤ mean \< 2.5, * 3, if 2.5 ≤ mean \< 3.5, * 4, if mean ≥ 3.5.

Time frame:
GPPGA was regularly assessed at baseline (Week 1) and up to Week 48 (at Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48). Patients could come to site for flare confirmation anytime as unscheduled visit. Visit window was ±7 days.
Reported as:
Number · proportion of patients
Sustained Remission
proportion of patientsPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High Dose
Sustained Remission0.290 (0.161 to 0.466)0.516 (0.348 to 0.680)0.452 (0.292 to 0.622)0.633 (0.471 to 0.770)
SecondaryThe Occurrence of Treatment Emergent Adverse Events (TEAEs)

Percentage of patients with treatment emergent adverse events (TEAEs) is reported. Percentages were rounded up to one decimal places. Time Frame: Placebo, Spesolimab (Speso) SC low, medium, high: From randomized study treatment start until the first use of rescue medication with IV spesolimab or until last dose + 16 weeks, up to 62 weeks. Speso IV SD and Speso IV DD: From first use of rescue medication with IV spesolimab until OL maintenance spesolimab SC or until last dose of spesolimab IV + 16 weeks, up to 17 weeks. Speso OL SC: From the first dose of OL spesolimab SC treatment until last dose + 16 weeks, up to 62 weeks.

Time frame:
Up to 62 weeks (for detailed timeframe see description).
Reported as:
Number · percentage of patients
The Occurrence of Treatment Emergent Adverse Events (TEAEs)
percentage of patientsPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseSpesolimab IV SDSpesolimab IV DDSpesolimab OL SC
The Occurrence of Treatment Emergent Adverse Events (TEAEs)86.790.693.586.768.260.075.0

Adverse events

Collected over Placebo, Spesolimab (Speso) SC low, medium, high: From randomized study treatment start until the first use of rescue medication with IV spesolimab or until last dose + 16 weeks, up to 62 weeks. Speso IV SD and Speso IV DD: From first use of rescue medication with IV spesolimab until OL maintenance spesolimab SC or until last IV dose + 16 weeks, up to 17 weeks. Speso OL SC: From the first dose of OL spesolimab SC treatment until last dose + 16 weeks, up to 62 weeks.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo0/30 (0%)1/30 (3.3%)24/30 (80%)
Spesolimab SC Low Dose0/32 (0%)5/32 (15.6%)25/32 (78.1%)
Spesolimab SC Medium Dose0/31 (0%)1/31 (3.2%)23/31 (74.2%)
Spesolimab SC High Dose0/30 (0%)3/30 (10%)20/30 (66.7%)
Spesolimab IV SD0/22 (0%)1/22 (4.5%)12/22 (54.5%)
Spesolimab IV DD0/10 (0%)4/10 (40%)3/10 (30%)
Spesolimab OL SC0/20 (0%)1/20 (5%)14/20 (70%)
Most frequent serious events
Showing 10 of 17
Most frequent serious events
EventPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseSpesolimab IV SDSpesolimab IV DDSpesolimab OL SC
OedemaGeneral disorders0/300/320/310/300/221/100/20
CellulitisInfections and infestations0/300/320/310/300/221/100/20
PneumoniaInfections and infestations0/301/320/310/300/221/100/20
Septic shockInfections and infestations0/300/320/310/300/221/100/20
Cerebral ischaemiaNervous system disorders0/300/320/310/300/221/100/20
Pustular psoriasisSkin and subcutaneous tissue disorders0/301/321/311/300/221/100/20
Basal cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/300/320/310/300/220/101/20
Urinary tract infectionInfections and infestations0/300/320/310/301/220/100/20
CholelithiasisHepatobiliary disorders0/300/320/311/300/220/100/20
Breast cancerNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/300/320/311/300/220/100/20
Most frequent other events
Showing 10 of 36
Most frequent other events
EventPlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseSpesolimab IV SDSpesolimab IV DDSpesolimab OL SC
Pustular psoriasisSkin and subcutaneous tissue disorders16/309/329/312/305/220/104/20
Upper respiratory tract infectionInfections and infestations4/303/326/310/301/221/104/20
Injection site erythemaGeneral disorders1/304/324/315/300/220/102/20
PsoriasisSkin and subcutaneous tissue disorders3/304/325/314/301/221/100/20
Urinary tract infectionInfections and infestations0/301/320/314/302/221/103/20
Blood creatine phosphokinase increasedInvestigations2/304/321/310/300/220/100/20
ArthralgiaMusculoskeletal and connective tissue disorders1/304/321/313/302/220/100/20
ConstipationGastrointestinal disorders0/300/320/310/300/221/100/20
OdynophagiaGastrointestinal disorders0/300/320/310/300/221/100/20
AstheniaGeneral disorders0/300/320/310/300/221/101/20

Baseline characteristics

Randomized Set: This patient set includes all randomized patients.

Age, Continuous
Age, Continuous(Years)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
Mean39.5 ± 14.038.9 ± 16.542.9 ± 16.740.2 ± 16.440.4 ± 15.8
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
Female1820201876
Male1311111247
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
Hispanic or Latino33017
Not Hispanic or Latino28283129116
Unknown or Not Reported00000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
American Indian or Alaska Native00000
Asian1720212179
Native Hawaiian or Other Pacific Islander00000
Black or African American00000
White141110944
More than one race00000
Unknown or Not Reported00000
Number of patients in the categories 0 or 1 of GPPGA score
Number of patients in the categories 0 or 1 of GPPGA score(Participants)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
0428317
127292327106
Number of patients in the categories 0 or 1 of GPPGA pustules subscore
Number of patients in the categories 0 or 1 of GPPGA pustules subscore(Participants)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
02123242088
110871035
Concomitant use of systemic GPP medication at randomization
Concomitant use of systemic GPP medication at randomization(Participants)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
No968831
Yes2225232292
Psoriasis Symptom Scale (PSS) total score at baseline
Psoriasis Symptom Scale (PSS) total score at baseline(score on a scale)PlaceboSpesolimab SC Low DoseSpesolimab SC Medium DoseSpesolimab SC High DoseTotal
Mean3.6 ± 2.94.1 ± 3.83.9 ± 2.95.3 ± 3.84.2 ± 3.4

1 further baseline measures are reported on the registry.

08

Study locations

72 sites
  • Oakland Hills Dermatology
    Auburn Hills, Michigan 48326, United States
  • Washington University School of Medicine
    St Louis, Missouri 63108, United States
  • Buenos Aires Skin S.A.
    CABA, C1055AA0, Argentina
  • Hospital Italiano de Buenos Aires
    CABA, C1056AB, Argentina
  • Brussels - UNIV Saint-Luc
    Brussels, 1200, Belgium
  • Clínica Dermacross S.A.
    Vitacura, 7640881, Chile
  • Sun yet-sen Memorial Hospital, Sun yet-sen Univesity
    Guangzhou, 510288, China
  • The Second Affiliated Hospital Zhejiang University School of Medicine
    Hangzhou, 310009, China
  • Shanghai Skin Disease Hospital
    Shanghai, 200000, China
  • Huashan Hospital, Fudan University
    Shanghai, 200040, China
  • The First Hospital of China Medical University
    Shenyang, 110001, China
  • Tianjin Academy of Traditional Chinese Medicine Affiliated Hospital
    Tianjin, 300120, China
  • Second Affiliated Hospital of Xi'an JiaoTong University
    Xi'an, 710004, China
  • HOP l'Archet
    Nice, 06200, France
  • HOP Saint-Louis
    Paris, 75010, France
  • Fachklinik Bad Bentheim
    Bad Bentheim, 48455, Germany
  • Universitätsklinikum Bonn AöR
    Bonn, 53127, Germany
  • Universitätsklinikum Frankfurt
    Frankfurt am Main, 60596, Germany
  • Klinikum der Universität München - Campus Innenstadt
    München, 80337, Germany
  • Universitätsklinikum Münster
    Münster, 48149, Germany
  • Klinikum Oldenburg AöR
    Oldenburg, 26133, Germany
  • Universitätsklinikum Würzburg AÖR
    Würzburg, 97080, Germany
  • General Hospital of Thessaloniki "Ippokrateio"
    Thessaloniki, 54643, Greece
  • Istituto Clinico Humanitas
    Rozzano (MI), 20089, Italy
  • Nagoya City University Hospital
    Aichi, Nagoya, 467-8602, Japan
  • Kyushu Rosai Hospital
    Fukuoka, Kitakyushu, 800-0296, Japan
  • Tokyo Medical University Ibaraki Medical Center
    Ibaraki, Inashiki-gun, 300-0395, Japan
  • Saitama Medical University Hospital
    Saitama, Iruma-gun, 350-0495, Japan
  • Tokyo Medical University Hachioji Medical Center
    Tokyo, Hachioji, 193-0998, Japan
  • Tokyo Medical University Hospital
    Tokyo, Shinjuku-ku, 160-0023, Japan
  • Hospital Raja Permaisuri Bainun
    Ipoh, 30450, Malaysia
  • Hospital Sultanah Aminah
    Johor Bahru, 80100, Malaysia
  • Hospital Sultan Ismail
    Johor Bahru, 81100, Malaysia
  • Queen Elizabeth Hospital
    Kota Kinabalu, 88586, Malaysia
  • Hospital Kuala Lumpur
    Kuala Lumpur, 50586, Malaysia
  • Sarawak General Hospital
    Kuching, Sarawak, 93586, Malaysia
  • Hospital Pakar Sultanah Fatimah
    Muar town, 84000, Malaysia
  • Hospital Pulau Pinang
    Pulau Pinang, 10990, Malaysia
  • Centro de Investigación de Enfermedades Autoinmunes S.C.
    Guadalajara, 44610, Mexico
  • Hospital Universitario Dr Jose Eleuterio Gonzalez
    Monterrey, 64460, Mexico
  • Erasmus Medisch Centrum
    Rotterdam, 3015 GD, Netherlands
  • Southern Philippines Medical Center
    Davao City, 8000, Philippines
  • Iloilo Doctors Hospital
    Iloilo City, Iloilo, 5000, Philippines
  • Center for Skin Research, Testing and Product Development
    Makati City, 1229, Philippines
  • SBHI Chelyabinsk Reg.Clin.Derma.Dispen.
    Chelyabinsk, 454048, Russia
  • LLC "Medical Center Azbuka Zdorovia"
    Kazan', 420111, Russia
  • FSBEI HE "Kirov State Medical University"
    Kirov, 610035, Russia
  • LLC Skin Disease Clinic of Pier Volkenstein, St. Petersburg
    Saint Petersburg, 190123, Russia
  • LLC "Avrora Medfort"
    Saint Petersburg, 194156, Russia
  • 1stPavlov St.Med.Univ.St.-Petersburg Res.Inst.
    Saint Petersburg, 197022, Russia
  • Saratov State Med.Univ.n.a.Razumovskogo
    Saratov, 410028, Russia
  • Arthritis Clinical Research Trials
    Cape Town, 7405, South Africa
  • Pusan National Univ. Hosp
    Busan, 49241, South Korea
  • Severance Hospital
    Seoul, 03722, South Korea
  • Hospital Sant Joan de Déu
    Esplugues Del Llobregat, 08950, Spain
  • Hospital Universitario 12 de Octubre
    Madrid, 28041, Spain
  • Chang Gung Medical Foundation (CGMF) - Linkou Bran
    Linkou District, 333, Taiwan
  • National Taiwan University Hospital
    Taipei, 10002, Taiwan
  • King Chulalongkorn Memorial Hospital
    Bangkok, 10330, Thailand
  • Institute of Dermatology
    Bangkok, 10400, Thailand
  • Ramathibodi Hospital
    Ratchatewi, Bangkok, 10400, Thailand
  • Hedi Chaker Hospital, Department of Dermatology
    Sfax, 1053, Tunisia
  • Farhat Hached Hospital
    Sousse, 4000, Tunisia
  • La Rabta Hospital
    Tunis, 1007, Tunisia
  • Charles Nicolle Hospital
    Tunis, 1008, Tunisia
  • Habib Thameur Hospital
    Tunis, 1008, Tunisia
  • Uludag University Medicine Faculty Departmant of Dermatology
    Bursa, 16059, Turkey (Türkiye)
  • Bezmi Alem Valide Sultan Vakif Gureba Egitim ve Arastirma Hastanesi
    Istanbul, 34093, Turkey (Türkiye)
  • Istanbul Universitesi Cerrahpasa Tip Fakultesi
    Istanbul, 34098, Turkey (Türkiye)
  • Marmara Universitesi Tip Fakultesi
    Istanbul, 34460, Turkey (Türkiye)
  • National Hospital of Dermatology and Venereology
    Hà Nội, 10000, Vietnam
  • HCMC Hospital of Dermato-Venereology
    Ho Chi Minh City, 70000, Vietnam
09

References and documents

Publications

  • Gordon KB, Augustin M, Barker J, Tada Y, Lebwohl MG, Tang M, Hofmann P, Thoma C, Gottlieb AB. Effect of spesolimab on sustained disease control in patients with generalized pustular psoriasis: Post hoc analysis of the EFFISAYIL 2 study. J Am Acad Dermatol. 2025 Jun;92(6):1235-1242. doi: 10.1016/j.jaad.2025.01.089. Epub 2025 Mar 7. PubMed 40057892 ↗
  • Morita A, Choon SE, Bachelez H, Anadkat MJ, Marrakchi S, Zheng M, Tsai TF, Turki H, Hua H, Rajeswari S, Thoma C, Burden AD. Design of Effisayil 2: A Randomized, Double-Blind, Placebo-Controlled Study of Spesolimab in Preventing Flares in Patients with Generalized Pustular Psoriasis. Dermatol Ther (Heidelb). 2023 Jan;13(1):347-359. doi: 10.1007/s13555-022-00835-6. Epub 2022 Nov 5. PubMed 36333618 ↗

Related links

Study documents

  • Study protocol · Jul 28, 2022
  • Statistical analysis plan · Dec 2, 2022

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No — Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.mystudywindow.com/msw/datatransparency

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 20, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04399837
Lead sponsor
Boehringer Ingelheim
Responsible party
Sponsor
First posted
May 22, 2020
Start date
Jun 4, 2020
Primary completion
Nov 23, 2022
Completion
Nov 23, 2022
Results posted
Dec 14, 2023
Last update
Oct 20, 2025

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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