Clinical trial NCT04214002: The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
Official title: A Prospective Study to Evaluate the Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Dystrophic Epidermolysis Bullosa
DEB - Dystrophic Epidermolysis Bullosa
Keywords: DEB
Dystrophic Epidermolysis Bullosa
COL7A1
Lead sponsor: Krystal Biotech, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Stanford University
First posted: 2019-12-30
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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.

Eligibility 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.

Primary outcomes:
measure: Change in wound surface area
timeFrame: Up to four months
Secondary outcomes:
measure: Time to Closure
timeFrame: Up to four months
measure: Duration of wound closure
timeFrame: Up to four months
Other outcomes:
Locations:
city: Redwood City
country: United States
facility: Stanford University
state: California
Study officials:
References: