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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02178969




Registration number
NCT02178969
Ethics application status
Date submitted
4/06/2014
Date registered
1/07/2014
Date last updated
17/03/2021

Titles & IDs
Public title
Short Term Observational Study in DEB Patients
Scientific 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)
Secondary ID [1] 0 0
SHP-608-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dystrophic Epidermolysis Bullosa 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Wound Surface Area (WSA) of patient- and investigator-selected wounds
Timepoint [1] 0 0
Over 4 weeks
Primary outcome [2] 0 0
Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB
Timepoint [2] 0 0
Over 4 weeks
Secondary outcome [1] 0 0
The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)
Timepoint [1] 0 0
Over 4 weeks

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/06/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/02/2015
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Premier Specialists Pty Ltd - Kogarah
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
Austria
State/province [4] 0 0
Salzburg
Country [5] 0 0
Czechia
State/province [5] 0 0
Brno
Country [6] 0 0
France
State/province [6] 0 0
Paris
Country [7] 0 0
Germany
State/province [7] 0 0
Freiburg im Breisgau
Country [8] 0 0
Italy
State/province [8] 0 0
Roma
Country [9] 0 0
Italy
State/province [9] 0 0
Rome

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Shire
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02178969
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02178969