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

NCT04399837 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: 1368-00272018-003081-14
• Study Type: interventional
• Target: 123
• Locations: 22 countries
• Sites: 68

Brief 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.

Conditions

Generalized Pustular Psoriasis

Outcome Measures

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.

  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

  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

  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

  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

  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)

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

Study Arms (4)

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

Interventions

Spesolimab DRUG

Solution for injection

Spesolimab SC high dose; Spesolimab SC low dose; Spesolimab SC medium dose

Placebo DRUG

Solution for injection

Placebo

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Boehringer Ingelheim: lead

Study Sites (72)

Oakland Hills Dermatology

Auburn Hills, Michigan, 48326, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63108, United States

Location

Buenos Aires Skin S.A.

CABA, C1055AA0, Argentina

Location

Hospital Italiano de Buenos Aires

CABA, C1056AB, Argentina

Location

Brussels - UNIV Saint-Luc

Brussels, 1200, Belgium

Location

Clínica Dermacross S.A.

Vitacura, 7640881, Chile

Location

Sun yet-sen Memorial Hospital, Sun yet-sen Univesity

Guangzhou, 510288, China

Location

The Second Affiliated Hospital Zhejiang University School of Medicine

Hangzhou, 310009, China

Location

Shanghai Skin Disease Hospital

Shanghai, 200000, China

Location

Huashan Hospital, Fudan University

Shanghai, 200040, China

Location

The First Hospital of China Medical University

Shenyang, 110001, China

Location

Tianjin Academy of Traditional Chinese Medicine Affiliated Hospital

Tianjin, 300120, China

Location

Second Affiliated Hospital of Xi'an JiaoTong University

Xi'an, 710004, China

Location

HOP l'Archet

Nice, 06200, France

Location

HOP Saint-Louis

Paris, 75010, France

Location

Fachklinik Bad Bentheim

Bad Bentheim, 48455, Germany

Location

Universitätsklinikum Bonn AöR

Bonn, 53127, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt am Main, 60596, Germany

Location

Klinikum der Universität München - Campus Innenstadt

München, 80337, Germany

Location

Universitätsklinikum Münster

Münster, 48149, Germany

Location

Klinikum Oldenburg AöR

Oldenburg, 26133, Germany

Location

Universitätsklinikum Würzburg AÖR

Würzburg, 97080, Germany

Location

General Hospital of Thessaloniki "Ippokrateio"

Thessaloniki, 54643, Greece

Location

Istituto Clinico Humanitas

Rozzano (MI), 20089, Italy

Location

Nagoya City University Hospital

Aichi, Nagoya, 467-8602, Japan

Location

Kyushu Rosai Hospital

Fukuoka, Kitakyushu, 800-0296, Japan

Location

Tokyo Medical University Ibaraki Medical Center

Ibaraki, Inashiki-gun, 300-0395, Japan

Location

Saitama Medical University Hospital

Saitama, Iruma-gun, 350-0495, Japan

Location

Tokyo Medical University Hachioji Medical Center

Tokyo, Hachioji, 193-0998, Japan

Location

Tokyo Medical University Hospital

Tokyo, Shinjuku-ku, 160-0023, Japan

Location

Hospital Raja Permaisuri Bainun

Ipoh, 30450, Malaysia

Location

Hospital Sultanah Aminah

Johor Bahru, 80100, Malaysia

Location

Hospital Sultan Ismail

Johor Bahru, 81100, Malaysia

Location

Queen Elizabeth Hospital

Kota Kinabalu, 88586, Malaysia

Location

Hospital Kuala Lumpur

Kuala Lumpur, 50586, Malaysia

Location

Sarawak General Hospital

Kuching, Sarawak, 93586, Malaysia

Location

Hospital Pakar Sultanah Fatimah

Muar town, 84000, Malaysia

Location

Hospital Pulau Pinang

Pulau Pinang, 10990, Malaysia

Location

Centro de Investigación de Enfermedades Autoinmunes S.C.

Guadalajara, 44610, Mexico

Location

Hospital Universitario Dr Jose Eleuterio Gonzalez

Monterrey, 64460, Mexico

Location

Erasmus Medisch Centrum

Rotterdam, 3015 GD, Netherlands

Location

Southern Philippines Medical Center

Davao City, 8000, Philippines

Location

Iloilo Doctors Hospital

Iloilo City, Iloilo, 5000, Philippines

Location

Center for Skin Research, Testing and Product Development

Makati City, 1229, Philippines

Location

SBHI Chelyabinsk Reg.Clin.Derma.Dispen.

Chelyabinsk, 454048, Russia

Location

LLC "Medical Center Azbuka Zdorovia"

Kazan', 420111, Russia

Location

FSBEI HE "Kirov State Medical University"

Kirov, 610035, Russia

Location

LLC Skin Disease Clinic of Pier Volkenstein, St. Petersburg

Saint Petersburg, 190123, Russia

Location

LLC "Avrora Medfort"

Saint Petersburg, 194156, Russia

Location

1stPavlov St.Med.Univ.St.-Petersburg Res.Inst.

Saint Petersburg, 197022, Russia

Location

Saratov State Med.Univ.n.a.Razumovskogo

Saratov, 410028, Russia

Location

Arthritis Clinical Research Trials

Cape Town, 7405, South Africa

Location

Pusan National Univ. Hosp

Busan, 49241, South Korea

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Hospital Sant Joan de Déu

Esplugues Del Llobregat, 08950, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Chang Gung Medical Foundation (CGMF) - Linkou Bran

Linkou District, 333, Taiwan

Location

National Taiwan University Hospital

Taipei, 10002, Taiwan

Location

King Chulalongkorn Memorial Hospital

Bangkok, 10330, Thailand

Location

Institute of Dermatology

Bangkok, 10400, Thailand

Location

Ramathibodi Hospital

Ratchatewi, Bangkok, 10400, Thailand

Location

Hedi Chaker Hospital, Department of Dermatology

Sfax, 1053, Tunisia

Location

Farhat Hached Hospital

Sousse, 4000, Tunisia

Location

La Rabta Hospital

Tunis, 1007, Tunisia

Location

Charles Nicolle Hospital

Tunis, 1008, Tunisia

Location

Habib Thameur Hospital

Tunis, 1008, Tunisia

Location

Uludag University Medicine Faculty Departmant of Dermatology

Bursa, 16059, Turkey (Türkiye)

Location

Bezmi Alem Valide Sultan Vakif Gureba Egitim ve Arastirma Hastanesi

Istanbul, 34093, Turkey (Türkiye)

Location

Istanbul Universitesi Cerrahpasa Tip Fakultesi

Istanbul, 34098, Turkey (Türkiye)

Location

Marmara Universitesi Tip Fakultesi

Istanbul, 34460, Turkey (Türkiye)

Location

National Hospital of Dermatology and Venereology

Hà Nội, 10000, Vietnam

Location

HCMC Hospital of Dermato-Venereology

Ho Chi Minh City, 70000, Vietnam

Location

Related Publications (1)

• 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.

  PMID: 36333618 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Psoriasis

Interventions

spesolimab

Condition Hierarchy (Ancestors)

Skin Diseases, Papulosquamous; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Boehringer Ingelheim, Call Center
• Organization: Boehringer Ingelheim

clintriage.rdg@boehringer-ingelheim.com

1-800-243-0127

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2020
• First Posted: May 22, 2020
• Study Start: June 4, 2020
• Primary Completion: November 23, 2022
• Study Completion: November 23, 2022
• Last Updated: October 20, 2025
• Results First Posted: December 14, 2023

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (7); CL (1); TU (5); DE (7); US (2); GR (1); NL (1); BE (1); MY (8); ZA (1); TW (2); JP (6); RU (7); TH (3); KR (2); IT (1); ME (2); AR (2); FR (2); VN (2); PH (3); ES (2)