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| Sponsored by: |
Stanford University |
|---|---|
| Information provided by: | Stanford University |
| ClinicalTrials.gov Identifier: | NCT00533572 |
Purpose
Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe inherited blistering skin disease caused by absence of type VII collagen. Patients with RDEB develop large, severly painful blisters and open wounds from minor trauma to their skin. In the future, we hope to start a gene transfer study on a specific group of RDEB subjects and we are screening subjects for that potential trial now.
| Condition |
|---|
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Epidermolysis Bullosa Dystrophica |
| Study Type: | Observational |
| Official Title: | Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer |
| Estimated Enrollment: | 34 |
| Study Start Date: | August 2007 |
This study is a preparation for a gene transfer trial. In the gene transfer trial, we will transfect autologous RDEB keratinocytes with a type VII collagen containing retrovirus vector. We then plan to graft the genetically engineered and corrected autologous keratinocytes back on to wounds of the RDEB subject. We expect the grafts to attach as normal keratinocytes and replace the damaged skin with skin that is able to make type VII collagen and anchor normally to the dermis. We are trying to define the number of subjects 18 or older who meet the criteria for the gene transfer trial. We are offering the subjects the opportunity to be evaluated for the potential trial.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:1. Clinical diagnosis of RDEB by local dermatologist 2. 18 years of age or more and willing to give consent 3. Estimated to have at least 100 to 200 sq. cm. areas of open erosions on the trunk or extremities suitable for skin grafting 4. Ability to undergo adequate anesthesia to allow grafting procedures to take place 5. Parents are alive, do not have EB, and they are willing to give consent for genetic testing Exclusion Criteria:1. Medical instability limiting ability to travel to Stanford University Medical Center 2. Participation in another clinical trial without prior approval 3. The presence of medical illness expected to complicate participation and/or compromise the safety of this technique
Contacts and Locations| United States, California | |
| Stanford University School of Medicine | |
| Stanford, California, United States, 94305 | |
| Principal Investigator: | Alfred T Lane | Stanford University |
More Information
| Study ID Numbers: | 97822 |
| Study First Received: | September 20, 2007 |
| Last Updated: | June 25, 2008 |
| ClinicalTrials.gov Identifier: | NCT00533572 |
| Health Authority: | United States: Food and Drug Administration |
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Epidermolysis bullosa Collagen Diseases Skin Diseases, Vesiculobullous Genetic Diseases, Inborn Skin Diseases Connective Tissue Diseases |
Skin Abnormalities Epidermolysis Bullosa Dystrophica Epidermolysis Bullosa Dystrophic epidermolysis bullosa Congenital Abnormalities Skin Diseases, Genetic |
