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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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Recruitment Status : Withdrawn (Due to the COVID-19 Pandemic, the study was withdrawn.)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
Sponsor:
Collaborator:
Stanford University
Information provided by (Responsible Party):
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.

Condition or disease
Dystrophic Epidermolysis Bullosa DEB - Dystrophic Epidermolysis Bullosa

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.

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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





Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Time to Closure [ Time Frame: Up to four months ]
    The time it takes for a wound to completely close.

  2. 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.



Information from the National Library of Medicine

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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
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.
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.

Information from the National Library of Medicine

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


Locations
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United States, California
Stanford University
Redwood City, California, United States, 94063
Sponsors and Collaborators
Krystal Biotech, Inc.
Stanford University
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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

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Krystal Biotech, Inc.:
DEB
Dystrophic Epidermolysis Bullosa
COL7A1
Additional relevant MeSH terms:
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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