Trial record 7 of 16 for:
"Epidermolysis Bullosa Dystrophica" OR "dystrophic epidermolysis bullosa"
Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
This study has been terminated.
(Study was suspended until FDA approved the IND. Study has since been terminated and a new screening process has been implemented.)
Information provided by (Responsible Party):
Alfred Lane, Stanford University
First received: September 20, 2007
Last updated: December 12, 2012
Last verified: December 2012
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.
Epidermolysis Bullosa Dystrophica
||Observational Model: Cohort
Time Perspective: Cross-Sectional
||Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
Primary Outcome Measures:
- Disease documentation [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Documentation of disease
- documentation of disease [ Time Frame: 1 year ] [ Designated as safety issue: No ]
documentation of disease
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||August 2008 (Final data collection date for primary outcome measure)
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.
|Ages Eligible for Study:
||18 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00533572
|Stanford University School of Medicine
|Stanford, California, United States, 94305 |
||Alfred T Lane
No publications provided
||Alfred Lane, Professor of Dermatology and Pediatrics, Stanford University
History of Changes
|Other Study ID Numbers:
|Study First Received:
||September 20, 2007
||December 12, 2012
||United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on August 26, 2014
Epidermolysis Bullosa Dystrophica
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous
Connective Tissue Diseases