Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa

Dystrophic Epidermolysis Bullosa Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01768026
• Other study ID #: DEB-101-12
• Status: Withdrawn
• Phase: N/A
• First received: January 10, 2013
• Last updated: May 20, 2013
• Start date: February 2013
• Est. completion date: September 2014

Study information

• Verified date: February 2013
• Source: Lotus Tissue Repair, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: September 2014
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Eligibility Criteria

• Subjects of any age (newborns included) may participate

• Subjects over 18 years of age and parent(s)/legal guardian(s) of subjects <18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors at least 7 years of age

• Subjects must have a documented diagnosis of DEB based on clinical presentation and either skin biopsy results showing an absence or reduction in C7 or anchoring fibrils or genetic analysis showing a mutation in collagen, type VII, alpha 1 (Col7A1); alternatively, subjects must have a clinical diagnosis of DEB and a documented diagnosis of DEB (as above) in a first degree relative

• No experimental systemic therapy for DEB including, but not limited to, bone marrow transplantation, systemic immune suppression, or experimental therapies that involve live cells which have the potential for systemic spread such as gene transfer, stem cell infusions or other cell type injections

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

Locations

Country	Name	City	State
United States	Stanford University School of Medicine	Palo Alto	California

Sponsors (1)

Lead Sponsor	Collaborator
Lotus Tissue Repair, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Characterize the progression of disease severity in subjects with DEB over 6 - 12 months.	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.	One year period	No

External Links

•   Dystrophic Epidermolysis Bullosa Research Association International
•   Dystrophic Epidermolysis Bullosa Research Association of America (DEBRA)
•   Lotus Tissue Repair, Inc.