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Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Primary Purpose

Epidermolysis Bullosa

Status

Withdrawn

Phase

Not Applicable

Locations

Study Type

Interventional

Intervention

Grafting of Autologous Cultured Revertant Keratinocytes

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Outcomes

Primary Outcome Measures

Expression of the correct protein at the basement membrane zone

Engraftment and healing of wounds with genetically revertant keratinocytes

Secondary Outcome Measures

Engraftment and healing of wounds with genetically revertant keratinocytes

Expression of correct protein at the basement membrane zone

Expression of the correct protein at the basement membrane zone

Full Information

NCT ID

NCT01454687

First Posted

October 13, 2011

Last Updated

April 10, 2014

Sponsor

Stanford University

1. Study Identification

Unique Protocol Identification Number

NCT01454687

Brief Title

Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Official Title

Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Study Type

Interventional

2. Study Status

Record Verification Date

April 2014

Overall Recruitment Status

Withdrawn

Study Start Date

October 2011 (undefined)

Primary Completion Date

April 2014 (Actual)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Stanford University

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Epidermolysis Bullosa

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Intervention Type

Procedure

Intervention Name(s)

Grafting of Autologous Cultured Revertant Keratinocytes

Intervention Description

Grafting of two to four epidermal sheets 40cm2 - 50cm2 onto wounded areas

Primary Outcome Measure Information:

Title

Expression of the correct protein at the basement membrane zone

Time Frame

Week 52

Title

Engraftment and healing of wounds with genetically revertant keratinocytes

Time Frame

Week 52

Secondary Outcome Measure Information:

Title

Engraftment and healing of wounds with genetically revertant keratinocytes

Time Frame

Week 8-12

Title

Engraftment and healing of wounds with genetically revertant keratinocytes

Time Frame

Week 25

Title

Expression of correct protein at the basement membrane zone

Time Frame

Week 8-12

Title

Expression of the correct protein at the basement membrane zone

Time Frame

Week 25

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Alfred Lane, MD

Organizational Affiliation

Stanford University

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

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