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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6+
SponsorKrystal Biotech, Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

None

Trial population

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

Trial design

Design model

Case-only

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

1

Corneal abrasion frequency

Frequency of corneal abrasion symptoms

Time frame
Baseline through up to 1 year

Secondary outcomes

1

Epidermolysis Bullosa Eye Disease Index Symptom Score

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

Time frame
Baseline through up to 1 year

Other outcomes

Sponsors and contacts

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