A Natural History Study of Corneal Abrasions in Patients... | NCT06563414

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

NCT06563414

Sponsor

Krystal Biotech, Inc.

Status

Recruiting

Last Update Posted

Jul 2, 2025Actual

Enrollment

40Estimated

Phase

Not provided

Conditions

Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa Dystrophica, Recessive
Epidermolysis Bullosa Dystrophica Dominans

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 2, 2024Actual

Primary Completion Date

Dec 2025Estimated

Completion Date

Dec 2025Estimated

First Submitted Date

Aug 19, 2024

First Submission Date that Met QC Criteria

Aug 19, 2024

First Posted Date

Aug 20, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 1, 2025

Last Update Posted Date

Jul 2, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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).

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

40Estimated

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Epidermolysis Bullosa Eye Disease Index Symptom Score	Symptom score for Epidermolysis Bullosa related eye problems, 0 to 30 (higher score indicates worse symptoms)	Baseline through up to 1 year

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
David Chien, MD	Contact	412-586-5830		dchien@krystalbio.com
Brittani Agostini, RN, CCRC	Contact	412-586-5830		bagostini@krystalbio.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Krystal Biotech	Recruiting	Pittsburgh	Pennsylvania	15203	United States

Condition Browse Module

MeSH Terms

ID	Term
D016108	Epidermolysis Bullosa Dystrophica
D065306	Corneal Injuries

Ancestor Terms

ID	Term
D004820	Epidermolysis Bullosa
D012868	Skin Abnormalities
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided