An Expanded Access Program in China 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 Program of 900mg Intravenous (i.v.) Spesolimab in Patients With Generalized Pustular Psoriasis (GPP) Presenting With a Flare

This Expanded Access Program in China 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. This means that no therapy exists and participation in a clinical study is not possible.

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 times. Participants who benefit from the treatment during that time may repeat the treatment in case they experience a new GPP flare-up. 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): 39
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100050
    • Beijing Friendship Hospital
  • Chengdu, China, 610041
    • West China Hospital
  • Guangzhou, China, 510091
    • Southern Medical University Dermatology Hospital
  • Hangzhou, China, 310009
    • The Second Affiliated Hospital Zhejiang University School of Medicine
  • Hangzhou, China, 310013
    • Hangzhou Third people's Hospital
  • Jinan, China, 250063
    • Shandong Provincial Hospital of Dermatology
  • Nanjing, China, 210000
    • Dermatology Hospital, Chinese Academy of Medical Sciences
  • Shanghai, China, 200000
    • Shanghai skin disease hospital
  • Shenyang, China, 110001
    • The First Hospital of China Medical University
  • Shenzhen, China, 518053
    • The University of Hong Kong-Shenzhen Hospital
  • Tianjin, China, 30052
    • Tianjin Medical University General Hospital
  • Wuhan, China, 430022
    • Wuhan Union 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 Generalized Pustular Psoriasis (GPP), consistent with European Rare and Severe Psoriasis Expert Network (ERASPEN) criteria, defined as primary, sterile, macroscopically visible pustules on non-acral skin (excluding cases where pustulation is restricted to psoriatic plaques). GPP can occur with or without systemic inflammation, with or without plaque-type psoriasis, and be either relapsing (>1 episode) or persistent (>3 months).
• 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 treating physician.
• Male or female patients, aged 18 to 75 years at time of enrolment. 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 program.
• No satisfactory authorized alternative therapy exists, as assessed by the treating physician.

Exclusion criteria

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

  -- 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 Limit 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 treating physician.
• 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 treating physician.

• 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: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Spesolimab single dose treatment; Patients with Generalized Pustular Psoriasis (GPP) presenting a flare received a single intravenous dose of 900 milligrams of spesolimab.	Drug: spesolimab; spesolimab
Experimental: Spesolimab double dose treatment; Patients with Generalized Pustular Psoriasis (GPP) presenting a flare received a single intravenous dose of 900 milligrams of spesolimab. One week after the initial dose, patients received a second intravenous single dose of 900 mg of spesolimab due to persistence of flare symptoms.	Drug: spesolimab; spesolimab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Treatment Emergent Adverse Events (AEs)	This outcome measured the number of patients with any treatment-emergent adverse event (AE). Treatment-emergent AEs are untoward medical events that appear or worsen during treatment.	From initial drug administration to: end of study treatment + residual effect period or end of study visit. Up to 16 weeks for the single dose group and up to 17 weeks for the double dose group.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Treatment Emergent Serious Adverse Events (SAEs)	This outcome measured the number of patients with serious adverse events (SAEs). SAEs are untoward medical occurrences that result in death, are life threatening, require inpatient hospitalisation, require prolongation of existing hospitalisation, result in persistent or significant disability/incapacity, result in a congenital anomaly/birth defect, or are deemed serious for any other medically important reason.	From initial drug administration to: end of study treatment + residual effect period or end of study visit. Up to 16 weeks for the single dose group and up to 17 weeks for the double dose group.
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESIs)	This outcome measured the number of patients with any treatment-emergent adverse event of special interest (AESIs). AESIs relates to any specific AE that has been identified at the project level as being of particular concern for prospective safety monitoring and safety assessment within this program. Potential severe DILIs (drug-induced liver injury), systemic hypersensitivity reactions, severe infections, opportunistic and mycobacterium tuberculosis infections, and peripheral neuropathy were considered as AESIs.	From initial drug administration to: end of study treatment + residual effect period or end of study visit. Up to 16 weeks for the single dose group and up to 17 weeks for the double dose group.

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2022
• Primary Completion (Actual): July 17, 2023
• Study Completion (Actual): July 17, 2023

Study Registration Dates

• First Submitted: February 3, 2022
• First Submitted That Met QC Criteria: February 3, 2022
• First Posted (Actual): February 14, 2022

Study Record Updates

• Last Update Posted (Estimated): October 20, 2025
• Last Update Submitted That Met QC Criteria: October 13, 2025
• Last Verified: October 1, 2025

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: 1368-0077

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
• product manufactured in and exported from the U.S.: No