Epidermolysis Bullosa Dystrophica, Recessive Clinical Trial
Official title:
A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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
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. ;
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