Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa
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ClinicalTrials.gov Identifier: NCT01768026 |
Recruitment Status
:
Withdrawn
First Posted
: January 15, 2013
Last Update Posted
: May 21, 2013
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Condition or disease |
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Dystrophic Epidermolysis Bullosa |
Study Type : | Observational |
Actual Enrollment : | 0 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | A Prospective, Longitudinal Assessment of Disease Severity in Subjects With Dystrophic Epidermolysis Bullosa (DEB) |
Study Start Date : | February 2013 |
Estimated Primary Completion Date : | September 2014 |
Estimated Study Completion Date : | September 2014 |

Group/Cohort |
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No treatment
Subjects diagnosed with Dystrophic Epidermolysis Bullosa
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- 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.

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Ages Eligible for Study: | Child, Adult, Senior |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01768026
United States, California | |
Stanford University School of Medicine | |
Palo Alto, California, United States, 94304 |
Study Director: | Hal Landy, MD | Lotus Tissue Repair, Inc. |
Additional Information:
Responsible Party: | Lotus Tissue Repair, Inc. |
ClinicalTrials.gov Identifier: | NCT01768026 History of Changes |
Other Study ID Numbers: |
DEB-101-12 |
First Posted: | January 15, 2013 Key Record Dates |
Last Update Posted: | May 21, 2013 |
Last Verified: | February 2013 |
Keywords provided by Lotus Tissue Repair, Inc.:
Dystrophic Epidermolysis Bullosa Type VII Collagen QOL Evaluation in Epidermolysis Bullosa Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa Birmingham Epidermolysis Bullosa Severity Score |
Additional relevant MeSH terms:
Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic |
Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous Collagen Diseases Connective Tissue Diseases |