Clinical trial NCT03782792: Effisayil™ 1: A Study to Test Spesolimab (BI 655130) in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis
Official title: Effisayil™ 1:Multi-center, Double-blind, Randomised, Placebo-controlled, Phase II Study to Evaluate Efficacy, Safety and Tolerability of a Single Intravenous Dose of Spesolimab (BI 655130) in Patients With Generalized Pustular Psoriasis (GPP) Presenting With an Acute Flare of Moderate to Severe Intensity
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Generalized Pustular Psoriasis
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2018-12-20
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To evaluate efficacy, safety, and tolerability of spesolimab (BI 655130) compared to placebo in patients with Generalized Pustular Psoriasis (GPP) presenting with an acute flare of moderate to severe intensity.


Eligibility criteria: Inclusion Criteria:

* Patients with GPPGA of 0 or 1 and a known and documented history of GPP per European Rare And Severe Psoriasis Expert Network (ERASPEN) criteria regardless of IL36RN mutation status, with previous evidence of fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis with peripheral blood neutrophilia (above ULN) OR

  -- Patients with an acute flare of moderate to severe intensity meeting the (ERASPEN) criteria of GPP with a known and documented history of GPP (per ERASPEN criteria) regardless of IL36RN mutation status, with previous evidence of fever, and/or asthenia, and/or myalgia, and/or elevated C-reactive protein, and/or leucocytosis with peripheral blood neutrophilia (above ULN)
* Male or female patients, aged 18 to 75 years at screening.
* Signed and dated written informed consent prior to admission to the study in accordance with ICH GCP and local legislation prior to start of any screening procedures.
* Women of childbearing potential 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. Note: A woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. Tubal ligation is not a method of permanent sterilization. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause
* Further inclusion criteria apply

Exclusion Criteria:

* Patients with SAPHO (Synovitis-acne-pustulosis-hyperostosis-osteitis) syndrome.
* Patients with primary erythrodermic psoriasis vulgaris.
* Patients with primary plaque psoriasis vulgaris without presence of pustules or with pustules that are restricted to psoriatic plaques.
* Drug-triggered Acute Generalized Exanthematous Pustulosis (AGEP).
* 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 renal failure.
* Severe, progressive, or uncontrolled hepatic disease, defined as \>3- fold Upper Limit of Normal (ULN) elevation in AST or ALT or alkaline phosphatase, or \>2-fold ULN elevation in total bilirubin.
* Further exclusion criteria apply

Primary outcomes:
measure: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1
timeFrame: At Week 1.
Secondary outcomes:
measure: Key Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1
timeFrame: At Week 1.
measure: Proportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4
timeFrame: At Week 4.
measure: Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4
timeFrame: Baseline and at Week 4.
measure: Change From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4
timeFrame: Baseline and at Week 4.
measure: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Score at Week 4
timeFrame: Baseline and at Week 4.
measure: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 4
timeFrame: At Week 4.
measure: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Sub-score of 0 Indicating no Visible Pustules at Week 4
timeFrame: At Week 4.
measure: Proportion of Patients With a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 4
timeFrame: At Week 4.
measure: Percent Change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) From Baseline at Week 4
timeFrame: Baseline and at Week 4.
measure: Proportion of Patients With a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 1
timeFrame: At Week 1.
measure: Percent Change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) From Baseline at Week 1
timeFrame: At Week 1.
measure: Occurrence of Treatment Emergent Adverse Events (TEAEs) up to Week 1
timeFrame: From start of treatment until Day 7, up to 7 days.
measure: Number of Patients With Treatment Emergent Adverse Events (TEAEs) up to Week 1
timeFrame: From start of treatment until Day 7, up to 7 days.
measure: Occurrence of Treatment Emergent Adverse Events (TEAEs) Within the Treatment Phase
timeFrame: From start of treatment until end of the residual effect period (REP) but censored at any use of open label spesolimab, up to 16 weeks.
measure: Number of Patients With Treatment Emergent Adverse Events (TEAEs) Within the Treatment Phase
timeFrame: From start of treatment until end of the residual effect period (REP) but censored at any use of open label spesolimab, up to 16 weeks.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Dalian
country: China
facility: The First Hospital of Dalian Medical University
city: Hangzhou
country: China
facility: 2nd Affiliated Hosp Zhejiang University College of Medical
city: Shanghai
country: China
facility: Shanghai Skin Disease Hospital
city: Shanghai
country: China
facility: Huashan Hospital, Fudan University
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
city: Bordeaux
country: France
facility: HOP Saint-André
city: Paris
country: France
facility: HOP Saint-Louis
city: Reims
country: France
facility: HOP Robert Debré
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn AöR
city: Essen
country: Germany
facility: Universitätsklinikum Essen AöR
city: München
country: Germany
facility: Klinikum der Universität München - Campus Innenstadt
city: Aichi, Nagoya
country: Japan
facility: Nagoya City University Hospital
city: Fukuoka, Fukuoka
country: Japan
facility: Fukuoka University Hospital
city: Hokkaido, Asahikawa
country: Japan
facility: Asahikawa Medical University Hospital
city: Miyagi, Sendai
country: Japan
facility: Tohoku University Hospital
city: Tokyo, Hachioji
country: Japan
facility: Tokyo Medical University Hachioji Medical Center
city: Tokyo, Shinjuku-ku
country: Japan
facility: Tokyo Medical University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
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: Muar
country: Malaysia
facility: Hospital Pakar Sultanah Fatimah
city: Negeri Perak/Ipoh
country: Malaysia
facility: Hospital Raja Permaisuri Bainun
city: Pulau Pinang
country: Malaysia
facility: Hospital Pulau Pinang
city: Selangor
country: Malaysia
facility: Hospital Selayang
city: Singapore
country: Singapore
facility: National University Hospital
city: Lausanne
country: Switzerland
facility: University Hospital of Lausanne
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Ratchatewi, Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Sousse
country: Tunisia
facility: Farhat Hached Hospital
city: Tunisia
country: Tunisia
facility: Hedi Chaker Hospital, Department of Dermatology
city: Tunis
country: Tunisia
facility: La Rabta Hospital
Study officials:
References:
citation: Bachelez H, Choon SE, Marrakchi S, Burden AD, Tsai TF, Morita A, Navarini AA, Zheng M, Xu J, Turki H, Anadkat MJ, Rajeswari S, Hua H, Vulcu SD, Hall D, Tetzlaff K, Thoma C, Lebwohl MG; Effisayil 1 Trial Investigators. Trial of Spesolimab for Generalized Pustular Psoriasis. N Engl J Med. 2021 Dec 23;385(26):2431-2440. doi: 10.1056/NEJMoa2111563.
citation: Choon SE, Lebwohl MG, Marrakchi S, Burden AD, Tsai TF, Morita A, Navarini AA, Zheng M, Xu J, Turki H, Rajeswari S, Deng H, Tetzlaff K, Thoma C, Bachelez H. Study protocol of the global Effisayil 1 Phase II, multicentre, randomised, double-blind, placebo-controlled trial of spesolimab in patients with generalized pustular psoriasis presenting with an acute flare. BMJ Open. 2021 Mar 30;11(3):e043666. doi: 10.1136/bmjopen-2020-043666.