Short Term Observational Study in DEB Patients

Completed

Conditions studied: Dystrophic Epidermolysis Bullosa

In brief

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.

Key facts

Study ID

NCT02178969

Run by

Shire

People needed

29

Starts

2014-06-24

Expected to finish

2015-02-23

Last updated by the study team

2021-03-17

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).
• Patient must have at least 5 wounds that are 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.

You may not qualify if…

• 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.
• Patient has squamous cell carcinoma with evidence of locally invasive disease or distant metastases.
• Patient is pregnant.

Where it is running

• Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States
• Columbia University Medical Center — New York, New York, United States
• Cincinnati Childrens Hospital Medical Center — Cincinnati, Ohio, United States
• Premier Specialists Pty Ltd — Kogarah, Australia
• Salzburger Landeskliniken — Salzburg, Austria
• Fakultni nemocnice Brno — Brno, Czechia
• Service de Génétique et INSERM UMR 1163, Laboratoire de Génétique des maladies cutanées - Institut Imagine — Paris, France
• Universitätsklinikum Freiburg — Freiburg im Breisgau, Germany
• Ospedale Pediatrico Bambino Gesù — Roma, Italy
• Istituto Dermopatico Dell'immacolata IRCCS — Rome, Italy

Full record on ClinicalTrials.gov

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