Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Study Overview
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Brief Summary
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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.

Official Title
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Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Conditions
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Epidermolysis Bullosa

Intervention / Treatment
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* Procedure: Grafting of Autologous Cultured Revertant Keratinocytes


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Procedure: Grafting of Autologous Cultured Revertant Keratinocytes|Grafting of two to four epidermal sheets 40cm2 - 50cm2 onto wounded areas|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | Measure Description | 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 |