An Expanded Access Trial in Japan to Provide Spesolimab to People With a Flare-up in Generalized Pustular Psoriasis Who Have no Other Treatment Options

Multi-centre, Open-label, Expanded Access Trial of Spesolimab i.v. in Patients With Generalized Pustular Psoriasis (GPP) Presenting With a Flare

This Expanded Access trial in Japan is open to people with a serious skin disease called Generalized Pustular Psoriasis (GPP). This program provides a medicine called spesolimab to people with a GPP flare-up who have no alternative treatment options.

Participants get a single infusion of spesolimab into a vein. They can get another spesolimab infusion one week after the first infusion if the doctors think it is helpful.

Participants are in the program for about 4 months and visit the study site about 5 to 6 times. The doctors regularly check participants' health and take note of any unwanted effects.

Study Overview

• Status: Completed
• Conditions: Generalized Pustular Psoriasis
• Intervention / Treatment: Drug: spesolimab

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Aichi, Nagoya, Japan, 467-8602
    • Nagoya City University Hospital
  • Fukuoka, Fukuoka, Japan, 814-0180
    • Fukuoka University Hospital
  • Kagoshima, Kagoshima, Japan, 890-8520
    • Kagoshima University Hospital
  • Mie, Tsu, Japan, 514-8507
    • Mie University Hospital
  • Miyagi, Sendai, Japan, 980-8574
    • Tohoku University Hospital
  • Saitama, Iruma-gun, Japan, 350-0495
    • Saitama Medical University Hospital
  • Tochigi, Shimotsuke, Japan, 329-0498
    • Jichi Medical University Hospital
  • Tokyo, Itabashi-ku, Japan, 173-8606
    • Teikyo University Hospital
  • Tokyo, Shinjuku-ku, Japan, 160-0023
    • Tokyo Medical University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria

• Diagnosis of GPP confirmed based on the Japanese Dermatological Association (JDA) guidelines for the management and treatment of GPP.
• Patient is experiencing a flare, defined as new or worsening of widespread eruption of sterile macroscopically visible pustules, with or without systemic inflammation, as assessed by the investigator.
• Male or female patients, aged 18 to 75 years at time of enrollment. Women of childbearing potential (WOCBP) must be willing and able to use a highly effective method of birth control per International Council for Harmonization (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 patient information.
• Signed and dated written informed consent in accordance with International Council for Harmonization-Good Clinical Practice (ICH-GCP) and local legislation prior to admission to the trial.
• No satisfactory authorised alternative therapy exists, as assessed by the investigator.

Exclusion criteria

• Women who are pregnant, nursing, or who plan to become pregnant while in the trial.

  -- Women who stop nursing before study drug administration do not need to be excluded from participating; they should refrain from breastfeeding for 16 weeks after the last spesolimab infusion.

• Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Level of Normal (ULN) elevation in Aspartate Transaminase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin.
• Active systemic infections (fungal and bacterial disease) during the last 2 weeks prior to drug administration, as assessed by the investigator.
• Increased risk of infectious complications (e.g. recent pyogenic infection, any congenital or acquired immunodeficiency (e.g. Human Immunodeficiency Virus (HIV)), past organ or stem cell transplantation), as assessed by the investigator.

• Relevant chronic or acute infections, including active tuberculosis (TB), HIV infection or viral hepatitis at the time of drug administration.

  • Patients should be evaluated for TB infection prior to initiating treatment with spesolimab.
  • Anti-TB therapy should be considered, in accordance with local guidelines, prior to initiating spesolimab in patients with latent TB or a history of TB.
• History of allergy / hypersensitivity to systemically administered spesolimab or its excipients.
• Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal or squamous cell carcinoma of the skin or in situ carcinoma of uterine cervix.
• Immediate life-threatening flare of GPP requiring intensive care treatment according to the investigator's judgement. Life-threatening complications include cardiovascular / cytokine driven shock, pulmonary distress syndrome, or renal failure.

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: Spesolimab	Drug: spesolimab; solution for infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Treatment Emergent Adverse Events (TEAEs)	Number of patients with any treatment emergent adverse events (TEAEs) is reported. An adverse events (AE) was defined as any untoward medical occurrence in a patient or clinical investigation subject administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE that started before first drug intake and deteriorated under treatment were also considered as 'treatment-emergent'.	From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Treatment Emergent Serious Adverse Events (SAEs)	Number of patients with treatment emergent serious adverse events (SAEs) is reported. A serious adverse event (SAE) was defined as any AE which fulfils at least one of the following criteria: results in death,; is life-threatening, which refers to an event in which the patient was at risk of death at the time of the event;; requires inpatient hospitalisation or prolongation of existing hospitalisation,; results in persistent or significant disability or incapacity,; is a congenital anomaly / birth defect,; is deemed serious for any other reason if it is an important medical event when based on appropriate medical judgement which may jeopardise the patient and may require medical or surgical intervention to prevent one of the other outcomes listed in the above definitions.; An event that possibly leads to disability will be handled as 'deemed serious for any other reason' and, therefore, reported as an SAE.	From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESIs)	Number of patients with treatment emergent adverse events of special interest (AESIs) is reported. The term adverse events of special interest (AESI) relates to any specific adverse event (AE) that has been identified at the project level as being of particular concern for prospective safety monitoring and safety assessment within this trial, e.g. the potential for AEs based on knowledge from other compounds in the same class. The following are considered as AESIs: Potential Severe Drug Induced Liver Injury (DILI); Systemic hypersensitivity reactions including infusion reactions and anaphylactic reaction; Severe infections (according to RCTC grading in the ISF); Opportunistic and mycobacterium tuberculosis infections; Peripheral neuropathy.	From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): February 17, 2022
• Primary Completion (Actual): March 20, 2023
• Study Completion (Actual): March 20, 2023

Study Registration Dates

• First Submitted: January 11, 2022
• First Submitted That Met QC Criteria: January 11, 2022
• First Posted (Actual): January 20, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 10, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases, Papulosquamous; Skin Diseases; Psoriasis
• Other Study ID Numbers: 1368-0073

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).

For more details refer to: https://www.mystudywindow.com/msw/datatransparency

• IPD Sharing Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program.
• 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: No
• Studies a U.S. FDA-regulated device product: No