Clinical trial NCT05239039: 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
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Generalized Pustular Psoriasis
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2022-02-14
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Occurrence of Treatment Emergent Adverse Events (AEs)
timeFrame: 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 outcomes:
measure: Occurrence of Treatment Emergent Serious Adverse Events (SAEs)
timeFrame: 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.
measure: Occurrence of Treatment Emergent Adverse Events of Special Interest (AESIs)
timeFrame: 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.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Friendship Hospital
city: Chengdu
country: China
facility: West China Hospital
city: Guangzhou
country: China
facility: Southern Medical University Dermatology Hospital
city: Hangzhou
country: China
facility: The Second Affiliated Hospital Zhejiang University School of Medicine
city: Hangzhou
country: China
facility: Hangzhou Third People's Hospital
city: Jinan
country: China
facility: Shandong Provincial Hospital of Dermatology
city: Nanjing
country: China
facility: Dermatology Hospital, Chinese Academy of Medical Sciences
city: Shanghai
country: China
facility: Shanghai Skin Disease Hospital
city: Shenyang
country: China
facility: The First Hospital of China Medical University
city: Shenzhen
country: China
facility: The University of Hong Kong-Shenzhen Hospital
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
city: Wuhan
country: China
facility: Wuhan Union Hospital
Study officials:
References: