Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa

Withdrawn

• Conditions: Dystrophic Epidermolysis Bullosa

• Registration Number: NCT01768026
• Lead Sponsor: Lotus Tissue Repair, Inc.

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 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.

Study Timeline

• Study First Submitted: 2013-01-10
• Study First Submitted QC: 2013-01-11
• Study First Posted: 2013-01-15
• Status Verified: 2013-02
• Study Start: 2013-02
• Last Update Submitted: 2013-05-20
• Last Update Posted: 2013-05-21
• Primary Completion: 2014-09
• Study Completion: 2014-09

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Characterize the progression of disease severity in subjects with DEB over 6 - 12 months.	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.

Trial Locations

Locations (1)

Stanford University School of Medicine; 🇺🇸 Palo Alto, California, United States