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

Withdrawn

• Conditions: Dystrophic Epidermolysis Bullosa; DEB - Dystrophic Epidermolysis Bullosa

• Registration Number: NCT04214002
• Lead Sponsor: Krystal Biotech, Inc.

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.

Study Timeline

• Study First Submitted: 2019-12-23
• Study First Submitted QC: 2019-12-26
• Study First Posted: 2019-12-30
• Study Start: 2020-01
• Primary Completion: 2020-02-24
• Study Completion: 2020-02-24
• Status Verified: 2021-09
• Last Update Submitted: 2021-09-10
• Last Update Posted: 2021-09-17

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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.

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in wound surface area	Up to four months	Percent change in wound surface area from baseline will be measured from images uploaded to an imaging application.

Secondary Outcome Measures

Name	Time	Method
Time to Closure	Up to four months	The time it takes for a wound to completely close.
Duration of wound closure	Up to four months	If a closure occurs, the time a wound remains epithelialized after the first, and any subsequent closures.

Trial Locations

Locations (1)

Stanford University; 🇺🇸 Redwood City, California, United States