Clinical trial NCT04359394: International Rare And Severe Psoriasis Expert Network
Official title: International Rare And Severe Psoriasis Expert Network (IRASPEN) - A Prospective Registry With Genotype-Phenotype Correlation
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pustular Psoriasis (PP)
Keywords: Palmoplantar Psoriasis (PPP)
IRASPEN International Rare and Severe Psoriasis Expert Network
ERASPEN European Rare and Severe Psoriasis Expert Network
Psoriasis
Generalized pustular psoriasis (GPP)
Acrodermatitis continua of Hallopeau (ACH)
Interleukin 36 Receptor Antagonist Gene (IL36RN)
Caspase Recruitment Domain-containing protein 14 (CARD14)
Adaptor Related Protein Complex 1 Subunit Sigma 3 (AP1S3)
Lead sponsor: University Hospital, Basel, Switzerland
Sponsor class: OTHER
Collaborators: name: Boehringer Ingelheim
First posted: 2020-04-24
Enrollment: 180
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The project population encompasses all patients aged 6 months and over with active PP in the participating countries. Patients will be recruited by consecutive ongoing recruitment in the investigators' clinical practice during 5 years.

Brief summary: This registry is a prospective observational study in order to describe primarily the natural course of PP subtypes and to gain detailed information about their phenotype.

Detailed description: This project is to describe the natural course of disease in different subtypes of PP. The network builds on a static registry that was based on a one-time clinical characterization of PP patients in Europe (ERASPEN). The International Rare and Severe Psoriasis Expert Network (IRASPEN) already has multiple clinicians involved who have successfully characterized and included their patients in ERASPEN. IRASPEN addresses the question of temporal evolution of clinical features and is actually a non-interventional prospective registry that aims to describe the clinical course and responses to already established treatments of a large number of PP patients over a period of 5 years. The data collection with this registry will give insight on the natural course of PP disease revealing the burden of disease including frequency and severity of flares and the role of therapeutic interventions.

Eligibility criteria: Inclusion Criteria:

* Written informed consent of the patient or legal proxy in the registry
* Diagnosis of PP confirmed by a dermatologist in the participant. The type of PP can be any one of PPP, GPP/Acute Generalized Exanthematous Pustulosis (AGEP), ACH or a mixed phenotype, according to the judgment of the investigator
* GPP: Primary, sterile, macroscopically visible epidermal pustules on non-acral Skin with or without systemic Inflammation; with or without plaque psoriasis; either relapsing (\>1 episode) or persistent (\>3 months)
* PPP: Primary, persistent (\>3 months), sterile, macroscopically visible epidermal pustules on palms and/or soles with or without plaque psoriasis
* ACH: Primary, persistent (\>3 months), sterile, macroscopically visible epidermal pustules affecting the nail apparatus with or without plaque psoriasis
* At the timepoint of inclusion, the participant must have had active pustulation with either white, yellow or brown pustules within six month before baseline. Active postulation at baseline is not mandatory for inclusion.
* Sufficient language skills (in the languages which the patient information and the consent form is available) for the informed consent to participate
* Patients of all ancestries and skin pigment type can be included
* Direct non-affected adult (\>18 years old) relatives of the participant (up to two, namely mother, father, sibling) with the purpose to provide DNA for family trio sequencing analysis. The patient is not excluded from the study if no relatives are included.

Exclusion Criteria:

* Any medical or psychological condition in the treating physician's opinion which may prevent the patient in registry participation for the next 5 years
* Lack of informed consent for registry participation

Primary outcomes:
measure: Change in Physician Global Assessment (PGA)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
Secondary outcomes:
measure: Change in Generalized Pustular Psoriasis (GPP) Area and Severity Index (GPPASI)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in Dermatology Life Quality Index (DLQI)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in EuroQol (EQ-5D)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in Work Productivity and Activity Impairment Questionnaire-General Health (WPAI-GH)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in psoriasis symptom scale (PSS)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in Disease activity Visual analogue Scale (VAS)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in Pain Visual analogue Scale (VAS)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Number of flares in the last 2 years
timeFrame: at Baseline
measure: Number of flares since the last visit
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in Palmoplantar Pustulosis (PPP) Area and Severity Index (PPPASI)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
measure: Change in Psoriasis Area and Severity Index (PASI)
timeFrame: At Baseline, week 12, week 24, week 52, week 104, week 156, week 208, week 260
Other outcomes:
Locations:
city: München
country: Germany
facility: Klinikum der Universität München
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario "Agostino Gemelli" IRCCS
city: Porto
country: Portugal
facility: Universitário do Porto
city: Singapore
country: Singapore
facility: National Skin Centre
city: Basel
country: Switzerland
facility: Dermatology, University Hospital Basel
city: Zürich
country: Switzerland
facility: University Hospital Zürich
city: Antalya
country: Turkey
facility: Akdeniz University School of Medicine; Department of Dermatology and Venereology
city: Edirne
country: Turkey
facility: Trakya University, Faculty of Medicine; Department of Dermatology and Venereology
Study officials:
affiliation: Dermatologie, Universitätsspital Basel
name: Alexander Navarini, Prof. Dr. med. Dr. sc. nat.
role: PRINCIPAL_INVESTIGATOR
References: