Effisayil™ ON: A Study to Test Long-term Treatment With Spesolimab in People With Generalized Pustular Psoriasis Who Took Part in a Previous Study

Effisayil™ ON: An Open-label, Long Term Extension Study to Assess the Safety and Efficacy of Spesolimab Treatment in Patients With Generalized Pustular Psoriasis (GPP)

This study is open to people with generalized pustular psoriasis (GPP). People can only take part if they have completed treatment in a previous study with spesolimab (1368-0013 or 1368-0027).

The goal of this study is to find out how well people with GPP tolerate long-term treatment with spesolimab. The study also tests whether spesolimab helps improve GPP symptoms and how quickly the symptoms improve after a flare-up.

Every participant gets spesolimab for almost 5 years (252 weeks). Depending on their symptoms and whether they had a GPP flare during the previous trial, they get spesolimab every few weeks. When participants have a GPP flare during this trial, they get spesolimab as an infusion into a vein.

Participants visit their doctors regularly. During these visits, the doctors collect information on any health problems of the participants. To assess the study endpoints, doctors regularly check participants' skin.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 131
• Phase: Phase 2

Contacts and Locations

Study Locations

• Argentina
  • Caba, Argentina, C1199ABD
    • Hospital Italiano de Buenos Aires
• Belgium
  • Bruxelles, Belgium, 1200
    • Brussels - UNIV Saint-Luc
• Chile
  • Vitacura, Chile, 7640881
    • Clinica Dermacross S.A.
• China
  • Guangzhou, China, 510288
    • Sun yet-sen Memorial Hospital, Sun yet-sen Univesity
  • Hangzhou, China, 310009
    • The Second Affiliated Hospital Zhejiang University School of Medicine
  • Shanghai, China, 200000
    • Shanghai skin disease hospital
  • Shanghai, China, 200040
    • Huashan Hospital, Fudan University
  • Xi'an, China, 710004
    • Second Affiliated Hospital of Xi'an JiaoTong University
• France
  • Bordeaux, France, 33000
    • HOP Saint-André
  • Nice, France, 06200
    • HOP l'Archet
  • Paris, France, 75010
    • HOP Saint-Louis
  • Reims, France, 51092
    • HOP Robert Debré
• Germany
  • Bad Bentheim, Germany, 48455
    • Fachklinik Bad Bentheim
  • Berlin, Germany, 10117
    • Charite - Universitatsmedizin Berlin
  • Bonn, Germany, 53127
    • Universitätsklinikum Bonn AöR
  • Frankfurt am Main, Germany, 60596
    • Universitatsklinikum Frankfurt
  • München, Germany, 80337
    • Klinikum der Universität München - Campus Innenstadt
  • Münster, Germany, 48149
    • Westfälische Wilhelms-Universität Münster
  • Oldenburg, Germany, 26133
    • Klinikum Oldenburg AöR
• Italy
  • Rozzano (MI), Italy, 20089
    • Istituto Clinico Humanitas
• Japan
  • Aichi, Nagoya, Japan, 467-8602
    • Nagoya City University Hospital
  • Ibaraki, Inashiki-gun, Japan, 300-0395
    • Tokyo Medical University Ibaraki Medical Center
  • Saitama, Iruma-gun, Japan, 350-0495
    • Saitama Medical University Hospital
  • Tokyo, Hachioji, Japan, 193-0998
    • Tokyo Medical University Hachioji Medical Center
• Korea, Republic of
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital
• Malaysia
  • George Town, Malaysia, 10990
    • Hospital Pulau Pinang
  • Ipoh, Malaysia, 30450
    • Hospital Raja Permaisuri Bainun
  • Johor Bahru, Malaysia, 80100
    • Hospital Sultanah Aminah
  • Johor Bahru, Malaysia, 81100
    • Hospital Sultan Ismail
  • Kota Kinabalu, Malaysia, 88586
    • Queen Elizabeth Hospital
  • Kuala Lumpur, Malaysia, 50586
    • Hospital Kuala Lumpur
  • Kuching, Sarawak, Malaysia, 93586
    • Sarawak General Hospital
  • Muar, Malaysia, 84000
    • Hospital Pakar Sultanah Fatimah
  • Selangor, Malaysia, 68100
    • Hospital Selayang
• Mexico
  • Monterrey, Mexico, 64460
    • Hospital Universitario Dr Jose Eleuterio Gonzalez
• Philippines
  • Davao City, Philippines, 8000
    • Southern Philippines Medical Center
  • Iloilo City, Iloilo, Philippines, 5000
    • Iloilo Doctors Hospital
  • Makati City, Philippines, 1229
    • Center for Skin Research, Testing and Product Development
• Russian Federation
  • Chelyabinsk, Russian Federation, 454048
    • SBHI Chelyabinsk Reg.Clin.Derma.Dispen.
  • Kazan, Russian Federation, 420111
    • LLC "Medical Center Azbuka Zdorovia"
  • Kirov, Russian Federation, 610035
    • FSBEI HE "Kirov State Medical University"
  • Saratov, Russian Federation, 410028
    • Saratov State Med.Univ.n.a.Razumovskogo
  • St. Petersburg, Russian Federation, 190123
    • LLC Skin Disease Clinic of Pier Volkenstein, St. Petersburg
• Spain
  • Esplugues Del Llobregat, Spain, 08950
    • Hospital Sant Joan de Deu
• Taiwan
  • Linkou, Taiwan, 333
    • Chang Gung Medical Foundation (CGMF) - Linkou Bran
  • Taipei, Taiwan, 10002
    • National Taiwan University Hospital
• Thailand
  • Bangkok, Thailand, 10400
    • Institute of Dermatology
  • Bangkok, Thailand, 10400
    • Ramathibodi hospital
• Tunisia
  • Sousse, Tunisia, 4000
    • Farhat Hached Hospital
  • Tunis, Tunisia, 1007
    • La Rabta Hospital
  • Tunisia, Tunisia, 1053
    • Hedi Chaker Hospital, Department of Dermatology
• Turkey
  • Istanbul, Turkey, 34890
    • Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi
• United States
  • Michigan
    • Auburn Hills, Michigan, United States, 48326
      • Oakland Hills Dermatology
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai
• Vietnam
  • Ha Noi, Vietnam, 10000
    • National Hospital of Dermatology and Venereology
  • Ho Chi Minh, Vietnam, 70000
    • HCMC Hospital of Dermato-Venereology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female patients who have completed the treatment period without premature discontinuation in the previous spesolimab trial and are willing and able to continue treatment in the current trial
• 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. A list of contraception methods meeting these criteria is provided in Section 4.2.2.3 as well as in the patient information. 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.
• Signed and dated written informed consent and assent for the current trial 1368-0025, in accordance with ICH-GCP and local legislation prior to admission to the current trial

Exclusion Criteria:

• Evidence of flare symptoms of moderate/severe intensity at screening.
• Treatment with any restricted medication as specified in the protocol, or any drugs considered by the investigator likely to interfere with the safe conduct of the study since the last visit of the previous spesolimab trial and during the screening period for the current trial, with the exception of methotrexate, cyclosporine, or retinoids started following rescue treatment for GPP flare in trial 1368-0027.
• Severe, progressive, or uncontrolled hepatic disease, defined as >3- fold Upper Limit of Normal (ULN) elevation in Aspartate Transaminase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2- fold ULN elevation in total bilirubin.
• Patients with congestive heart disease, as assessed by the investigator.
• Relevant chronic or acute infections including human immunodeficiency virus (HIV) or viral hepatitis. A patient can be re-screened if the patient was treated and is cured from acute infection.

• Active or Latent tuberculosis (TB):

  • Patients with active tuberculosis should be excluded
  • Patients will be screened with Interferon Gamma Release Assay (IGRA) such as QuantiFERON®-TB-Gold Plus or T-spot®. Patients with positive IGRA (indicating active or latent tuberculosis) are excluded unless they have completed treatment for active or latent tuberculosis per investigator discretion, at the time of screening.
  • Patients with indeterminate QuantiFERON®-TB-Gold Plus or invalid/borderline T-spot® may be retested with IGRA (once) or Tuberculin Skin test (TST).
  • TST or any alternative test/procedure (as per local standards) to rule out TB can be performed if IGRA is not available or indeterminate. A TST reaction ≥10mm (≥5mm if receiving ≥15mg/d prednisone or other immunosuppressant) is considered positive. Patients with a positive TST are excluded unless they have completed treatment as above.
• History of allergy/hypersensitivity to a systemically administered trial medication agent or its excipients.
• Any documented active or suspected malignancy at screening, except appropriately treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin or in situ carcinoma of uterine cervix.

Further exclusion criteria apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Spesolimab (every 6 weeks)	Drug: Spesolimab; Solution for injection; Drug: Spesolimab; Solution for infusion
Experimental: Spesolimab (every 12 weeks)	Drug: Spesolimab; Solution for injection; Drug: Spesolimab; Solution for infusion
Experimental: Spesolimab (every 4 weeks)	Drug: Spesolimab; Solution for injection; Drug: Spesolimab; Solution for infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment	Up to 252 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The reoccurrence of a GPP flare defined by GPPGA	The total GPPGA score ranges from 0 to 4 with a higher score indicating a higher grade of inflammation.	Up to 252 Weeks
Time to first achievement of a GPPGA score of 0 or 1 (Patients received flare rescue Treatment)		Up to 252 Weeks
A GPPGA pustulation sub-score of 0 indicating no visible pustules, by visit		Up to 252 Weeks
Change from baseline in Psoriasis Symptom Scale (PSS) score, by visit	The PSS score ranges from None to Very Severe with a higher score indicating a higher severity of psoriasis symptom.	Up to 252 Weeks

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): May 27, 2019
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): January 20, 2028

Study Registration Dates

• First Submitted: March 21, 2019
• First Submitted That Met QC Criteria: March 21, 2019
• First Posted (Actual): March 22, 2019

Study Record Updates

• Last Update Posted (Actual): January 5, 2024
• Last Update Submitted That Met QC Criteria: January 3, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Skin Diseases, Papulosquamous; Psoriasis
• Other Study ID Numbers: 1368-0025; 2018-003080-56 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: After the study is completed and the primary manuscript is accepted for publishing, researchers can use this following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Also, Researchers can use the following link https://www.mystudywindow.com/msw/datasharing to find information in order to request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website. The data shared are the raw clinical study data sets.
• IPD Sharing Time Frame: After 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 both the independent review panel and the sponsor, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a 'Data Sharing 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