Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Adverse Events and Effects
研究概览
简要总结
The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves wound healing and affects the levels of C7 and HSV-1 antibody levels in serum.
Fewer wounds, more rapidly healing wounds, and decreased C7 and HSV-1 antibodies could improve quality of life.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of generalized Recessive dystrophic epidermolysis bullosa (RDEB) demonstrated by COL7A1 mutations.
- •Diagnosis of EBA demonstrated by the presence of levels of serum C7 antibodies above the normal ELISA range
- •Baseline skin blistering greater than 5% total body surface area
- •1 wound at least 20 cm^2 able to be entirely treated with Vyjuvek weekly
- •1 wound at least 20 cm^2 that has never been treated with Vyjuvek
- •Ongoing VYJUVEK treatment.
排除标准
- •History of thrombotic event(s)
- •History of cardiac failure
- •History of renal failure
- •IgA deficiency
研究组 & 干预措施
Intra-Personal Control
There is one arm of the study. First, each participant undergoes a 3-month observational period, during which they continue application of their prescribed Vyjuvek. After 3 months, they enter the treatment period, during which they continue their Vyjuvek application and, in addition, receive the IV IgG treatment.
干预措施: Immunoglobulin G (Biological)
结局指标
主要结局
Adverse Events and Effects
时间窗: 9 Months
Occurrence of adverse events and effects
Percent Change in Wound Area
时间窗: 9 Months
The wound area at the end of the treatment phase compared to the wound area at the beginning of the treatment phase. Wound healing (% and cm\^2) over the course of the treatment phase compared to the wound healing (% and cm\^2), over the observation phase.
次要结局
未报告次要终点
研究者
M. Peter Marinkovich
Associate Professor of Dermatology
Stanford University