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Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

This study is currently recruiting participants.
Verified December 2012 by Stanford University
Sponsor:
Information provided by (Responsible Party):
Alfred Lane, Stanford University
ClinicalTrials.gov Identifier:
NCT01454687
First received: October 13, 2011
Last updated: December 12, 2012
Last verified: December 2012
History of Changes
  Purpose

The term epidermolysis bullosa (EB) is used to describe a group of genetic skin diseases associated with skin weakness, blisters, and chronic wounds. "Revertant mosaicism" means that there are two genetically different populations of cells due to spontaneous mutations. Some EB patients have normal, non-fragile skin patches which may be areas of revertant mosaicism. In the revertant areas, the proteins function normally, like non-EB skin. In this study, we plan to culture cells from the revertant areas and graft them on to the wounded areas.


Condition Intervention
Epidermolysis Bullosa
 Procedure: Grafting of Autologous Cultured Revertant Keratinocytes

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Stanford University:

Primary Outcome Measures:
  • Expression of the correct protein at the basement membrane zone [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
  • Engraftment and healing of wounds with genetically revertant keratinocytes [ Time Frame: Week 52 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Engraftment and healing of wounds with genetically revertant keratinocytes [ Time Frame: Week 8-12 ] [ Designated as safety issue: No ]
  • Engraftment and healing of wounds with genetically revertant keratinocytes [ Time Frame: Week 25 ] [ Designated as safety issue: No ]
  • Expression of correct protein at the basement membrane zone [ Time Frame: Week 8-12 ] [ Designated as safety issue: No ]
  • Expression of the correct protein at the basement membrane zone [ Time Frame: Week 25 ] [ Designated as safety issue: No ]

Estimated Enrollment: 5
Study Start Date: October 2011
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: Grafting of Autologous Cultured Revertant Keratinocytes
    Grafting of two to four epidermal sheets 40cm2 - 50cm2 onto wounded areas
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of EB (simplex, junctional or dystrophic)
  • Areas of revertant skin that has been confirmed by biopsy
  • 18 years or older subject willing and able to give consent
  • Confirmation of EB diagnosis by immunofluorescence (IF), electron microscopy (EM), and genetic testing confirming mutation
  • At least 100 to 200 cm2 of open erosions on the trunk and/or extremities suitable for skin grafting
  • Able to undergo adequate anesthesia to allow grafting procedures to take place

Exclusion Criteria:

  • Medical instability limiting ability to travel to Stanford University Medical Center
  • The presence of medical illness expected to complicate participation and/or compromise the safety of this technique
  • Active infection with HIV, hepatitis B, or hepatitis C
  • Active infection in the area that will undergo grafting
  • Evidence of a systemic infection
  • Current evidence or a history of skin cancer in the area that will undergo grafting
  • Active drug or alcohol addiction
  • Hypersensitivity to vancomycin or amikacin
  • Receipt of chemical or biological study product for the specific treatment ofEB in the past six months
  • Positive pregnancy test or breast-feeding
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01454687

Contacts
Contact: Emily S Gorell, MS 6507217166 egorell@stanford.edu

Locations
United States, California
Stanford School of Medicine Recruiting
Palo Alto, California, United States, 94304
Contact: Emily S Gorell, MS     650-721-7166     egorell@stanford.edu    
Sub-Investigator: M. Peter Marinkovich, MD            
Sub-Investigator: Zurab Siprashvili, PhD            
Sub-Investigator: Phuong Khuu, MD            
Sub-Investigator: Thomas Leung, MD, PhD            
Principal Investigator: Alfred Lane, MD            
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Alfred Lane, MD Stanford University
  More Information

No publications provided

Responsible Party: Alfred Lane, Professor, Departments of Dermatology and Pediatrics, Stanford University
ClinicalTrials.gov Identifier: NCT01454687     History of Changes
Other Study ID Numbers: IRB # 22005
Study First Received: October 13, 2011
Last Updated: December 12, 2012
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous

ClinicalTrials.gov processed this record on June 17, 2013