Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Not Applicable

Withdrawn

• Conditions: Epidermolysis Bullosa

• Registration Number: NCT01454687
• Lead Sponsor: Stanford University

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10
• Study First Submitted: 2011-10-13
• Study First Submitted QC: 2011-10-17
• Study First Posted: 2011-10-19
• Status Verified: 2014-04
• Primary Completion: 2014-04
• Last Update Submitted: 2014-04-10
• Last Update Posted: 2014-04-11

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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

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