The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
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ClinicalTrials.gov Identifier: NCT04214002 |
Recruitment Status :
Withdrawn
(Due to the COVID-19 Pandemic, the study was withdrawn.)
First Posted : December 30, 2019
Last Update Posted : September 17, 2021
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Condition or disease |
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Dystrophic Epidermolysis Bullosa DEB - Dystrophic Epidermolysis Bullosa |
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 Type : | Observational |
Actual Enrollment : | 0 participants |
Observational Model: | Case-Only |
Time Perspective: | Prospective |
Official Title: | A Prospective Study to Evaluate the Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB) |
Estimated Study Start Date : | January 2020 |
Actual Primary Completion Date : | February 24, 2020 |
Actual Study Completion Date : | February 24, 2020 |
- Change in wound surface area [ Time Frame: Up to four months ]Percent change in wound surface area from baseline will be measured from images uploaded to an imaging application.
- Time to Closure [ Time Frame: Up to four months ]The time it takes for a wound to completely close.
- Duration of wound closure [ Time Frame: Up to four months ]If a closure occurs, the time a wound remains epithelialized after the first, and any subsequent closures.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Months and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04214002
United States, California | |
Stanford University | |
Redwood City, California, United States, 94063 |
Responsible Party: | Krystal Biotech, Inc. |
ClinicalTrials.gov Identifier: | NCT04214002 |
Other Study ID Numbers: |
KB-WM-01 |
First Posted: | December 30, 2019 Key Record Dates |
Last Update Posted: | September 17, 2021 |
Last Verified: | September 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
DEB Dystrophic Epidermolysis Bullosa COL7A1 |
Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic |
Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous Collagen Diseases Connective Tissue Diseases |