International Rare And Severe Psoriasis Expert Network (IRASPEN)

• ClinicalTrials.gov Identifier: NCT04359394
• Recruitment Status: Not yet recruiting
• First Posted: April 24, 2020
• Last Update Posted: January 27, 2021
• Sponsor: University Hospital, Basel, Switzerland
• Collaborator: Boehringer Ingelheim
• Information provided by (Responsible Party): University Hospital, Basel, Switzerland

Study Description

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.

Condition or disease	Intervention/treatment
Pustular Psoriasis (PP)	Other: biological sampling; Other: Phenotypic description; Other: Photography

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.

Study Design

• Study Type: Observational [Patient Registry]
• Estimated Enrollment: 180 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Target Follow-Up Duration: 5 Years
• Official Title: International Rare And Severe Psoriasis Expert Network (IRASPEN) - A Prospective Registry With Genotype-Phenotype Correlation
• Estimated Study Start Date: March 2021
• Estimated Primary Completion Date: December 2030
• Estimated Study Completion Date: December 2030

Groups and Cohorts

Group/Cohort

Intervention/treatment

PP patients; patients with active PP

Other: biological sampling; In order to investigate the level of molecular pathophysiology, blood and punch biopsies will be collected of each patient. In up to two relatives per patient, 30mL blood will be collected only once.; Other: Phenotypic description; Phenotypic characterization of the patient's clinical features; Other: Photography; All affected areas will be photographed at each visit with 2-dimensional standardized photography

Outcome Measures

Primary Outcome Measures :

1. Change in Physician Global Assessment (PGA) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   modified PGA (physician's assessment of psoriatic lesions) scoring the erythema, pustules, and scaling of all GPP or PPP lesions from 0 to 4. Each component is graded separately, the average is calculated, and the final PGA is determined from this composite score. A lower score indicates a lesser severity, with 0 being clear and 1 being almost clear.

Secondary Outcome Measures :

1. Change in Generalized Pustular Psoriasis (GPP) Area and Severity Index (GPPASI) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   Measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool that provides a numeric scoring for a patient's overall GPP disease state,ranging from 0 to 72. It is a linear combination of percent of surface area of skin that is affected by erythema, pustules and scaling and the severity of erythema, pustules, and scaling (desquamation) over 4 body regions.
2. Change in Dermatology Life Quality Index (DLQI) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   The Dermatology Life Quality Index (DLQI) consists of 10 questions concerning patient's perception of the impact of skin diseases on different aspects of their health related QoL over the last week. The DLQI evaluates the impact of the patient's skin disease on daily activities, leisure, work and personal relationships. Each question is scored on a 4-point Likert scale.
3. Change in EuroQol (EQ-5D) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   The EuroQol is a generic questionnaire. It is a preference based health status and multi attribute utility scale that produces a single index score for each state of health. These score ranges from 0 to 1, where 1 is equivalent to full health and 0 equivalent to death
4. Change in Work Productivity and Activity Impairment Questionnaire-General Health (WPAI-GH) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   The Work Productivity and Activity Impairment Questionnaire-General Health (WPAI-GH) consists of six questions: 1 = currently employed; 2 = hours missed due to health problems; 3 = hours missed other reasons; 4 = hours actually worked; 5 = degree health affected productivity while working (using a 0 to 10 Visual Analogue Scale (VAS)); 6 = degree health affected productivity in regular unpaid activities (VAS)[
5. Change in psoriasis symptom scale (PSS) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   The psoriasis symptom scale (PSS) is a measure of the degree to which situations in one's life are appraised as stressful. Items were designed to assess how unpredictable, uncontrollable, and overloaded respondents find their lives to be. The scale also includes a number of direct queries about current levels of experienced stress. Moreover, the questions are of a general nature and hence are relatively free of content specific to any sub-population group. The questions in the PSS ask about feelings and thoughts during the last month.
6. Change in Disease activity Visual analogue Scale (VAS) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   The disease VAS is a unidimensional measure of disease intensity. The VAS is a continuous scale comprised of a horizontal line, 10 centimeters (100 mm) in length, anchored by 2 verbal descriptors, one for each symptom extreme. To avoid clustering of scores around a preferred numeric value, numbers or verbal descriptors at intermediate points are not given. The disease intensity VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their current disease intensity.
7. Change in Pain Visual analogue Scale (VAS) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   The pain VAS is a unidimensional measure of pain intensity, which has been widely used in diverse adult populations. The pain VAS is a continuous scale comprised of a horizontal line, 10 centimeters (100 mm) in length, anchored by 2 verbal descriptors, one for each symptom extreme. To avoid clustering of scores around a preferred numeric value, numbers or verbal descriptors at intermediate points are not given. The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity.
8. Number of flares in the last 2 years [ Time Frame: at Baseline ]
   Number of flares in the last 2 years
9. Number of flares since the last visit [ Time Frame: At week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
   Number of flares since the last visit
10. Change in Palmoplantar Pustulosis (PPP) Area and Severity Index (PPPASI) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
    investigator assessment of the extent and severity of pustular and plaque lesions on the palms and soles presenting in PPP patients. This tool provides a numeric scoring for patients overall PPP disease state, ranging from 0 to 72. It is a linear combination of the percent of surface area of skin that is affected on the palms and soles of the body and the severity of erythema, pustules, and scaling (desquamation).
11. Change in Psoriasis Area and Severity Index (PASI) [ Time Frame: At Baseline, week 12, week 24, week 36, week 52, week 64, Week 76, week 104, week 130, week 156, week 182, week 208, week 234, week 260 ]
    The Psoriasis Area and Severity Index (PASI) is a composite variable used to assess the severity of Psoriasis. The PASI evaluates the area of psoriatic involvement in 4 main areas (head, trunk, upper and lower extremities) and the severity of the psoriatic lesions with respect to three target symptoms: erythema, infiltration and desquamation (actual percentages of area involvement) .

Biospecimen Retention:   Samples With DNA

fresh blood (whole blood) and skin biopsies (formalin-fixation followed by embedding in paraffin and fixation in RNAlater) for isolation of RNA, DNA

Eligibility Criteria

• Ages Eligible for Study: 6 Months and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• 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.

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 active pustulation with either white, yellow or brown pustules
• 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

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

Contacts and Locations

Contacts

Contact: Alexander Navarini, Prof. Dr. med. Dr. sc. nat.; +41 61 328 60 80; alexander.navarini@usb.ch

Locations

Switzerland

Dermatology, University Hospital Basel

Basel, Switzerland, 4031

Contact: Alexander Navarini, Prof. Dr. med. Dr. sc. nat. +41 61 328 60 80 alexander.navarini@usb.ch

Sponsors and Collaborators

University Hospital, Basel, Switzerland

Boehringer Ingelheim

Investigators

• Principal Investigator: Alexander Navarini, Prof. Dr. med. Dr. sc. nat.; Dermatologie, Universitätsspital Basel

More Information

• Responsible Party: University Hospital, Basel, Switzerland
• ClinicalTrials.gov Identifier: NCT04359394
• Other Study ID Numbers: 2020-00425; sp18Navarini2
• First Posted: April 24, 2020
• Last Update Posted: January 27, 2021
• Last Verified: January 2021

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University Hospital, Basel, Switzerland:

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)

Additional relevant MeSH terms:

Psoriasis; Skin Diseases, Papulosquamous; Skin Diseases