Actively Recruiting
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
Led by Krystal Biotech, Inc. · Updated on 2025-07-02
40
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to understand the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB), including both recessive and dominant forms. The study is observational and non-interventional, focusing on how often corneal abrasions occur and their symptoms over time in this patient population. It is sponsored by Krystal Biotech, Inc. and seeks to gather detailed information to better characterize these eye issues in DEB. Participants will not receive any treatment as part of this study. Instead, they will be asked to record their symptoms weekly using remote diaries and complete monthly questionnaires to report on the frequency and duration of corneal abrasions. The study will monitor these patterns over a period of up to one year. During the study, participants will regularly provide information about their eye symptoms through digital symptom diaries and questionnaires. The researchers will track the frequency of corneal abrasions as the main measure and also evaluate symptom severity using a specific eye disease index over the course of the study. The total duration of participation may last up to one year, with no interventional procedures involved.
CONDITIONS
Brief Title
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- The subject or their legal guardian must have read, understood, and signed an IRB-approved Informed Consent/Assent Form and be willing to follow study procedures and instructions.
- Age 6 months or older at time of Informed Consent/Assent.
- Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 year
Participants complete weekly symptom diaries and monthly questionnaires to track corneal abrasion symptoms, frequency, and duration.
Weekly remote symptom diary entries and monthly remote questionnaires
Trial Site Locations
Total: 1 location
1
Krystal Biotech
Pittsburgh, Pennsylvania, United States, 15203
Actively Recruiting
Research Team
D
David Chien, MD
B
Brittani Agostini, RN, CCRC
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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