Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Epidermolysis Bullosa Clinical Trial

Official title:

Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01454687
• Other study ID #: IRB # 22005
• Status: Withdrawn
• Phase: N/A
• First received: October 13, 2011
• Last updated: April 10, 2014
• Start date: October 2011

Study information

• Verified date: April 2014
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. primary completion date: April 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Epidermolysis Bullosa

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Stanford University

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Expression of the correct protein at the basement membrane zone		Week 52	No
Primary	Engraftment and healing of wounds with genetically revertant keratinocytes		Week 52	No
Secondary	Engraftment and healing of wounds with genetically revertant keratinocytes		Week 8-12	No
Secondary	Engraftment and healing of wounds with genetically revertant keratinocytes		Week 25	No
Secondary	Expression of correct protein at the basement membrane zone		Week 8-12	No
Secondary	Expression of the correct protein at the basement membrane zone		Week 25	No