Short Term Observational Study in DEB Patients

Dystrophic Epidermolysis Bullosa Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02178969
• Other study ID #: SHP-608-002
• Status: Completed
• Start date: June 24, 2014
• Est. completion date: February 23, 2015

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: February 23, 2015
• Est. primary completion date: February 23, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Czechia,  France,  Germany,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Wound Surface Area (WSA) of patient- and investigator-selected wounds		Over 4 weeks
Primary	Wound Surface Area (WSA) as a percentage of Body Surface Area (BSA) in patients with DEB		Over 4 weeks
Secondary	The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)		Over 4 weeks

External Links

•   To obtain more information on the study, click here/on this link.