NCT06563414

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).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
40

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Aug 2024

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 2, 2024

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

August 19, 2024

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 20, 2024

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2025

Completed
Last Updated

July 2, 2025

Status Verified

July 1, 2025

Enrollment Period

1.3 years

First QC Date

August 19, 2024

Last Update Submit

July 1, 2025

Conditions

Epidermolysis Bullosa DystrophicaEpidermolysis Bullosa Dystrophica, RecessiveEpidermolysis Bullosa Dystrophica Dominans

Keywords

Dystrophic Epidermolysis BullosaDEBCorneal Abrasions

Outcome Measures

Primary Outcomes (1)

  • Corneal abrasion frequency

    Frequency of corneal abrasion symptoms

    Baseline through up to 1 year

Secondary Outcomes (1)

  • Epidermolysis Bullosa Eye Disease Index Symptom Score

    Baseline through up to 1 year

Eligibility Criteria

Age6 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Krystal Biotech

Pittsburgh, Pennsylvania, 15203, United States

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa DystrophicaCorneal Injuries

Condition Hierarchy (Ancestors)

Epidermolysis BullosaSkin AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, GeneticGenetic Diseases, InbornCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin DiseasesSkin Diseases, VesiculobullousEye InjuriesFacial InjuriesCraniocerebral TraumaTrauma, Nervous SystemNervous System DiseasesCorneal DiseasesEye DiseasesWounds and Injuries

Central Study Contacts

David Chien, MD

CONTACT

412-586-5830dchien@krystalbio.com

Brittani Agostini, RN, CCRC

CONTACT

412-586-5830bagostini@krystalbio.com

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 19, 2024

First Posted

August 20, 2024

Study Start

August 2, 2024

Primary Completion

December 1, 2025

Study Completion

December 1, 2025

Last Updated

July 2, 2025

Record last verified: 2025-07

Locations

US(1)