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

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Brief Title

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)

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.
    


Study Type

Observational


Primary Outcome

Change in wound surface area

Secondary Outcome

 Time to Closure

Condition

Dystrophic Epidermolysis Bullosa



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

0

Start Date

January 2020

Completion Date

February 24, 2020

Primary Completion Date

February 24, 2020

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.
      

Gender

All

Ages

6 Months - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04214002

Organization ID

KB-WM-01


Responsible Party

Sponsor

Study Sponsor

Krystal Biotech, Inc.

Collaborators

 Stanford University

Study Sponsor

, , 


Verification Date

September 2021