Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
Status:
Recruiting
Trial end date:
2021-10-01
Target enrollment:
Participant gender:
Summary
This is an open-label, single institution, phase II study in patients with epidermolysis
bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord
blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin
deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary
hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and
efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of
renewable cells for the treatment of focal areas of residual blistering.