Effisayil™ 1: A Study to Test Spesolimab (BI 655130) in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis

NCT ID: NCT03782792

Last Updated: 2025-10-16

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-01-31

Study Completion Date

2021-01-05

Brief Summary

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To evaluate efficacy, safety, and tolerability of spesolimab (BI 655130) compared to placebo in patients with Generalized Pustular Psoriasis (GPP) presenting with an acute flare of moderate to severe intensity.

Detailed Description

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Conditions

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Generalized Pustular Psoriasis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Spesolimab

Group Type EXPERIMENTAL

Spesolimab

Intervention Type DRUG

Solution for infusion

Placebo

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Solution for infusion

Interventions

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Spesolimab

Solution for infusion

Intervention Type DRUG

Placebo

Solution for infusion

Intervention Type DRUG

Other Intervention Names

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BI 655130

Eligibility Criteria

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Inclusion Criteria

* Patients with GPPGA of 0 or 1 and a known and documented history of GPP per European Rare And Severe Psoriasis Expert Network (ERASPEN) criteria regardless of IL36RN mutation status, with previous evidence of fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis with peripheral blood neutrophilia (above ULN) OR

\-- Patients with an acute flare of moderate to severe intensity meeting the (ERASPEN) criteria of GPP with a known and documented history of GPP (per ERASPEN criteria) regardless of IL36RN mutation status, with previous evidence of fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis with peripheral blood neutrophilia (above ULN)
* Male or female patients, aged 18 to 75 years at screening.
* Signed and dated written informed consent prior to admission to the study in accordance with ICH GCP and local legislation prior to start of any screening procedures.
* Women of childbearing potential 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. Note: A woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. Tubal ligation is not a method of permanent sterilization. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause

Exclusion Criteria

* Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome.
* Patients with primary erythrodermic psoriasis vulgaris.
* Patients with primary plaque psoriasis vulgaris without presence of pustules or with pustules that are restricted to psoriatic plaques.
* Drug-triggered Acute Generalized Exanthematous Pustulosis (AGEP).
* Immediate life-threatening flare of GPP or requiring intensive care treatment, according to the investigator's judgement. Life-threatening complications mainly include, but are not limited to, cardiovascular/cytokine driven shock, pulmonary distress syndrome, or renal failure.
* Severe, progressive, or uncontrolled hepatic disease, defined as \>3- fold Upper Limit of Normal (ULN) elevation in AST or ALT or alkaline phosphatase, or \>2-fold ULN elevation in total bilirubin.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Boehringer Ingelheim

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

The First Hospital of Dalian Medical University

Dalian, , China

Site Status

2nd Affiliated Hosp Zhejiang University College of Medical

Hangzhou, , China

Site Status

Shanghai Skin Disease Hospital

Shanghai, , China

Site Status

Huashan Hospital, Fudan University

Shanghai, , China

Site Status

Tianjin Medical University General Hospital

Tianjin, , China

Site Status

HOP Saint-André

Bordeaux, , France

Site Status

HOP Saint-Louis

Paris, , France

Site Status

HOP Robert Debré

Reims, , France

Site Status

Charité - Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Universitätsklinikum Bonn AöR

Bonn, , Germany

Site Status

Universitätsklinikum Essen AöR

Essen, , Germany

Site Status

Klinikum der Universität München - Campus Innenstadt

München, , Germany

Site Status

Nagoya City University Hospital

Aichi, Nagoya, , Japan

Site Status

Fukuoka University Hospital

Fukuoka, Fukuoka, , Japan

Site Status

Asahikawa Medical University Hospital

Hokkaido, Asahikawa, , Japan

Site Status

Tohoku University Hospital

Miyagi, Sendai, , Japan

Site Status

Tokyo Medical University Hachioji Medical Center

Tokyo, Hachioji, , Japan

Site Status

Tokyo Medical University Hospital

Tokyo, Shinjuku-ku, , Japan

Site Status

Hospital Sultanah Aminah

Johor Bahru, , Malaysia

Site Status

Hospital Sultan Ismail

Johor Bahru, , Malaysia

Site Status

Hospital Kuala Lumpur

Kuala Lumpur, , Malaysia

Site Status

Hospital Selayang

Kuala Selangor, , Malaysia

Site Status

Hospital Pakar Sultanah Fatimah

Muar town, , Malaysia

Site Status

Hospital Raja Permaisuri Bainun

Negeri Perak/Ipoh, , Malaysia

Site Status

Hospital Pulau Pinang

Pulau Pinang, , Malaysia

Site Status

National University Hospital

Singapore, , Singapore

Site Status

Severance Hospital

Seoul, , South Korea

Site Status

University Hospital of Lausanne

Lausanne, , Switzerland

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Ramathibodi Hospital

Ratchatewi, Bangkok, , Thailand

Site Status

Farhat Hached Hospital

Sousse, , Tunisia

Site Status

La Rabta Hospital

Tunis, , Tunisia

Site Status

Hedi Chaker Hospital, Department of Dermatology

Tunisia, , Tunisia

Site Status

Countries

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United States China France Germany Japan Malaysia Singapore South Korea Switzerland Taiwan Thailand Tunisia

References

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Lebwohl MG, Thoma C, Haeufel T. Spesolimab use in generalised pustular psoriasis flares - Authors' reply. Lancet. 2024 Aug 31;404(10455):847-848. doi: 10.1016/S0140-6736(24)01557-5. No abstract available.

Reference Type DERIVED
PMID: 39216969 (View on PubMed)

Gwillim EC, Nichols AJ. Spesolimab for generalized pustular psoriasis: a review of two key clinical trials supporting initial US regulatory approval. Front Immunol. 2024 Jul 22;15:1359481. doi: 10.3389/fimmu.2024.1359481. eCollection 2024.

Reference Type DERIVED
PMID: 39104539 (View on PubMed)

Tsai TF, Zheng M, Ding Y, Song Z, Liu Q, Chen Y, Hu H, Xu J. Efficacy and Safety of Spesolimab in Patients with Generalized Pustular Psoriasis: A Subgroup Analysis of Chinese Patients in the Effisayil 1 Trial. Dermatol Ther (Heidelb). 2023 Dec;13(12):3097-3110. doi: 10.1007/s13555-023-01037-4. Epub 2023 Oct 16.

Reference Type DERIVED
PMID: 37840119 (View on PubMed)

Bachelez H, Choon SE, Marrakchi S, Burden AD, Tsai TF, Morita A, Navarini AA, Zheng M, Xu J, Turki H, Anadkat MJ, Rajeswari S, Hua H, Vulcu SD, Hall D, Tetzlaff K, Thoma C, Lebwohl MG; Effisayil 1 Trial Investigators. Trial of Spesolimab for Generalized Pustular Psoriasis. N Engl J Med. 2021 Dec 23;385(26):2431-2440. doi: 10.1056/NEJMoa2111563.

Reference Type DERIVED
PMID: 34936739 (View on PubMed)

Choon SE, Lebwohl MG, Marrakchi S, Burden AD, Tsai TF, Morita A, Navarini AA, Zheng M, Xu J, Turki H, Rajeswari S, Deng H, Tetzlaff K, Thoma C, Bachelez H. Study protocol of the global Effisayil 1 Phase II, multicentre, randomised, double-blind, placebo-controlled trial of spesolimab in patients with generalized pustular psoriasis presenting with an acute flare. BMJ Open. 2021 Mar 30;11(3):e043666. doi: 10.1136/bmjopen-2020-043666.

Reference Type DERIVED
PMID: 33785490 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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2017-004231-37

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

1368-0013

Identifier Type: -

Identifier Source: org_study_id

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