Trial document





This study has been imported from ClinicalTrials.gov without additional data checks.
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  DRKS00006565

Trial Description

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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)

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Trial Acronym

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URL of the Trial

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Brief Summary in Lay Language

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.

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Brief Summary in Scientific Language

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Organizational Data

  •   DRKS00006565
  •   2014/08/01
  •   2014/06/04
  •   no
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Secondary IDs

  •   NCT02178969  (ClinicalTrials.gov)
  •   SHP-608-002  (Shire)
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Health Condition or Problem studied

  •   Dystrophic Epidermolysis Bullosa
  •   Q81 -  Epidermolysis bullosa
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Interventions/Observational Groups

  • [---]*
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Characteristics

  •   Non-interventional
  •   Observational study
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  •   N/A
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Primary Outcome

- Wound Surface Area (WSA) of patient- and investigator-selected wounds; time frame: Over 4 weeks
- Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB; time frame: Over 4 weeks

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Secondary Outcome

- The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs); time frame: Over 4 weeks

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Countries of Recruitment

  •   United States
  •   Australia
  •   Austria
  •   Czech Republic
  •   France
  •   Germany
  •   Italy
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Locations of Recruitment

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Recruitment

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  •   2014/06/30
  •   30
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Both, male and female
  •   no minimum age
  •   no maximum age
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Additional 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.

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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.

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Addresses

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    • Shire
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    • Northwestern University
    • Amy Paller, MD 
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    • Northwestern University
    • Amy Paller, MD 
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Sources of Monetary or Material Support

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    • Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
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Status

  •   Recruiting complete, follow-up complete
  •   2015/03/01
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Trial Publications, Results and other Documents

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov required adjustments. For full details please see the DRKS FAQs .
  •   3
  •   2015/05/04
* This entry means the parameter is not applicable or has not been set.