A Study to Test BI 655130 in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis

• Register: ClinicalTrials.gov
• Last refreshed on: 27 September 2021
• Main ID: NCT03782792
• Date of registration: 19/12/2018
• Prospective Registration: Yes
• Primary sponsor: Boehringer Ingelheim
• Public title: A Study to Test BI 655130 in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis
• Scientific 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 BI 655130 in Patients With Generalized Pustular Psoriasis (GPP) Presenting With an Acute Flare of Moderate to Severe Intensity.
• Date of first enrolment: January 31, 2019
• Target sample size: 53
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03782792
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

China, France, Germany, Japan, Korea, Republic of, Malaysia, Singapore, Switzerland, Taiwan, Thailand, Tunisia, United States

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Generalized Pustular Psoriasis

Intervention(s)

Drug: Placebo

Drug: Spesolimab

Primary Outcome(s)

Proportion of patients with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation subscore of 0 indicating no visible pustules at Week 1 [Time Frame: At Week 1.]

Secondary Outcome(s)

Key secondary: Proportion of patients with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 at Week 1 [Time Frame: At Week 1.]

Occurrence Treatment Emergent Adverse Events (TEAEs) up to Week 1 [Time Frame: From start of treatment until Day 7, up to 7 days.]

Change from baseline in Psoriasis Symptom Scale (PSS) score at Week 4 [Time Frame: Baseline and at Week 4.]

Number of patients with Treatment Emergent Adverse Events (TEAEs) up to Week 1 [Time Frame: From start of treatment until Day 7, up to 7 days.]

Number of patients with Treatment Emergent Adverse Events (TEAEs) within the treatment phase [Time Frame: From start of trreatment until end of the residual effect period (REP) but censored at any use of open label spesolimab, up to 16 weeks.]

Percent change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) from baseline at Week 1 [Time Frame: At Week 1.]

Percent change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) from baseline at Week 4 [Time Frame: Baseline and at Week 4.]

Proportion of patients with a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 4 [Time Frame: At Week 4.]

Proportion of patients with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 at Week 4 [Time Frame: At Week 4.]

Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue score at Week 4 [Time Frame: Baseline and at Week 4.]

Change from baseline in Pain Visual Analog Scale (VAS) score at Week 4 [Time Frame: Baseline and at Week 4.]

Occurrence of Treatment Emergent Adverse Events (TEAEs) within the treatment phase [Time Frame: From start of trreatment until end of the residual effect period (REP) but censored at any use of open label spesolimab, up to 16 weeks.]

Proportion of patients with a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 1 [Time Frame: At Week 1.]

Proportion of patients with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation sub-score of 0 indicating no visible pustules at Week 4 [Time Frame: At Week 4.]

Proportion of patients with a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4 [Time Frame: At Week 4.]

Secondary ID(s)

1368-0013

2017-004231-37

Source(s) of Monetary Support

Please refer to primary and secondary sponsors