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Trial record 8 of 14 for:    RDEB | California, United States

Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer

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.
 
ClinicalTrials.gov Identifier: NCT00533572
Recruitment Status : Terminated (Study was suspended until FDA approved the IND. Study has since been terminated and a new screening process has been implemented.)
First Posted : September 21, 2007
Last Update Posted : May 27, 2015
Sponsor:
Information provided by (Responsible Party):
Alfred Lane, Stanford University

Tracking Information
First Submitted Date September 20, 2007
First Posted Date September 21, 2007
Last Update Posted Date May 27, 2015
Study Start Date August 2007
Actual Primary Completion Date August 2008   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 12, 2012)
  • Disease documentation [ Time Frame: 1 year ]
    Documentation of disease
  • documentation of disease [ Time Frame: 1 year ]
    documentation of disease
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
Official Title Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
Brief Summary 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.
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.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Adults with RDEB
Condition Epidermolysis Bullosa Dystrophica
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Terminated
Actual Enrollment
 (submitted: December 12, 2012)
1
Original Estimated Enrollment
 (submitted: September 20, 2007)
34
Actual Study Completion Date August 2008
Actual Primary Completion Date August 2008   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00533572
Other Study ID Numbers 97822
8557
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Alfred Lane, Stanford University
Study Sponsor Stanford University
Collaborators Not Provided
Investigators
Principal Investigator: Alfred T Lane Stanford University
PRS Account Stanford University
Verification Date May 2015