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

July 1, 2025 updated by: Krystal Biotech, Inc.
This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

40

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with Dystrophic Epidermolysis Bullosa, aged 6 months or older.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Corneal abrasion frequency
Time Frame: Baseline through up to 1 year
Frequency of corneal abrasion symptoms
Baseline through up to 1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
Baseline through up to 1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Krystal Biotech, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 2, 2024

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

August 19, 2024

First Submitted That Met QC Criteria

August 19, 2024

First Posted (Actual)

August 20, 2024

Study Record Updates

Last Update Posted (Actual)

July 2, 2025

Last Update Submitted That Met QC Criteria

July 1, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NHS

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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