Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 主要终点
- Expression of the correct protein at the basement membrane zone
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Expression of the correct protein at the basement membrane zone
时间窗: Week 52
Engraftment and healing of wounds with genetically revertant keratinocytes
时间窗: Week 52
次要结局
- 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)
研究者
Alfred Lane
Professor, Departments of Dermatology and Pediatrics
Stanford University