A Study to Test Whether Spesolimab Helps People With Generalized Pustular Psoriasis (GPP) Who Need Treatment for Repeated Flares

Effisayil® REP:An Open-label, Multicenter, Single-arm, Post-marketing Trial (in Select Countries) to Evaluate Efficacy and Safety and the Impact of Immunogenicity on Efficacy, Safety, and Pharmacokinetics of Spesolimab i.v. in Treatment of Patients With Generalized Pustular Psoriasis (GPP) Presenting With a Recurrent Flare Following Their Initial GPP Flare Treatment With Spesolimab i.v.

This study is open to adults with a serious skin disease called generalized pustular psoriasis (GPP) who have repeated flares of GPP. The purpose of this study is to find out whether a medicine called spesolimab helps people with repeated flares of GPP.

Participants are given a single dose of spesolimab as an infusion into a vein on the first day of an outbreak of GPP. They may be given a second dose 1 week later if doctors think it is helpful. They are also treated for additional GPP flares.

During the time of the study, doctors regularly examine participants' skin for signs of GPP to see how well the treatment works and take blood samples. The doctors also regularly check participants' health and take note of any unwanted effects.

Study Overview

• Status: Active, not recruiting
• Conditions: Generalized Pustular Psoriasis
• Intervention / Treatment: Drug: Spesolimab

• Study Type: Interventional
• Enrollment (Actual): 42
• Phase: Phase 4

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Liverpool, New South Wales, Australia, 2170
      • Liverpool Hospital
    • Westmead, New South Wales, Australia, 2145
      • Westmead Hospital
• Belgium
  • Brussels, Belgium, 1200
    • Cliniques Universitaires Saint-Luc
• Brazil
  • Curitiba, Brazil, 80440-220
    • Hospital Universitario Evangelico Mackenzie
  • Santo André, Brazil, 09060-870
    • Faculdade de Medicina do ABC
• China
  • Guangzhou, China, 510091
    • Southern Medical University Dermatology Hospital
  • Shanghai, China, 200000
    • Shanghai skin disease hospital
  • Shanghai, China, 200072
    • Shanghai Tenth People's Hospital
• France
  • Chambray-lès-Tours, France, 37170
    • HOP Trousseau
  • Paris, France, 75010
    • HOP Saint-Louis
• Germany
  • Frankfurt am Main, Germany, 60596
    • Universitatsklinikum Frankfurt
  • München, Germany, 80337
    • Klinikum der Universität München AÖR
• India
  • Bikaner, India, 334003
    • SP medical college and associated group of hospitals
  • Nagpur, India, 440019
    • Nkp Salve Institute of Medical Sciences and Lata Mangeshkar Hospital
• Italy
  • Brescia, Italy, 25123
    • Asst Degli Spedali Civili Di Brescia
• Malaysia
  • Batu Caves, Malaysia, 68100
    • Hospital Selayang
  • Georgetown Pulau Pinang, Malaysia, 10990
    • Hospital Pulau Pinang-Pulau Pinang-21953
  • Johor Bahru, Malaysia, 80100
    • Hospital Sultanah Aminah
  • Johor Bahru, Malaysia, 81100
    • Hospital Sultan Ismail
  • Kuala Lumpur, Malaysia, 50586
    • Hospital Kuala Lumpur
  • Putrajaya, Malaysia, 62250
    • Hospital Putrajaya
  • Selangor Darul Ehsan, Malaysia, 47500
    • Sunway Medical Centre
• Singapore
  • Singapore, Singapore, 119228
    • National University Hospital-Singapore-42005
• South Africa
  • Mthatha, Eastern Cape, South Africa, 5099
    • Nelson Mandela Academic Clinical Research Unit (NeMACRU)
• South Korea
  • Seoul, South Korea, 03080
    • Seoul National University Hospital
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
  • Madrid, Spain, 28034
    • Hospital Universitario Ramón y Cajal
• Taiwan
  • Taipei, Taiwan, 100
    • National Taiwan University Hospital
  • Taoyuan, Taiwan, 330
    • Chang Gung Memorial Hospital, Linkou
• Thailand
  • Khon Kaen, Thailand, 40000
    • Srinagarind Hospital
  • Muang Chiang Mai, Thailand, 50200
    • Maharat Nakhonchiangmai Hospital
• Tunisia
  • Sfax, Tunisia, 1053
    • Hedi Chaker Hospital, Department of Dermatology
  • Sousse, Tunisia, 4000
    • Farhat Hached Hospital
• United States
  • California
    • Irvine, California, United States, 92697
      • University of California Irvine
  • North Dakota
    • Fargo, North Dakota, United States, 58103
      • Red River Research Partners, LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation sub-score of 0 or 1 and a known and documented history of Generalized Pustular Psoriasis (GPP) (per European Rare And Severe Psoriasis Expert Network - ERASPEN - criteria), regardless of Interleukin 36 Receptor Antagonist (IL-36RN) gene mutation status or Patients with a GPP flare and a known and documented history of GPP (per ERASPEN criteria) regardless of IL-36RN gene mutation status.
• Patients must have a history of frequent GPP flares in the past
• Male or female patients, aged ≥18 years (if local legislation for age of consent differs, then local legislation will be followed) at screening
• Signed and dated written informed consent prior to admission to the trial in accordance with International Conference on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to start of any screening procedures
• Women of childbearing potential (WOCBP) 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

Exclusion Criteria:

• Drug-triggered Acute Generalized Exanthematous Pustulosis (AGEP)
• Patients with primary plaque psoriasis vulgaris without presence of pustules or with pustules that are restricted to psoriatic plaques
• Patients with primary erythrodermic psoriasis vulgaris
• Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome
• 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 acute renal failure
• Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold upper limit normal (ULN) elevation in Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin
• Presence of acute demyelinating neuropathy
• Treatment with any drug considered likely to interfere with the safe conduct of the trial, as assessed by the investigator
• Further exclusion criteria apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GPP Patients; Generalized Pustular Psoriasis (GPP) patients with a recurrent flare following initial GPP flare treatment with intravenous (i.v.) spesolimab.	Drug: Spesolimab; Spesolimab intravenous (i.v.) infusion; Other Names: Spevigo®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Achievement of a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation sub-score of 0 indicating no visible pustules at Week 1	Up to week 1

Secondary Outcome Measures

Outcome Measure	Time Frame
Achievement of a GPPGA pustulation sub-score of 0 or 1, with a ≥2-point reduction from baseline at Week 1	Up to week 1

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): October 22, 2023
• Primary Completion (Estimated): July 23, 2026
• Study Completion (Estimated): August 14, 2026

Study Registration Dates

• First Submitted: August 23, 2023
• First Submitted That Met QC Criteria: August 23, 2023
• First Posted (Actual): August 28, 2023

Study Record Updates

• Last Update Posted (Actual): May 27, 2026
• Last Update Submitted That Met QC Criteria: May 26, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases, Papulosquamous; Skin Diseases; Skin and Connective Tissue Diseases; Psoriasis; spesolimab
• Other Study ID Numbers: 1368-0120; 2022-502128-38-00 (Registry Identifier: CTIS); U1111-1291-3088 (Registry Identifier: WHO International Clinical Trials Registry Platform (ICTRP))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD Plan Description

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).

https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing

• IPD Sharing Time Frame: After structured results have been posted, all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication.

IPD Sharing Access Criteria

For study documents - upon signing of a 'Document Sharing Agreement'.

For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No