Investigation of cell therapy for recessive dystrophic epidermolysis bullosa (RDEB) in Australia through intradermal injection of allogeneic fibroblasts.

This is a randomized, single-institution double-blind, placebo-controlled clinical trial. Enrolled patients with a diagnosis of RDEB-GS will be randomized to receive either cell therapy with intradermal injection of cultured allogeneic fibroblasts in transport media or placebo (i.e. transport media alone) on two to eight symmetrical wounds on each side of their body (arms, trunk, upper and lower legs). Up to 5 patients will be enrolled. All patients will undergo clinical examination, photography and skin punch biopsies for immunofluorescence and electron microscopy studies (if not previously done). The efficacy endpoint for the primary objective is median healing time of lesions for each treatment group (fibroblast-treated versus placebo). Secondary efficacy end points are percentage change in size, change in appearance score (VAS), pain score (VAS) and pruritus score (VAS) of wounds for each treatment group. The efficacy endpoint for the co-primary objective are quantitative change in expression of collagen VII on immunofluorescence mapping and change in electron microscopy ultrastructure (i.e. numbers and appearance of anchoring fibrils) after cell therapy.