Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

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.

Study Overview

• Status: Withdrawn
• Conditions: Epidermolysis Bullosa
• Intervention / Treatment: Procedure: Grafting of Autologous Cultured Revertant Keratinocytes

• Study Type: Interventional
• Phase: Not Applicable

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Expression of the correct protein at the basement membrane zone	Week 52
Engraftment and healing of wounds with genetically revertant keratinocytes	Week 52

Secondary Outcome Measures

Outcome Measure	Time Frame
Engraftment and healing of wounds with genetically revertant keratinocytes	Week 8-12
Engraftment and healing of wounds with genetically revertant keratinocytes	Week 25
Expression of correct protein at the basement membrane zone	Week 8-12
Expression of the correct protein at the basement membrane zone	Week 25

Collaborators and Investigators

• Sponsor: Stanford University

Study record dates

Study Major Dates

• Study Start: October 1, 2011
• Primary Completion (Actual): April 1, 2014

Study Registration Dates

• First Submitted: October 13, 2011
• First Submitted That Met QC Criteria: October 17, 2011
• First Posted (Estimate): October 19, 2011

Study Record Updates

• Last Update Posted (Estimate): April 11, 2014
• Last Update Submitted That Met QC Criteria: April 10, 2014
• Last Verified: April 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa
• Other Study ID Numbers: IRB # 22005