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

• ClinicalTrials.gov Identifier: NCT05200247
• Recruitment Status: Completed
• First Posted: January 20, 2022
• Last Update Posted: January 12, 2024
• Sponsor: Boehringer Ingelheim
• Information provided by (Responsible Party): Boehringer Ingelheim

Study Description

Brief Summary:

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.

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

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Multi-centre, Open-label, Expanded Access Trial of Spesolimab i.v. in Patients With Generalized Pustular Psoriasis (GPP) Presenting With a Flare
• Actual Study Start Date: February 17, 2022
• Actual Primary Completion Date: March 20, 2023
• Actual Study Completion Date: March 20, 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: Spesolimab	Drug: spesolimab; solution for infusion

Outcome Measures

Primary Outcome Measures :

1. Occurrence of treatment emergent adverse events (TEAEs) [ Time Frame: up to 6.3 months. ]

Secondary Outcome Measures :

1. Occurrence of treatment emergent serious adverse events (SAEs) [ Time Frame: up to 6.3 months. ]
2. Occurrence of treatment emergent adverse events of special interest (AESIs) [ Time Frame: up to 6.3 months. ]

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 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.

Contacts and Locations

Locations

Japan

Nagoya City University Hospital

Aichi, Nagoya, Japan, 467-8602

Fukuoka University Hospital

Fukuoka, Fukuoka, Japan, 814-0180

Kagoshima University Hospital

Kagoshima, Kagoshima, Japan, 890-8520

Mie University Hospital

Mie, Tsu, Japan, 514-8507

Tohoku University Hospital

Miyagi, Sendai, Japan, 980-8574

Saitama Medical University Hospital

Saitama, Iruma-gun, Japan, 350-0495

Jichi Medical University Hospital

Tochigi, Shimotsuke, Japan, 329-0498

Teikyo University Hospital

Tokyo, Itabashi-ku, Japan, 173-8606

Tokyo Medical University Hospital

Tokyo, Shinjuku-ku, Japan, 160-0023

Sponsors and Collaborators

Boehringer Ingelheim

More Information

Additional Information:

Related Info 

• Responsible Party: Boehringer Ingelheim
• ClinicalTrials.gov Identifier: NCT05200247
• Other Study ID Numbers: 1368-0073
• First Posted: January 20, 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