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

Dystrophic Epidermolysis Bullosa Clinical Trial

Official title:

A Prospective Study to Evaluate the Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04214002
• Other study ID #: KB-WM-01
• Status: Withdrawn
• Start date: January 2020
• Est. completion date: February 24, 2020

Study information

• Verified date: September 2021
• Source: Krystal Biotech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: February 24, 2020
• Est. primary completion date: February 24, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months and older

Eligibility

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.

Related Conditions & MeSH terms

• DEB - Dystrophic Epidermolysis Bullosa
• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

Locations

Country	Name	City	State
United States	Stanford University	Redwood City	California

Sponsors (2)

Lead Sponsor	Collaborator
Krystal Biotech, Inc.	Stanford University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in wound surface area	Percent change in wound surface area from baseline will be measured from images uploaded to an imaging application.	Up to four months
Secondary	Time to Closure	The time it takes for a wound to completely close.	Up to four months
Secondary	Duration of wound closure	If a closure occurs, the time a wound remains epithelialized after the first, and any subsequent closures.	Up to four months