Telederm and Bullous Pemphigoid

Improving Clinical Trial Recruitment and Outcome Measures in Bullous Pemphigoid

The purpose of the study is to improve the quality of future clinical trials in bullous pemphigoid (BP), the investigators will monitor repeated measurement data from patients with BP on standard-of-care treatments at four week intervals for four months.

Study Overview

• Status: Completed
• Conditions: Bullous Pemphigoid

Detailed Description

The study team plans to recruit 45 subjects from the autoimmune blistering disease clinic at Emory Dermatology Clinic. The plan is to improve the quality of future clinical trials in bullous pemphigoid (BP), the team will monitor repeated measurement data from patients with BP on standard-of-care treatments at four week intervals for four months. This time frame will allow completion of both aims in the three year time period. Aim 1 will evaluate the BPDAI scoring over time with specific reductions in the activity score as future outcomes for therapeutic response and assess changes in patient reported outcomes particularly related to pruritus for future clinical trial endpoints. Aim 2 will focus on the development of teledermatology platform for improving patient recruitment and retention. The data from this proposal will be critical for future BP clinical trials and clarify gaps in the current knowledge related to the natural disease history of BP patients on standard-of-care therapies, changes in BPDAI scores over time, and pruritus specific outcome measures to define the quality of life impact.

• Study Type: Observational
• Enrollment (Actual): 21

Contacts and Locations

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory Dermatology Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

45 patients from Emory Dermatology Clinic will be enrolled

Description

Inclusion Criteria:

• Males or females > age 18
• Clinical and histological confirmation of bullous pemphigoid including at least subepidermal separation on H&E or a positive direct immunofluorescence along with indirect immunofluorescence which demonstrates staining to the roof on salt split skin or ELISA positivity for BP180 and/or BP230 autoantibodies
• Baseline BPDAI-TAS >5

Exclusion Criteria:

• Subjects who are unable to consent, language barriers, or other unspecified reason that in the opinion of the investigator makes the subject unsuitable for enrollment. Subjects that do not meet all of the enrollment criteria may not be enrolled. Any violations of these criteria must be reported in accordance with IRB Policies and Procedures.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Adult bullous pemphigoid patients; Patients with diagnosis of bullous pemphigoid will participate in monitoring with face to face assessment and remote telehealth visits with store and forward images captured.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Bullous pemphigoid disease area index (BPDAI)	Change in Bullous pemphigoid disease area index (BPDAI) activity score is the arithmetic sum of 3 subcomponents: cutaneous blisters/erosions, cutaneous urticaria/erythema, and mucosal blisters/erosions. Scores can range from 0 to 360 for BPDAI total activity (maximum 240 for total skin activity and 120 for mucosal activity), with higher scores indicating greater disease activity (worse outcome).	Baseline, monthly post-baseline for 4 months, 6 months post-baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in ItchyQol score	ItchyQoL focuses on pruritus as the main symptom impacting quality of life. ItchyQol score is based on an averaged 10-point visual analogue scale (VAS) over three time periods. Lower score correlates with better outcome.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Change in Autoimmune Bullous Disease Quality of Life score (ABQoL)	The Autoimmune Bullous Disease Quality of Life (ABQoL) score is a 17 item questionnaire assessing quality of life in patients with autoimmune blistering diseases. It is a patient reported score. Each question ranges from 0 to 3 points, with higher scores indicating poorer quality of life. The maximum ABQOL score is 51.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Change in Treatment of Autoimmune Bullous Disease Quality of Life (TABQoL) score	The Treatment of Autoimmune Bullous Disease Quality of Life (TABQoL) score is a patient reported score. The score is a 17 item questionnaire assessing quality of life in patients with autoimmune blistering diseases. Each question ranges from 0 to 3 points, with higher scores indicating poorer quality of life. The maximum TABQOL score is 51.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Change in patient and physician global assessment	The Physician and Patient Global Assessments will assess skin related health on a 0-5 scale at each visit. The lower score correlates with disease improvement (better outcome).	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Number of participants with confidence that the dermatologist can help by looking at photos	This outcome is part of the patient satisfaction survey regarding teledermatology (TD) visits in order to encourage patient enrollment and retention for the length of the trial. Higher number of patients correlates with better outcome.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Number of participants with confidence that the teledermatology visit is more convenient than going to the dermatology clinic	This outcome is part of the patient satisfaction survey regarding teledermatology (TD) visits in order to encourage patient enrollment and retention for the length of the trial. Higher number of patients correlates with better outcome.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Number of participants that believe they will use the teledermatology service again	This outcome is part of the patient satisfaction survey regarding teledermatology (TD) visits in order to encourage patient enrollment and retention for the length of the trial. Higher number of patients correlates with better outcome.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline
Change in teledermatology satisfaction score	Teledermatology satisfaction will be evaluated using a survey that includes 3 questions. Possible score ranges from 0 to 3, with higher score correlating with better outcome.	Baseline, monthly post-baseline for 4 months, 6 months post-baseline

Collaborators and Investigators

• Sponsor: Emory University
• Collaborators: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Investigators: Principal Investigator: Ron Feldman, MD, PhD, Emory University

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2021
• Primary Completion (Actual): October 6, 2023
• Study Completion (Actual): October 6, 2023

Study Registration Dates

• First Submitted: January 25, 2021
• First Submitted That Met QC Criteria: January 25, 2021
• First Posted (Actual): January 28, 2021

Study Record Updates

• Last Update Posted (Actual): October 13, 2023
• Last Update Submitted That Met QC Criteria: October 12, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Bullous Pemphigoid
• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Autoimmune Diseases; Skin Diseases, Vesiculobullous; Pemphigoid, Bullous
• Other Study ID Numbers: STUDY00001274; 1R01AR076089-01A1 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No