Clinical Trial: Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Observational

Official Title: A Prospective, Longitudinal Assessment of Disease Severity in Subjects With Dystrophic Epidermolysis Bullosa (DEB)

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Lotus Tissue Repair, Inc.

Current Primary Outcome: Characterize the progression of disease severity in subjects with DEB over 6 - 12 months. [ Time Frame: One year period ]

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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Lotus Tissue Repair, Inc.

Dates:
Date Received: January 10, 2013
Date Started: February 2013
Date Completion: September 2014
Last Updated: May 20, 2013
Last Verified: February 2013