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

NCT04214002 | Withdrawn

• 1 other identifier: KB-WM-01
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Dystrophic Epidermolysis Bullosa; DEB - Dystrophic Epidermolysis Bullosa

Keywords

DEB; Dystrophic Epidermolysis Bullosa; COL7A1

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Time to Closure

  Up to four months

• Duration of wound closure

  Up to four months

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients must be 6 months of age or older, and have a diagnosis of DEB based on genetic analysis or histologic criteria. In addition, patients must have at least 1 wound that is suitable for imaging in investigator's opinion at time of screening.

You may qualify if:

• 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.

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead
• Stanford University: collaborator

Study Sites (1)

Stanford University

Redwood City, California, 94063, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Design

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

Study Record Dates

• First Submitted: December 23, 2019
• First Posted: December 30, 2019
• Study Start: January 1, 2020
• Primary Completion: February 24, 2020
• Study Completion: February 24, 2020
• Last Updated: September 17, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)