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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.)
Sponsor:
ClinicalTrials.gov Identifier:
NCT00533572
First Posted: September 21, 2007
Last Update Posted: May 27, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alfred Lane, Stanford University
  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
Epidermolysis Bullosa Dystrophica

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer

Resource links provided by NLM:


Further study details as provided by Alfred Lane, Stanford University:

Primary Outcome Measures:
  • Disease documentation [ Time Frame: 1 year ]
    Documentation of disease

  • documentation of disease [ Time Frame: 1 year ]
    documentation of disease


Enrollment: 1
Study Start Date: August 2007
Study Completion Date: August 2008
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
Detailed Description:
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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adults with RDEB
Criteria

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
Information from the National Library of Medicine

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): NCT00533572


Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Alfred T Lane Stanford University
  More Information

Responsible Party: Alfred Lane, Professor of Dermatology and of Pediatricsy, Emeritus, Stanford University
ClinicalTrials.gov Identifier: NCT00533572     History of Changes
Other Study ID Numbers: 97822
8557
First Submitted: September 20, 2007
First Posted: September 21, 2007
Last Update Posted: May 27, 2015
Last Verified: May 2015

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