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

NCT03782792 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: 1368-00132017-004231-37
• Study Type: interventional
• Target: 53
• Locations: 12 countries
• Sites: 37

Brief Summary

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.

Conditions

Generalized Pustular Psoriasis

Outcome Measures

Primary Outcomes (1)

• Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1

  The Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) relies on clinical assessment of the Generalized Pustular Psoriasis (GPP) patient's skin presentation. The investigator (or qualified site personnel) scored the erythema, pustules, and scaling of all GPP lesions from 0 to 4. The GPPGA pustulation subscore ranges from 0 to 4 where: 0 = clear; 1. = almost clear; 2. = mild: 3. = moderate; 4. = severe. A lower GPPGA pustulation subscore indicates a better outcome. A GPPGA pustulation subscore of 0 means no visible pustules. The proportion of patients who achieved a GPPGA pustulation subscore of 0 at Week 1 is reported.

  At Week 1.

Secondary Outcomes (15)

• Key Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1

  At Week 1.

• Proportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4

  At Week 4.

• Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4

  Baseline and at Week 4.

• Change From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4

  Baseline and at Week 4.

• Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Score at Week 4

  Baseline and at Week 4.

Study Arms (2)

Spesolimab

EXPERIMENTAL

Drug: Spesolimab

Placebo

EXPERIMENTAL

Drug: Placebo

Interventions

Spesolimab DRUG

Solution for infusion

Also known as: BI 655130

Spesolimab

Placebo DRUG

Solution for infusion

Placebo

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Boehringer Ingelheim: lead

Study Sites (37)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

University of Miami

Miami, Florida, 33125, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

The First Hospital of Dalian Medical University

Dalian, 116011, China

Location

2nd Affiliated Hosp Zhejiang University College of Medical

Hangzhou, 310009, China

Location

Shanghai Skin Disease Hospital

Shanghai, 200000, China

Location

Huashan Hospital, Fudan University

Shanghai, 200040, China

Location

Tianjin Medical University General Hospital

Tianjin, 30052, China

Location

HOP Saint-André

Bordeaux, 33000, France

Location

HOP Saint-Louis

Paris, 75010, France

Location

HOP Robert Debré

Reims, 51092, France

Location

Charité - Universitätsmedizin Berlin

Berlin, 10117, Germany

Location

Universitätsklinikum Bonn AöR

Bonn, 53127, Germany

Location

Universitätsklinikum Essen AöR

Essen, 45147, Germany

Location

Klinikum der Universität München - Campus Innenstadt

München, 80337, Germany

Location

Nagoya City University Hospital

Aichi, Nagoya, 467-8602, Japan

Location

Fukuoka University Hospital

Fukuoka, Fukuoka, 814-0180, Japan

Location

Asahikawa Medical University Hospital

Hokkaido, Asahikawa, 078-8510, Japan

Location

Tohoku University Hospital

Miyagi, Sendai, 980-8574, 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 Sultanah Aminah

Johor Bahru, 80100, Malaysia

Location

Hospital Sultan Ismail

Johor Bahru, 81100, Malaysia

Location

Hospital Kuala Lumpur

Kuala Lumpur, 50586, Malaysia

Location

Hospital Selayang

Kuala Selangor, 68100, Malaysia

Location

Hospital Pakar Sultanah Fatimah

Muar town, 84000, Malaysia

Location

Hospital Raja Permaisuri Bainun

Negeri Perak/Ipoh, 30450, Malaysia

Location

Hospital Pulau Pinang

Pulau Pinang, 10990, Malaysia

Location

National University Hospital

Singapore, 119074, Singapore

Location

Severance Hospital

Seoul, 03722, South Korea

Location

University Hospital of Lausanne

Lausanne, 1011, Switzerland

Location

National Taiwan University Hospital

Taipei, 10016, Taiwan

Location

Ramathibodi Hospital

Ratchatewi, Bangkok, 10400, Thailand

Location

Farhat Hached Hospital

Sousse, 4000, Tunisia

Location

La Rabta Hospital

Tunis, 1007, Tunisia

Location

Hedi Chaker Hospital, Department of Dermatology

Tunisia, 1053, Tunisia

Location

Related Publications (5)

• 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.

  PMID: 39216969 DERIVED

• 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.

  PMID: 39104539 DERIVED

• 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.

  PMID: 37840119 DERIVED

• 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.

  PMID: 34936739 DERIVED

• 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.

  PMID: 33785490 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Psoriasis

Interventions

spesolimab

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

Due to the trial design, a large proportion of patients had received escape medication or non-randomized spesolimab by Week 4, based on worsening, insufficient response, or non-response. This should be taken into account for the interpretation of the results at Week 4. A large proportion of patients in both arms had been treated as non-responders at Week 4, and the true efficacy outcomes for the randomized treatment at this time-point were never observed for the analysis.

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: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 19, 2018
• First Posted: December 20, 2018
• Study Start: January 31, 2019
• Primary Completion: September 23, 2020
• Study Completion: January 5, 2021
• Last Updated: October 16, 2025
• Results First Posted: March 9, 2022

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3); US (4); JP (6); CN (5); TU (3); TH (1); MY (7); KR (1); CH (1); DE (4); TW (1); SG (1)