The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)

NCT ID: NCT04214002

Last Updated: 2021-09-17

Basic Information

• Recruitment Status: WITHDRAWN
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-01-31
• Study Completion Date: 2020-02-24

Brief Summary

This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC (previously KB103). Wound recurrence and wound size will be evaluated for up to four months.

Detailed Description

The objective of this study is to analyze the selected wounds' natural history prior to including the selected wounds in the evaluation of safety and efficacy in Krystal Biotech's Phase III protocol of B-VEC.

Subjects will be enrolled upon obtaining consent and meeting eligibility criteria.

Patient Screening is done on-site, followed by the imaging of at least 1 wound, but up to 15 wounds. All remaining imaging will be performed remotely with a smartphone or tablet application.

Patients are on-trial for approximately four months.

Conditions

Dystrophic Epidermolysis Bullosa; DEB - Dystrophic Epidermolysis Bullosa

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Written informed consent is provided. Patients 18 years of age and older, and parent(s)/legal guardian(s) of patients younger than 18 years of age, must provide written informed consent prior to participating in the study; additionally, informed assent will be obtained from patients younger than 18 years of age as specified by local requirements.
• Patient must have a documented diagnosis of DEB based on genetic analysis showing a mutation in COL7A1 or, in the opinion of the investigator, a confirmed diagnosis of DEB based on histologic criteria (antigen mapping or electron microscopy).
• Age: 6 months and older
• Patient must have at least 1 wound that is suitable for imaging, in the opinion of the investigator, at the time of enrollment.
• Patient is willing and able to undergo the protocol-specified procedures.

Exclusion Criteria

• In the opinion of the investigator, inclusion poses an unacceptable risk to the patient or interpretation of these study data.

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stanford University

OTHER

Sponsor Role: collaborator

Krystal Biotech, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Stanford University

Redwood City, California, United States

Countries

United States

Other Identifiers

KB-WM-01

Identifier Type: -

Identifier Source: org_study_id