A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

Recruiting

• Conditions: Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa Dystrophica, Recessive; Epidermolysis Bullosa Dystrophica Dominans

• Registration Number: NCT06563414
• Lead Sponsor: Krystal Biotech, Inc.

Brief Summary

This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Corneal abrasion symptomology, frequency, and outcomes will be evaluated for up to 6 months.

Detailed Description

The objective of this study is to characterize the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Patients will be followed via remote weekly symptom diaries and monthly questionnaires to assess corneal abrasion symptoms, frequency, and duration.

At designated clinical sites, subjects may also have the option to participate in a concurrent substudy with clinical assessments including slit lamp exam with fluorescein staining, imaging, and measurement of visual acuity.

Patients are followed for up to 6 months.

Study Timeline

• Status Verified: 2024-08
• Study Start: 2024-08-02
• Study First Submitted: 2024-08-19
• Study First Submitted QC: 2024-08-19
• Last Update Submitted: 2024-08-19
• Study First Posted: 2024-08-20
• Last Update Posted: 2024-08-21
• Primary Completion: 2025-01
• Study Completion: 2025-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. The subject or their respective legal guardian must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent/Assent Form and must be able to and willing to follow study procedures and instructions.
2. Age 6 months or older at time of Informed Consent/Assent.
3. Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Corneal abrasion frequency	Baseline through 6 months	Frequency of corneal abrasions

Secondary Outcome Measures

Name	Time	Method
Epidermolysis Bullosa Eye Disease Index Symptom Score	Baseline through 6 months	Symptom score for Epidermolysis Bullosa related eye problems, 0 to 30 (higher score indicates worse symptoms)

Trial Locations

Locations (1)

Krystal Biotech; 🇺🇸 Pittsburgh, Pennsylvania, United States