Short Term Observational Study in DEB Patients

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)

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT02178969

Enrollment

Registered

2014-07-01

Start date

2014-06-24

Completion date

2015-02-23

Last updated

2021-03-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Dystrophic Epidermolysis Bullosa

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

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

Secondary

Measure	Time frame
The number, severity and relationship to study procedures of adverse events (AEs) and serious AEs (SAEs)	Over 4 weeks

Countries

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

Outcome results