NCT01768026

Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa

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

• Status: Withdrawn
• Phase: —
• Study type: Observational
• Enrollment: 0 (actual)
• Sponsor: Lotus Tissue Repair, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Dystrophic Epidermolysis Bullosa

Timeline

• Start date: 2013-02-01
• Primary completion: 2014-09-01
• Completion: 2014-09-01
• First posted: 2013-01-15
• Last updated: 2013-05-21

Locations

1 site across 1 country: United States