Clinical trial NCT05200247: 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
Official title: Multi-centre, Open-label, Expanded Access Trial of Spesolimab i.v. 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: 2022-01-20
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Occurrence of Treatment Emergent Adverse Events (TEAEs)
timeFrame: From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Secondary outcomes:
measure: Occurrence of Treatment Emergent Serious Adverse Events (SAEs)
timeFrame: From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
measure: Occurrence of Treatment Emergent Adverse Events of Special Interest (AESIs)
timeFrame: From first administration of study drug until last administration of study drug + 16 weeks of follow up, up to 6.3 months.
Other outcomes:
Locations:
city: Aichi, Nagoya
country: Japan
facility: Nagoya City University Hospital
city: Fukuoka, Fukuoka
country: Japan
facility: Fukuoka University Hospital
city: Kagoshima, Kagoshima
country: Japan
facility: Kagoshima University Hospital
city: Mie, Tsu
country: Japan
facility: Mie University Hospital
city: Miyagi, Sendai
country: Japan
facility: Tohoku University Hospital
city: Saitama, Iruma-gun
country: Japan
facility: Saitama Medical University Hospital
city: Tochigi, Shimotsuke
country: Japan
facility: Jichi Medical University Hospital
city: Tokyo, Itabashi-ku
country: Japan
facility: Teikyo University Hospital
city: Tokyo, Shinjuku-ku
country: Japan
facility: Tokyo Medical University Hospital
Study officials:
References: