Validation of 5-Point Investigator Global Assessments for Pemphigus

Pemphigus Clinical Trial

Official title:

Validation of 5-Point Investigator Global Assessments for Pemphigus

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05534776
• Other study ID #: IGA Pemphigus
• Status: Recruiting
• Start date: September 21, 2022
• Est. completion date: August 30, 2023

Study information

• Verified date: September 2022
• Source: Premier Specialists, Australia
• Contact: Bianca Wills
• Phone: 0452409850
• Email: premierspectrialsbianca[@]gmail.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study aims to explore the reliability and validity of newly developed Investigator Global Assessments (IGAs) in scoring the severity of pemphigus. IGAs are simple 5-point scales ranging from clear - severe and are preferred by the FDA as endpoints in clinical trials.

Description:

This study aims to obtain statistical data regarding the inter-rater and intra-rater reliability of the IGAs, Pemphigus Disease Activity Index (PDAI) and Autoimmune Bullous Skin Disease Intensity Score (ABSIS) as well as the convergent validity of the IGAs with the PDAI and objective component of the ABSIS.

Photograph sets of pemphigus lesions will be gathered from the lead site and participant sites. They will be de-identified and printed in a two hardcopy booklets; the first containing 20 photo sets and the second containing 17 new photo sets and 3 repeat photo sets. In the first part of the study, 8 dermatologists will score 20 sets of photographs of pemphigus lesions (a mixture of skin and mucosal lesions), using the IGAs for pemphigus as well as the PDAI and ABSIS. The first booklet will be mailed to dermatologists and they will use the booklet to score each photo set and will submit their scores in a confidential online survey. In the second part of the study, the second booklet will be sent to dermatologists in the same manner 4 weeks later, and each dermatologist will score the photo sets using the same scoring tools. It is estimated that each scoring session will take two hours to complete. Data gathered here will allow for calculation of inter-rater reliability and intra-rater reliability of the IGAs and convergent validity of the IGAs with PDAI and ABSIS.

Later, a sub-study will occur to calculate minimal clinically important differences (MCID) for the IGAs and PDAI. This will involve scoring pemphigus severity of patients at Premier Specialists using PDAI and IGA scores and a Likert score to classify patients as improved, stable or deteriorated compared to previous visits.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: August 30, 2023
• Est. primary completion date: August 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Having pemphigus diagnosed by a qualified dermatologist

• Aged 18 or older at the time of photography

Exclusion Criteria:

• Those whose photographs are unable to be adequately de-identified

Related Conditions & MeSH terms

• Pemphigus

Intervention

Other:
• Investigator Global Assessments (IGAs)
5-Point scale from 0-4 to score the severity of pemphigus, where 0=clear, 1=almost clear, 2=mild, 3=moderate and 4=severe.

Locations

Country	Name	City	State
Australia	Premier Specialists	Kogarah	New South Wales
Bulgaria	Medical University of Sofia	Sofia
Greece	Aristotle University of Thessaloniki	Thessaloníki
Iran, Islamic Republic of	Razi Hospital	Tehran
Israel	Tel Aviv Medical Centre	Tel Aviv
Singapore	National University Hospital of Singapore	Singapore
Turkey	Akdeniz University Hospital	Antalya

Sponsors (1)

Lead Sponsor	Collaborator
Premier Specialists, Australia

Countries where clinical trial is conducted

Australia,  Bulgaria,  Greece,  Iran, Islamic Republic of,  Israel,  Singapore,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	IGA-PV-M (D/P/F), IGA-PV-S (D/P/F), IGA-PF (D/P/F)	IGA score of pemphigus severity, possible score from 0-4	Baseline
Primary	Pemphigus Disease Activity Index (PDAI) Score	Possible score from 0-263	Baseline
Primary	Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)	Possible score from 0-206	Baseline