Short Term Observational Study in DEB Patients

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Brief Title

Short Term Observational Study in DEB Patients

Official Title

A Prospective Short-term Study to Evaluate Methodologies for the Assessment of Disease Extent, Impact, and Wound Evolution in Patients With Dystrophic Epidermolysis Bullosa (DEB)

Brief Summary

      The purpose of this study is to better understand disease extent and to identify appropriate
      methodologies to evaluate (dystrophic epidermolysis bullosa) DEB in a quantitative and
      qualitative manner.
    



Study Type

Observational


Primary Outcome

Wound Surface Area (WSA) of patient- and investigator-selected wounds

Secondary Outcome

 The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)

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

29

Start Date

June 24, 2014

Completion Date

February 23, 2015

Primary Completion Date

February 23, 2015

Eligibility Criteria

        Inclusion Criteria:

          1. 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.

          2. 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).

          3. Patient must have at least 5 wounds that are suitable for imaging, in the opinion of
             the investigator, at the time of enrollment.

          4. Patient is willing and able to undergo the protocol-specified procedures.

        Exclusion Criteria:

          1. Patient has used or is currently using experimental treatment for DEB including, but
             not limited to, bone marrow transplantation, systemic immune suppression, or
             experimental procedures that involve live cells, with potential for systemic spread
             such as gene transfer, stem cell infusions, or other cell type injections such that,
             in the opinion of the investigator, inclusion poses an unacceptable risk to the
             patient or interpretation of these study data.

          2. Patient has squamous cell carcinoma with evidence of locally invasive disease or
             distant metastases.

          3. Patient is pregnant.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Study Director, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02178969

Organization ID

SHP-608-002


Responsible Party

Sponsor

Study Sponsor

Shire


Study Sponsor

Study Director, Study Director, Takeda


Verification Date

March 2021