PemphigusNCT05534776
Validation of 5-Point Investigator Global Assessments for Pemphigus
An observational study of Pemphigus, run by Premier Specialists, Australia at 7 sites in 7 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pemphigus
- What is being tested?
- Investigator Global Assessments (IGAs)
- How many people
- 40 participants
- Where
- 7 sitesin 7 countries
- Study dates
- 21 Sep 2022to 30 Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients of the special bullous clinic at the lead site's dermatology practice who have been diagnosed with pemphigus.
Who may be able to join
- Having pemphigus diagnosed by a qualified dermatologist
- Aged 18 or older at the time of photography
Who cannot join
- Those whose photographs are unable to be adequately de-identified
Trial locations and contacts
7 sites in 7 countries.
7 sites in 7 countries
Australia1 site, 1 recruiting
- Recruiting
Premier Specialists
Kogarah, New South Wales, 2217
Bianca Wills, 9598 5800
Darby Boucher
Bulgaria1 site, 1 recruiting
- Not yet recruiting
Medical University of Sofia
Sofia
Snejina Vassileva, snejinavassileva@gmail.com
Snejina Vassileva
Greece1 site, 1 recruiting
- Not yet recruiting
Aristotle University of Thessaloniki
Thessaloniki
Aikaterini Patsatsi, katerinapatsatsi@gmail.com
Aikaterini Patsatsi
Iran1 site, 1 recruiting
- Not yet recruiting
Razi Hospital
Tehran
Maryam Daneshpazhooh, maryamdanesh.pj@gmail.com
Maryam Daneshpazhooh
Israel1 site, 1 recruiting
- Not yet recruiting
Tel Aviv Medical Centre
Tel Aviv
Avital Baniel, avitalba@tlvmc.gov.il
Avital Baniel
Singapore1 site, 1 recruiting
- Not yet recruiting
National University Hospital of Singapore
Singapore
Nisha Chandran, nishasuch@gmail.com
Nisha Chandran
Turkey (Türkiye)1 site, 1 recruiting
- Not yet recruiting
Akdeniz University Hospital
Antalya
Asli Bilgic, aslibilgictemel@gmail.com
Asli Bilgic
About this study
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.
Read the 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.
- Conditions
- Pemphigus
- Being tested
- Investigator Global Assessments (IGAs)
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Investigator Global Assessments (IGAs)Other
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.
| Study group | Role | What they receive |
|---|---|---|
| First Scoring Session | Participants whose de-identified photos will be printed in the first booklet for the first scoring session. Receives: Other: Investigator Global Assessments (IGAs) | |
| Second Scoring Session | Participants whose de-identified photos will be printed in the second booklet for the second scoring session. Receives: Other: Investigator Global Assessments (IGAs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline
Pemphigus Disease Activity Index (PDAI) Score
Possible score from 0-263
Measured: Baseline
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)
Possible score from 0-206
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2022
- Study start21 Sep 2022 (estimated)
- Primary completion30 Aug 2023 (estimated)
- Study completion30 Aug 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PemphigusStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Premier Specialists, Australia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dedee Murrell, Premier Specialists, Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05534776
- Sponsor's protocol ID
- IGA Pemphigus
- Status last verified
- Sep 2022
- First submitted
- 29 Aug 2022
- Last updated
- 19 Sep 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of 5-Point Investigator Global Assessments for Pemphigus
- ClinicalTrials.gov ID
- NCT05534776
- Acronym / sponsor ID
- IGA Pemphigus
- Conditions
- Pemphigus
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Premier Specialists, AustraliaUniversity, hospital or other
- Study start
- 21 Sep 2022 (estimated)
- Primary completion
- 30 Aug 2023 (estimated)
- Study completion
- 30 Aug 2023 (estimated)
- First posted
- 9 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| First Scoring Session | – | Other: Investigator Global Assessments (IGAs)Participants whose de-identified photos will be printed in the first booklet for the first scoring session. |
| Second Scoring Session | – | Other: Investigator Global Assessments (IGAs)Participants whose de-identified photos will be printed in the second booklet for the second scoring session. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients of the special bullous clinic at the lead site's dermatology practice who have been diagnosed with pemphigus.
Key inclusion criteria (2 listed)
- Having pemphigus diagnosed by a qualified dermatologist
- Aged 18 or older at the time of photography
Key exclusion criteria (1 listed)
- Those whose photographs are unable to be adequately de-identified
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Other | First Scoring Session, Second Scoring Session |
Primary outcome
IGA-PV-M (D/P/F), IGA-PV-S (D/P/F), IGA-PF (D/P/F)
Time frame: Baseline
IGA score of pemphigus severity, possible score from 0-4
Pemphigus Disease Activity Index (PDAI) Score
Time frame: Baseline
Possible score from 0-263
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)
Time frame: Baseline
Possible score from 0-206
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
7 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pemphigus, run by Premier Specialists, Australia at 7 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Australia, Bulgaria, Greece, Iran, Israel and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Bianca Wills (0452409850, premierspectrialsbianca@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
IGA-PV-M (D/P/F), IGA-PV-S (D/P/F), IGA-PF (D/P/F) (time frame: Baseline). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2022.