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

NCT ID: NCT04399837

Last Updated: 2025-10-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 123 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-06-04
• Study Completion Date: 2022-11-23

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Spesolimab SC low dose

Group Type: EXPERIMENTAL

Spesolimab

Intervention Type: DRUG

Solution for injection

Spesolimab SC medium dose

Group Type: EXPERIMENTAL

Spesolimab

Intervention Type: DRUG

Solution for injection

Spesolimab SC high dose

Group Type: EXPERIMENTAL

Spesolimab

Intervention Type: DRUG

Solution for injection

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Solution for injection

Interventions

Spesolimab

Solution for injection

Intervention Type: DRUG

Placebo

Solution for injection

Intervention Type: DRUG

Eligibility Criteria

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

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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boehringer Ingelheim

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Oakland Hills Dermatology

Auburn Hills, Michigan, United States

Washington University School of Medicine

St Louis, Missouri, United States

Buenos Aires Skin S.A.

CABA, , Argentina

Hospital Italiano de Buenos Aires

CABA, , Argentina

Brussels - UNIV Saint-Luc

Brussels, , Belgium

Clínica Dermacross S.A.

Vitacura, , Chile

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

Guangzhou, , China

The Second Affiliated Hospital Zhejiang University School of Medicine

Hangzhou, , China

Shanghai Skin Disease Hospital

Shanghai, , China

Huashan Hospital, Fudan University

Shanghai, , China

The First Hospital of China Medical University

Shenyang, , China

Tianjin Academy of Traditional Chinese Medicine Affiliated Hospital

Tianjin, , China

Second Affiliated Hospital of Xi'an JiaoTong University

Xi'an, , China

HOP l'Archet

Nice, , France

HOP Saint-Louis

Paris, , France

Fachklinik Bad Bentheim

Bad Bentheim, , Germany

Universitätsklinikum Bonn AöR

Bonn, , Germany

Universitätsklinikum Frankfurt

Frankfurt am Main, , Germany

Klinikum der Universität München - Campus Innenstadt

München, , Germany

Universitätsklinikum Münster

Münster, , Germany

Klinikum Oldenburg AöR

Oldenburg, , Germany

Universitätsklinikum Würzburg AÖR

Würzburg, , Germany

General Hospital of Thessaloniki "Ippokrateio"

Thessaloniki, , Greece

Istituto Clinico Humanitas

Rozzano (MI), , Italy

Nagoya City University Hospital

Aichi, Nagoya, , Japan

Kyushu Rosai Hospital

Fukuoka, Kitakyushu, , Japan

Tokyo Medical University Ibaraki Medical Center

Ibaraki, Inashiki-gun, , Japan

Saitama Medical University Hospital

Saitama, Iruma-gun, , Japan

Tokyo Medical University Hachioji Medical Center

Tokyo, Hachioji, , Japan

Tokyo Medical University Hospital

Tokyo, Shinjuku-ku, , Japan

Hospital Raja Permaisuri Bainun

Ipoh, , Malaysia

Hospital Sultanah Aminah

Johor Bahru, , Malaysia

Hospital Sultan Ismail

Johor Bahru, , Malaysia

Queen Elizabeth Hospital

Kota Kinabalu, , Malaysia

Hospital Kuala Lumpur

Kuala Lumpur, , Malaysia

Sarawak General Hospital

Kuching, Sarawak, , Malaysia

Hospital Pakar Sultanah Fatimah

Muar town, , Malaysia

Hospital Pulau Pinang

Pulau Pinang, , Malaysia

Centro de Investigación de Enfermedades Autoinmunes S.C.

Guadalajara, , Mexico

Hospital Universitario Dr Jose Eleuterio Gonzalez

Monterrey, , Mexico

Erasmus Medisch Centrum

Rotterdam, , Netherlands

Southern Philippines Medical Center

Davao City, , Philippines

Iloilo Doctors Hospital

Iloilo City, Iloilo, , Philippines

Center for Skin Research, Testing and Product Development

Makati City, , Philippines

SBHI Chelyabinsk Reg.Clin.Derma.Dispen.

Chelyabinsk, , Russia

LLC "Medical Center Azbuka Zdorovia"

Kazan', , Russia

FSBEI HE "Kirov State Medical University"

Kirov, , Russia

LLC Skin Disease Clinic of Pier Volkenstein, St. Petersburg

Saint Petersburg, , Russia

LLC "Avrora Medfort"

Saint Petersburg, , Russia

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

Saint Petersburg, , Russia

Saratov State Med.Univ.n.a.Razumovskogo

Saratov, , Russia

Arthritis Clinical Research Trials

Cape Town, , South Africa

Pusan National Univ. Hosp

Busan, , South Korea

Severance Hospital

Seoul, , South Korea

Hospital Sant Joan de Déu

Esplugues Del Llobregat, , Spain

Hospital Universitario 12 de Octubre

Madrid, , Spain

Chang Gung Medical Foundation (CGMF) - Linkou Bran

Linkou District, , Taiwan

National Taiwan University Hospital

Taipei, , Taiwan

King Chulalongkorn Memorial Hospital

Bangkok, , Thailand

Institute of Dermatology

Bangkok, , Thailand

Ramathibodi Hospital

Ratchatewi, Bangkok, , Thailand

Hedi Chaker Hospital, Department of Dermatology

Sfax, , Tunisia

Farhat Hached Hospital

Sousse, , Tunisia

La Rabta Hospital

Tunis, , Tunisia

Charles Nicolle Hospital

Tunis, , Tunisia

Habib Thameur Hospital

Tunis, , Tunisia

Uludag University Medicine Faculty Departmant of Dermatology

Bursa, , Turkey (Türkiye)

Bezmi Alem Valide Sultan Vakif Gureba Egitim ve Arastirma Hastanesi

Istanbul, , Turkey (Türkiye)

Istanbul Universitesi Cerrahpasa Tip Fakultesi

Istanbul, , Turkey (Türkiye)

Marmara Universitesi Tip Fakultesi

Istanbul, , Turkey (Türkiye)

National Hospital of Dermatology and Venereology

Hà Nội, , Vietnam

HCMC Hospital of Dermato-Venereology

Ho Chi Minh City, , Vietnam

Countries

United States; Argentina; Belgium; Chile; China; France; Germany; Greece; Italy; Japan; Malaysia; Mexico; Netherlands; Philippines; Russia; South Africa; South Korea; Spain; Taiwan; Thailand; Tunisia; Turkey (Türkiye); Vietnam

References

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.

Reference Type: DERIVED

PMID: 36333618 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

http://www.mystudywindow.com

Related Info

Other Identifiers

2018-003081-14

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

1368-0027

Identifier Type: -

Identifier Source: org_study_id