Validation of 5-Point Investigator Global Assessments for Pemphigus

Sponsor

Premier Specialists, Australia (Other)

Overall Status

Recruiting

CT.gov ID

NCT05534776

Collaborator

(none)

Enrollment

Locations

11.3

Anticipated Duration (Months)

5.7

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Pemphigus	Other: Investigator Global Assessments (IGAs)

Detailed 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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

40 participants

Observational Model:

Other

Time Perspective:

Cross-Sectional

Official Title:

Validation of 5-Point Investigator Global Assessments for Pemphigus

Anticipated Study Start Date :

Sep 21, 2022

Anticipated Primary Completion Date :

Aug 30, 2023

Anticipated Study Completion Date :

Aug 30, 2023

Arms and Interventions

Arm	Intervention/Treatment
First Scoring Session Participants whose de-identified photos will be printed in the first booklet for the first scoring session.	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.
Second Scoring Session Participants whose de-identified photos will be printed in the second booklet for the second scoring session.	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.

Arm

Intervention/Treatment

First Scoring Session

Participants whose de-identified photos will be printed in the first booklet for the first scoring session.

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.

Second Scoring Session

Participants whose de-identified photos will be printed in the second booklet for the second scoring session.

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.

Outcome Measures

Primary Outcome Measures

IGA-PV-M (D/P/F), IGA-PV-S (D/P/F), IGA-PF (D/P/F) [Baseline]
IGA score of pemphigus severity, possible score from 0-4
Pemphigus Disease Activity Index (PDAI) Score [Baseline]
Possible score from 0-263
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) [Baseline]
Possible score from 0-206

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Premier Specialists	Kogarah	New South Wales	Australia	2217
2	Medical University of Sofia	Sofia		Bulgaria
3	Aristotle University of Thessaloniki	Thessaloníki		Greece
4	Razi Hospital	Tehran		Iran, Islamic Republic of
5	Tel Aviv Medical Centre	Tel Aviv		Israel
6	National University Hospital of Singapore	Singapore		Singapore
7	Akdeniz University Hospital	Antalya		Turkey

Sponsors and Collaborators

Premier Specialists, Australia

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Dedee Murrell, Professor, Director of Premier Specialists, Premier Specialists, Australia

ClinicalTrials.gov Identifier:

NCT05534776

Other Study ID Numbers:

IGA Pemphigus

First Posted:

Sep 9, 2022

Last Update Posted:

Sep 19, 2022

Last Verified:

Sep 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Pemphigus

Study Results

No Results Posted as of Sep 19, 2022