Phase 2 N=53 Randomized Double-blind Treatment

Effisayil™ 1: A Study to Test Spesolimab (BI 655130) in Patients With a Flare-up of a Skin Disease Called Generalized Pustular Psoriasis

Generalized Pustular Psoriasis

Bottom Line

View on ClinicalTrials.gov: NCT03782792 ↗

Enrolled (actual)

Serious AEs

19.8%

Results posted

Mar 2022

Primary outcome: Primary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1 — 0.056; 0.543 Proportion of participants — p=0.0004

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Spesolimab (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Boehringer Ingelheim
Primary completion: Sep 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Subscore of 0 Indicating no Visible Pustules at Week 1	0.056; 0.543	0.0004 sig
SECONDARY Key Secondary: Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 1	0.111; 0.429	0.0118 sig
SECONDARY Proportion of Patients With a Psoriasis Area and Severity Index for Generalized Pustular Psoriasis (GPPASI) 75 at Week 4	0.111; 0.457	0.0081 sig
SECONDARY Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 4	NA; -22.45	0.0012 sig
SECONDARY Change From Baseline in Psoriasis Symptom Scale (PSS) Score at Week 4	NA; -2.0	0.0044 sig
SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Score at Week 4	NA; 3.00	0.0012 sig
SECONDARY Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Score of 0 or 1 at Week 4	0.111; 0.486	—
SECONDARY Proportion of Patients With a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Pustulation Sub-score of 0 Indicating no Visible Pustules at Week 4	0.111; 0.514	—
SECONDARY Proportion of Patients With a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 4	0.111; 0.543	—
SECONDARY Percent Change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) From Baseline at Week 4	NA; -60.50	—
SECONDARY Proportion of Patients With a Generalized Pustular Psoriasis Area and Severity Index (GPPASI) 50 at Week 1	0.278; 0.429	—
SECONDARY Percent Change in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) From Baseline at Week 1	1.02; -42.80	—
SECONDARY Occurrence of Treatment Emergent Adverse Events (TEAEs) up to Week 1	6445.6; 8650.7	—
SECONDARY Number of Patients With Treatment Emergent Adverse Events (TEAEs) up to Week 1	12; 27	—
SECONDARY Occurrence of Treatment Emergent Adverse Events (TEAEs) Within the Treatment Phase	3083.3; 2391.0	—
SECONDARY Number of Patients With Treatment Emergent Adverse Events (TEAEs) Within the Treatment Phase	13; 29	—

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03782792). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.