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NCT Number: NCT06563414

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

This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).

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Key information

Conditions

Epidermolysis Bullosa Dystrophica Collagen Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Corneal Diseases Corneal Injuries Craniocerebral Trauma Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Dominans Epidermolysis Bullosa Dystrophica, Recessive Eye Diseases Eye Injuries Facial Injuries Genetic Diseases, Inborn Nervous System Diseases Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases Trauma, Nervous System Wounds and Injuries

Age range

6 month and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Krystal Biotech

Pittsburgh, Pennsylvania, 15203, United States

Location status: Recruiting

Location contact

Brittani Agostini

CONTACT

[email protected]

412-586-5830

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • 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.

Treatment and study plan

Primary outcomes

  1. Corneal abrasion frequency

    Time frame: Baseline through up to 1 year

    Frequency of corneal abrasion symptoms

Secondary outcomes

  1. Epidermolysis Bullosa Eye Disease Index Symptom Score

    Time frame: Baseline through up to 1 year

    Symptom score for Epidermolysis Bullosa related eye problems, 0 to 30 (higher score indicates worse symptoms)

Study contacts

Contact information is provided by the study sponsor or research team.

Brittani Agostini, RN, CCRC

CONTACT

[email protected]

412-586-5830

David Chien, MD

CONTACT

[email protected]

412-586-5830

Sponsors and collaborators

Lead sponsor

Krystal Biotech, Inc.

Industry

Registry information

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Aug 20, 2024
Registry last updated
Jul 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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