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

• ClinicalTrials.gov Identifier: NCT05239039
• Recruitment Status: Completed
• First Posted: February 14, 2022
• Last Update Posted: January 12, 2024
• Sponsor: Boehringer Ingelheim
• Information provided by (Responsible Party): Boehringer Ingelheim

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Generalized Pustular Psoriasis	Drug: spesolimab	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 39 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Multi-centre, Open-label, Expanded Access Program of 900mg Intravenous (i.v.) Spesolimab in Patients With Generalized Pustular Psoriasis (GPP) Presenting With a Flare
• Actual Study Start Date: March 15, 2022
• Actual Primary Completion Date: July 17, 2023
• Actual Study Completion Date: July 17, 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: Patients with Generalized Pustular Psoriasis (GPP) presenting with a flare	Drug: spesolimab; spesolimab

Outcome Measures

Primary Outcome Measures :

1. Occurrence of Treatment Emergent Adverse Events (AEs) [ Time Frame: up to 17 weeks ]

Secondary Outcome Measures :

1. Occurrence of Treatment Emergent Serious Adverse Events (SAEs) [ Time Frame: up to 17 weeks ]
2. Occurrence of Treatment Emergent Adverse Events of Special Interest (AESIs) [ Time Frame: up to 17 weeks ]

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

China

Beijing Friendship Hospital

Beijing, China, 100050

West China Hospital

Chengdu, China, 610041

Southern Medical University Dermatology Hospital

Guangzhou, China, 510091

The Second Affiliated Hospital Zhejiang University School of Medicine

Hangzhou, China, 310009

Hangzhou Third People's Hospital

Hangzhou, China, 310013

Shandong Provincial Hospital of Dermatology

Jinan, China, 250063

Dermatology Hospital, Chinese Academy of Medical Sciences

Nanjing, China, 210000

Shanghai Skin Disease Hospital

Shanghai, China, 200000

The First Hospital of China Medical University

Shenyang, China, 110001

The University of Hong Kong-Shenzhen Hospital

Shenzhen, China, 518053

Wuhan Union Hospital

Wuhan, China, 430022

Sponsors and Collaborators

Boehringer Ingelheim

More Information

Additional Information:

Related Info 

• Responsible Party: Boehringer Ingelheim
• ClinicalTrials.gov Identifier: NCT05239039
• Other Study ID Numbers: 1368-0077
• First Posted: February 14, 2022
• Last Update Posted: January 12, 2024
• Last Verified: January 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Once the criteria in section 'time frame' are fulfilled, researchers can use the 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". Furthermore, researchers can 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.
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Clinical Study Report (CSR)
• 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.
• 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.
• URL: https://www.mystudywindow.com/msw/datasharing

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Psoriasis; Skin Diseases, Papulosquamous; Skin Diseases