Clinical trial NCT06013969: A Study to Test Whether Spesolimab Helps People With Generalized Pustular Psoriasis (GPP) Who Need Treatment for Repeated Flares
Official title: Effisayil® REP:An Open-label, Multicenter, Single-arm, Post-marketing Trial (in Select Countries) to Evaluate Efficacy and Safety and the Impact of Immunogenicity on Efficacy, Safety, and Pharmacokinetics of Spesolimab i.v. in Treatment of Patients With Generalized Pustular Psoriasis (GPP) Presenting With a Recurrent Flare Following Their Initial GPP Flare Treatment With Spesolimab i.v.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
Conditions: Generalized Pustular Psoriasis
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2023-08-28
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is open to adults with a serious skin disease called generalized pustular psoriasis (GPP) who have repeated flares of GPP. The purpose of this study is to find out whether a medicine called spesolimab helps people with repeated flares of GPP.

Participants are given a single dose of spesolimab as an infusion into a vein on the first day of an outbreak of GPP. They may be given a second dose 1 week later if doctors think it is helpful. They are also treated for additional GPP flares.

During the time of the study, doctors regularly examine participants' skin for signs of GPP to see how well the treatment works and take blood samples. The doctors also regularly check participants' health and take note of any unwanted effects.


Eligibility criteria: Inclusion Criteria:

* Patients with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation sub-score of 0 or 1 and a known and documented history of Generalized Pustular Psoriasis (GPP) (per European Rare And Severe Psoriasis Expert Network - ERASPEN - criteria), regardless of Interleukin 36 Receptor Antagonist (IL-36RN) gene mutation status or Patients with a GPP flare and a known and documented history of GPP (per ERASPEN criteria) regardless of IL-36RN gene mutation status.
* Patients must have a history of frequent GPP flares in the past
* Male or female patients, aged ≥18 years (if local legislation for age of consent differs, then local legislation will be followed) at screening
* Signed and dated written informed consent prior to admission to the trial in accordance with International Conference on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to start of any screening procedures
* Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly

Exclusion Criteria:

* Drug-triggered Acute Generalized Exanthematous Pustulosis (AGEP)
* Patients with primary plaque psoriasis vulgaris without presence of pustules or with pustules that are restricted to psoriatic plaques
* Patients with primary erythrodermic psoriasis vulgaris
* Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome
* Immediate life-threatening flare of GPP or requiring intensive care treatment, according to the investigator's judgement. Life-threatening complications mainly include, but are not limited to, cardiovascular/cytokine driven shock, pulmonary distress syndrome, or acute renal failure
* Severe, progressive, or uncontrolled hepatic disease, defined as \>3-fold upper limit normal (ULN) elevation in Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or \>2-fold ULN elevation in total bilirubin
* Presence of acute demyelinating neuropathy
* Treatment with any drug considered likely to interfere with the safe conduct of the trial, as assessed by the investigator
* Further exclusion criteria apply.

Primary outcomes:
measure: Achievement of a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation sub-score of 0 indicating no visible pustules at Week 1
timeFrame: Up to week 1
Secondary outcomes:
measure: Achievement of a GPPGA pustulation sub-score of 0 or 1, with a ≥2-point reduction from baseline at Week 1
timeFrame: Up to week 1
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: Fargo
country: United States
facility: Red River Research Partners, LLC
state: North Dakota
city: Liverpool
country: Australia
facility: Liverpool Hospital
state: New South Wales
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Bruxelles
country: Belgium
facility: Brussels - UNIV Saint-Luc
city: Curitiba
country: Brazil
facility: Hospital Universitario Evangelico Mackenzie
city: Santo André
country: Brazil
facility: Faculdade de Medicina do ABC
city: Guangzhou
country: China
facility: Southern Medical University Dermatology Hospital
city: Shanghai
country: China
facility: Shanghai Skin Disease Hospital
city: Shanghai
country: China
facility: Shanghai Tenth People's Hospital
city: Chambray-lès-Tours
country: France
facility: HOP Trousseau
city: Paris
country: France
facility: HOP Saint-Louis
city: Frankfurt am Main
country: Germany
facility: Universitätsklinikum Frankfurt
city: München
country: Germany
facility: Klinikum der Universität München AÖR
city: Bikaner
country: India
facility: SP medical college and associated group of hospitals
city: Nagpur
country: India
facility: Nkp Salve Institute of Medical Sciences and Lata Mangeshkar Hospital
city: Brescia
country: Italy
facility: ASST degli Spedali Civili di Brescia
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Batu Caves
country: Malaysia
facility: Hospital Selayang
city: Georgetown Pulau Pinang
country: Malaysia
facility: Hospital Pulau Pinang-Pulau Pinang-21953
city: Johor Bahru
country: Malaysia
facility: Hospital Sultanah Aminah
city: Johor Bahru
country: Malaysia
facility: Hospital Sultan Ismail
city: Kuala Lumpur
country: Malaysia
facility: Hospital Kuala Lumpur
city: Putrajaya
country: Malaysia
facility: Hospital Putrajaya
city: Selangor Darul Ehsan
country: Malaysia
facility: Sunway Medical Centre
city: Mthatha, Eastern Cape
country: South Africa
facility: Nelson Mandela Academic Clinical Research Unit (NeMACRU)
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Ramón y Cajal
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taoyuan
country: Taiwan
facility: Chang Gung Memorial Hospital(Linkou)
city: Muang Chiang Mai
country: Thailand
facility: Maharat Nakhonchiangmai Hospital
city: Sfax
country: Tunisia
facility: Hedi Chaker Hospital, Department of Dermatology
city: Sousse
country: Tunisia
facility: Farhat Hached Hospital
Study officials:
References: