A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Adverse Events
研究概览
简要总结
The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects with recessive dystrophic epidermolysis bullosa (RDEB). Funding Source- FDA OOPD
详细描述
RDEB is a rare skin and connective tissue disease characterized clinically by skin fragility with easy blistering, erosion and scarring of skin and mucous membranes, and caused by the deficiency of the protein type VII collagen (COL7). The objective of this study is evaluate the safety of FCX-007 intradermal injections in RDEB subjects. Additionally, the trial will evaluate COL7 expression, the presence of anchoring fibrils, as well evidence of wound healing.
Approximately twelve subjects are expected to enroll in the Phase I/II trial. Phase I will enroll approximately six adult subjects. Phase II will enroll approximately six subjects both adults and pediatric (aged seven (7) years or older). All subjects will receive FCX-007 to one or more paired target RDEB wounds. Proof of mechanism will be monitored through digital photography of target wounds and assays conducted on biopsies taken from intact skin sites where FCX-007 is administered.
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
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Phase I: Eighteen (18) years or older.
- •Phase II: Seven (7) years or older.
- •Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
排除标准
- •Medical instability limiting ability to travel to the investigative center.
- •Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection
- •Current evidence of metastatic squamous cell carcinoma at the site to be injected
- •Clinically significant abnormal laboratory result or other significant clinical abnormalities
- •Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months
研究组 & 干预措施
FCX-007
In Phase I, a target of three adult subjects will be enrolled into Group A and a target of three adult subjects will be enrolled into Group B.
In Phase II the study will target enrolling subjects (aged seven (7 years or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal approximately 6 total subjects.
All subjects will receive FCX-007 into one or more paired target wounds as well as to intact skin at least one time during the study with a possible second administration pending laboratory results.
One wound in each target wound pair will be used as control for efficacy and safety evaluations.
干预措施: FCX-007 (Genetic)
结局指标
主要结局
Adverse Events
时间窗: 52 weeks post treatment
Number of subjects with adverse events.
次要结局
- Complete Wound Closure(Through Week 52)