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Autoimmune Blistering Diseases Study (AIBD)

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ClinicalTrials.gov Identifier: NCT02753777
Recruitment Status : Recruiting
First Posted : April 28, 2016
Last Update Posted : November 15, 2016
Sponsor:
Information provided by (Responsible Party):
VICTORIA WERTH, University of Pennsylvania

Brief Summary:
Pemphigus and bullous pemphigoid (BP) are severe autoimmune blistering diseases (AIBD) that pose a critical need for new therapeutic approaches. Clinical trials in pemphigus and BP will require the availability of validated disease severity measures that can be used to define primary outcomes.

Condition or disease Intervention/treatment
Pemphigus Vulgaris Pemphigus Foliaceus Bullous Pemphigoid Other: Questionnaires

Detailed Description:

The disease severity instruments for pemphigus and BP, the Pemphigus Disease Area Index (PDAI) and the Bullous Pemphigoid Disease Area Index (BPDAI), respectively, have the potential to capture changes in all grades of disease activity, including mild disease, and therefore represent a substantial improvement over simple lesion counts or measurements of affected body surface area. However, full validation of the PDAI and BPDAI for use in clinical trials will require additional and more extensive measurements that will enable us to classify patients correctly by disease severity and define the minimal change in score that is clinically significant.

This database study will compare the Pemphigus Disease Area and Severity Index (PDAI) and the Bullous Pemphigoid Disease Area Index (BPDAI) against other disease severity measures, including the Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) and a physician's global assessment. Additionally, patients will be asked to rate their overall disease severity using a visual analogue scale, the patient's global assessment. Participants will also have the option to donate blood samples to our AIBD blood bank for immunologic and pathophysiologic studies.

The purpose of this database study will also be to evaluate quality of life (QoL) measures and correlate these QoL measures with disease severity in patients with pemphigus and BP. QoL is an important and independent component of disease impact on patients. This database study will use the following QoL measures: the SF-36, which is a nonspecific QoL measure; the Skindez-29 and the Dermatology Life Quality Index (DLQI), which are dermatology-specific instruments; the Autoimmune Bullous Disease Quality of Life (ABQOL) and Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) questionnaires, which are AIBD-specific tools. The results will be utilized in planning future clinical studies.

The AIBD study will be conducted only at Penn at this time. Data from the study will be entered into a password protected, web-based database that is used only at Penn.


Study Type : Observational [Patient Registry]
Estimated Enrollment : 150 participants
Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration: 3 Years
Official Title: Prevalence and Clinical Severity of Autoimmune Blistering Diseases
Study Start Date : April 2016
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : April 2019





Primary Outcome Measures :
  1. Pemphigus Disease Area Index (PDAI) [ Time Frame: one year ]
  2. Bullous Pemphigoid Disease Area Index (BPDAI) [ Time Frame: one year ]

Secondary Outcome Measures :
  1. Autoimmune Bullous Disease Quality of Life (ABQOL) [ Time Frame: one year ]
  2. Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) [ Time Frame: one year ]
  3. Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) [ Time Frame: one year ]

Biospecimen Retention:   Samples With DNA
Blood specimens will be collected during regularly scheduled clinic visits. The specimens will be processed and stored at the research labs belonging to the principal investigator at the University of Pennsylvania and the Philadelphia VA Medical Center. The specimens will not be publicly available and will be coded and stored in a locked facility equipped with a freezer.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adult patients over the age of 18 who have been diagnosed with pemphigus vulgaris, pemphigus foliaceus or bullous pemphigoid.
Criteria

Inclusion Criteria:

  • Gender/Age: Males or females above 18 years old
  • Diagnosis: pemphigus vulgaris, pemphigus foliaceus, bullous pemphigoid
  • Subjects able to give informed consent

Exclusion Criteria:

  • Patients who are under age 18 years.
  • Patients without pemphigus or pemphigoid diseases
  • Penn employees
  • Penn students
  • Cognitively impaired persons

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02753777


Contacts
Contact: Victoria P Werth, MD 215-615-2940 werth@mail.med.upenn.edu
Contact: Joyce Okawa, RN 215-615-2960 Joyce.Okawa@uphs.upenn.edu

Locations
United States, Pennsylvania
Hospital of the University of Pennsylvania, Department of Dermatology Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Victoria P Werth, MD    215-615-2940    werth@mail.med.upenn.edu   
Principal Investigator: Victoria P Werth, MD         
Hospital of the University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Victoria P Werth, MD    215-898-0168    werth@mail.med.upenn.edu   
University of Pennsylvania, Department of Dermatology Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Victoria P Werth, MD    215-615-2940    werth@mail.med.upenn.edu   
Principal Investigator: Victoria P. Werth, MD         
Sponsors and Collaborators
University of Pennsylvania

Responsible Party: VICTORIA WERTH, Professor of Dermatology, University of Pennsylvania
ClinicalTrials.gov Identifier: NCT02753777     History of Changes
Other Study ID Numbers: 824790
First Posted: April 28, 2016    Key Record Dates
Last Update Posted: November 15, 2016
Last Verified: November 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Pemphigus
Pemphigoid, Bullous
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases