Recessive Dystrophic Epidermolysis Bullosa Clinical Trial
Official title:
VITAL: A Pivotal Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) (GENE TRANSFER)
VITAL is an open-label, Phase 3 study of one-time surgical application of at least 35 EB-101
grafts for the treatment of eligible, chronic open wound sites in up to 15 RDEB patients.
Patient wounds will be randomized to receive grafting or remain untreated (control). Up to 6
wound sites for EB 101 grafting and at least 1 untreated control wound site will be performed
for each patient.
This research project involves transferring type VII collagen into RDEB patients'
keratinocytes, which are the majority of the cells in the outer layer of skin. We plan to
take biopsies, grow cells in the laboratory (in sterile dishes with special fluid that allows
cells to grow and multiply) and then infect these cells with a virus that we have engineered
to insert the type of collagen VII gene that functions normally. This is known as "gene
transfer".
The virus is made so that it only delivers the collagen VII into skin cells and should not
spread to other parts of the body. Blood and skin tests will verify that the virus is not
spreading. We will then graft these corrected cells (EB-101) over wounds that have not healed
for some time.
The corrected cells are grown into sheets of cells that look like a plastic film. We plan to
graft the sheet of cells to wound sites.
Patients will not be able to choose which wounds will receive grafting. The investigator will
identify wounds that are suitable for grafting. A computer program will determine which
wounds to graft (this is called randomization).
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