Abeona Therapeutics will proceed with enrollment for its pivotal Phase 3 study of EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB), following a meeting with the U.S. Food and Drug Administration (FDA).
“We appreciate the clarity provided by the FDA and we are pleased to be aligned with the Agency on the co-primary endpoints [goals] for the Phase 3 VIITAL study,” Michael Amoroso, Abeona’s principal executive and chief operating officer, said in a press release.
“Following the successful completion of the FDA meeting, we continue with all necessary steps to enroll our next patient in the VIITAL study and aim to complete enrollment in 2021,” he added.
The study (NCT04227106) seeks 10 to 15 patients, 6 years and older, with large chronic wounds. Recruitment takes place at Stanford University Medical Center and had been briefly disrupted because of the COVID-19 pandemic. Each participant must have two matched wounds lasting for at least six months and be at least 20 square centimeters in size.
The trial has two primary goals, both to be measured at week 24 (nearly six months). The first is the proportion of wound sites that heal by at least 50% from the start of the study. This will be determined by direct comparison of treated and untreated wound sites. In turn, the second is a reduction in pain associated with changing wound dressings, measured by the mean differences in Wong-Baker FACES scores between treated and untreated sites.
EB-101 is a gene-corrected cell therapy. It consists of inserting a healthy copy of the gene mutated in RDEB — called COL7A1, which carries instructions for making the protein type VII collagen, key for skin integrity — into a patient’s own skin cells in a laboratory. These cells are fixed to a keratinocyte sheet, which is surgically implanted over the wound site to allow the cells to reintegrate into the patient’s body. Keratinocytes are the main cell type found in the top layer of skin.
Treated wound sites of participants in this trial continued to be pain-free up to five years after treatment.
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