Generalized Pustular PsoriasisPhase 2NCT04399837
A Study to Test Whether BI 655130 (Spesolimab) Prevents Flare-ups in Patients With Generalized Pustular Psoriasis
A Phase 2 study testing Spesolimab in people with Generalized Pustular Psoriasis, run by Boehringer Ingelheim at 72 sites in 23 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Generalized Pustular Psoriasis
- What is being tested?
- Spesolimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 123 participants
- Where
- 72 sitesin 23 countries
- Study dates
- 4 Jun 2020to 23 Nov 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome.
- Patients with primary erythrodermic psoriasis vulgaris.
- 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.
- Treatment with:
- 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.
- Any prior exposure to BI 655130 or another IL36R inhibitor biologic.
Show all 15Show fewer
- 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.
- 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.
- 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.
- History of allergy/hypersensitivity to the systemically administered trial medication agent or its excipients.
- Further exclusion criteria apply.
Trial locations and contacts
72 sites in 23 countries.
72 sites in 23 countries
Malaysia8 sites
Hospital Raja Permaisuri Bainun
Ipoh, 30450
Hospital Sultanah Aminah
Johor Bahru, 80100
Hospital Sultan Ismail
Johor Bahru, 81100
Queen Elizabeth Hospital
Kota Kinabalu, 88586
Hospital Kuala Lumpur
Kuala Lumpur, 50586
Sarawak General Hospital
Kuching, Sarawak, 93586
Hospital Pakar Sultanah Fatimah
Muar town, 84000
Hospital Pulau Pinang
Pulau Pinang, 10990
China7 sites
Sun yet-sen Memorial Hospital, Sun yet-sen Univesity
Guangzhou, 510288
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, 310009
Shanghai Skin Disease Hospital
Shanghai, 200000
Huashan Hospital, Fudan University
Shanghai, 200040
The First Hospital of China Medical University
Shenyang, 110001
Tianjin Academy of Traditional Chinese Medicine Affiliated Hospital
Tianjin, 300120
Second Affiliated Hospital of Xi'an JiaoTong University
Xi'an, 710004
Germany7 sites
Fachklinik Bad Bentheim
Bad Bentheim, 48455
Universitätsklinikum Bonn AöR
Bonn, 53127
Universitätsklinikum Frankfurt
Frankfurt am Main, 60596
Klinikum der Universität München - Campus Innenstadt
München, 80337
Universitätsklinikum Münster
Münster, 48149
Klinikum Oldenburg AöR
Oldenburg, 26133
Universitätsklinikum Würzburg AÖR
Würzburg, 97080
Russia7 sites
SBHI Chelyabinsk Reg.Clin.Derma.Dispen.
Chelyabinsk, 454048
LLC "Medical Center Azbuka Zdorovia"
Kazan', 420111
FSBEI HE "Kirov State Medical University"
Kirov, 610035
LLC Skin Disease Clinic of Pier Volkenstein, St. Petersburg
Saint Petersburg, 190123
LLC "Avrora Medfort"
Saint Petersburg, 194156
1stPavlov St.Med.Univ.St.-Petersburg Res.Inst.
Saint Petersburg, 197022
Saratov State Med.Univ.n.a.Razumovskogo
Saratov, 410028
Japan6 sites
Nagoya City University Hospital
Aichi, Nagoya, 467-8602
Kyushu Rosai Hospital
Fukuoka, Kitakyushu, 800-0296
Tokyo Medical University Ibaraki Medical Center
Ibaraki, Inashiki-gun, 300-0395
Saitama Medical University Hospital
Saitama, Iruma-gun, 350-0495
Tokyo Medical University Hachioji Medical Center
Tokyo, Hachioji, 193-0998
Tokyo Medical University Hospital
Tokyo, Shinjuku-ku, 160-0023
Tunisia5 sites
Hedi Chaker Hospital, Department of Dermatology
Sfax, 1053
Farhat Hached Hospital
Sousse, 4000
La Rabta Hospital
Tunis, 1007
Charles Nicolle Hospital
Tunis, 1008
Habib Thameur Hospital
Tunis, 1008
Turkey (Türkiye)4 sites
Uludag University Medicine Faculty Departmant of Dermatology
Bursa, 16059
Bezmi Alem Valide Sultan Vakif Gureba Egitim ve Arastirma Hastanesi
Istanbul, 34093
Istanbul Universitesi Cerrahpasa Tip Fakultesi
Istanbul, 34098
Marmara Universitesi Tip Fakultesi
Istanbul, 34460
Philippines3 sites
Southern Philippines Medical Center
Davao City, 8000
Iloilo Doctors Hospital
Iloilo City, Iloilo, 5000
Center for Skin Research, Testing and Product Development
Makati City, 1229
Thailand3 sites
King Chulalongkorn Memorial Hospital
Bangkok, 10330
Institute of Dermatology
Bangkok, 10400
Ramathibodi Hospital
Ratchatewi, Bangkok, 10400
United States2 sites
Oakland Hills Dermatology
Auburn Hills, Michigan, 48326
Washington University School of Medicine
St Louis, Missouri, 63108
Argentina2 sites
Buenos Aires Skin S.A.
CABA, C1055AA0
Hospital Italiano de Buenos Aires
CABA, C1056AB
Mexico2 sites
Centro de Investigación de Enfermedades Autoinmunes S.C.
Guadalajara, 44610
Hospital Universitario Dr Jose Eleuterio Gonzalez
Monterrey, 64460
South Korea2 sites
Pusan National Univ. Hosp
Busan, 49241
Severance Hospital
Seoul, 03722
Spain2 sites
Hospital Sant Joan de Déu
Esplugues Del Llobregat, 08950
Hospital Universitario 12 de Octubre
Madrid, 28041
Taiwan2 sites
Chang Gung Medical Foundation (CGMF) - Linkou Bran
Linkou District, 333
National Taiwan University Hospital
Taipei, 10002
Vietnam2 sites
National Hospital of Dermatology and Venereology
Hà Nội, 10000
HCMC Hospital of Dermato-Venereology
Ho Chi Minh City, 70000
Greece1 site
General Hospital of Thessaloniki "Ippokrateio"
Thessaloniki, 54643
About this study
Official title: Effisayil™ 2: Multi-center, Randomized, Parallel Group, Double Blind, Placebo Controlled, Phase IIb Dose-finding Study to Evaluate Efficacy and Safety of BI 655130 (Spesolimab) Compared to Placebo in Preventing Generalized Pustular Psoriasis (GPP) Flares in Patients With History of GPP.
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.
Read more
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.
- Conditions
- Generalized Pustular Psoriasis
- Being tested
- Spesolimab, Placebo
- Medical subject headings
- Psoriasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SpesolimabDrug
Solution for injection
PlaceboDrug
Solution for injection
| Study group | Role | What they receive |
|---|---|---|
| Spesolimab SC low dose | Experimental | Not described Receives: Drug: Spesolimab |
| Spesolimab SC medium dose | Experimental | Not described Receives: Drug: Spesolimab |
| Spesolimab SC high dose | Experimental | Not described Receives: Drug: Spesolimab |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 (5)
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,
- if 0 < mean < 1.5;
- if 1.5 ≤ mean < 2.5;
- if 2.5 ≤ mean < 3.5;
- if mean ≥ 3.5.
Measured: 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.
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)).
Measured: 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.
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).
Measured: 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.
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.
Measured: 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.
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.
Measured: Up to 62 weeks (for detailed timeframe see description).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2020
- Study start4 Jun 2020
- Primary completion23 Nov 2022
- Study completion23 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Generalized Pustular PsoriasisStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 123 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04399837
- Sponsor's protocol ID
- 1368-0027
- Other IDs
- 2018-003081-14, 2018
- Status last verified
- Oct 2025
- First submitted
- 20 May 2020
- Last updated
- 20 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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
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 40057892Morita 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
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effisayil™ 2: Multi-center, Randomized, Parallel Group, Double Blind, Placebo Controlled, Phase IIb Dose-finding Study to Evaluate Efficacy and Safety of BI 655130 (Spesolimab) Compared to Placebo in Preventing Generalized Pustular Psoriasis (GPP) Flares in Patients With History of GPP.
- ClinicalTrials.gov ID
- NCT04399837
- Acronym / sponsor ID
- 1368-0027
- Conditions
- Generalized Pustular Psoriasis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 123 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- 4 Jun 2020 (actual)
- Primary completion
- 23 Nov 2022 (actual)
- Study completion
- 23 Nov 2022 (actual)
- First posted
- 22 May 2020 (actual)
- Results
- Posted 14 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Spesolimab SC low dose | Experimental | Drug: Spesolimab |
| Spesolimab SC medium dose | Experimental | Drug: Spesolimab |
| Spesolimab SC high dose | Experimental | Drug: Spesolimab |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (15 listed)
- Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome.
- Patients with primary erythrodermic psoriasis vulgaris.
- 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.
- Treatment with:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SpesolimabSolution for injection | Drug | Spesolimab SC high dose, Spesolimab SC low dose, Spesolimab SC medium dose |
| PlaceboSolution for injection | Drug | Placebo |
Primary outcome
Time to First Generalized Pustular Psoriasis (GPP) Flare
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.
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.
5 secondary outcomes
Key Secondary Endpoint: The Occurrence of at Least One Generalized Pustular Psoriasis (GPP) Flare up to Week 48
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.
Time to First Worsening of Psoriasis Symptom Scale (PSS) up to Week 48
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.
Time to First Worsening of Dermatology Quality of Life Index (DLQI) up to Week 48
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.
Sustained Remission
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.
The Occurrence of Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 62 weeks (for detailed timeframe see description).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40057892Gordon 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.
- Derived from this study: PubMed 36333618Morita 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.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
72 sites in 23 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Spesolimab in people with Generalized Pustular Psoriasis, run by Boehringer Ingelheim at 72 sites in 23 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 72 sites in Malaysia, China, Germany, Russia, Japan and 18 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to First Generalized Pustular Psoriasis (GPP) Flare (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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04399837, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2025.