Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa
The objective of this study is to characterize the extent and severity of disease in subjects with DEB and the progression of disease over a timeframe relevant to interventional studies. The data from this study will be used to inform the study design and address statistical considerations of future treatment protocols.
Dystrophic Epidermolysis Bullosa
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||A Prospective, Longitudinal Assessment of Disease Severity in Subjects With Dystrophic Epidermolysis Bullosa (DEB)|
- Characterize the progression of disease severity in subjects with DEB over 6 - 12 months. [ Time Frame: One year period ] [ Designated as safety issue: No ]Disease severity and its impact on quality of life and function will be investigated over a one year period at the following timepoints: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment.
|Study Start Date:||February 2013|
|Estimated Study Completion Date:||September 2014|
|Estimated Primary Completion Date:||September 2014 (Final data collection date for primary outcome measure)|
Subjects diagnosed with Dystrophic Epidermolysis Bullosa
This is a prospective, multicenter, multinational, longitudinal assessment of disease severity in subjects with DEB. Subjects with either dominant or recessive DEB (dominant dystrophic epidermolysis bullosa (DDEB) and recessive dystrophic epidermolysis bullosa (RDEB), respectively) will be assessed four times over a one year period: upon enrollment, and at 1 to 2 weeks and 6 and 12 months after enrollment. All subjects with either DDEB or RDEB that meet the study entry criteria will be offered participation in the study, provided they can be accommodated within the anticipated study timeline. In addition to their usual clinic assessment, subjects will have a quantitative evaluation of skin involvement and will be asked to fill out questionnaires that measure among other things disease severity, QOL, pain, pruritus, and medical and family histories.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01768026
|United States, California|
|Stanford University School of Medicine|
|Palo Alto, California, United States, 94304|
|Study Director:||Hal Landy, MD||Lotus Tissue Repair, Inc.|