The VIITAL study is a pivotal Phase 3 clinical trial evaluating Abeona’s EB-101 gene therapy for RDEB. Approximately 30 large, chronic wound sites will be treated in total throughout the study, with investigators comparing treated with untreated wound sites on the same patient. Up to 15 patients will be enrolled in the study, beginning at Stanford, with additional study sites expected to open enrollment in Spring/Summer of 2020.
The primary endpoint is the proportion of wounds with at least 50% healing at three months. Investigators will measure wound healing at various other times. Patients will report on secondary endpoints, including changes in pain and its impact on daily living, pain during dressing changes, and physical function.
EB-101 treatment involves a biopsy of healthy skin, followed by gene transfer to deliver Type VII collagen genes into a patient’s own skin cells (keratinocytes). Up to six sheets, each approximately the size of a playing card , are transplanted back to the patient to enable normal Type VII collagen expression and facilitate wound healing.
Eligible RDEB patients must be at least 6 years of age; have Type VII collagen protein in their skin and two RDEB genetic mutations; Chronic wounds on the trunk and/or arms and legs that are at least 40 cm2; and at least two Stage 2 wound sites 20 cm2 or more in size that have been present for at least 6 months. A full list of inclusion/exclusion criteria can be found at ClinicalTrials.gov (NCT04227106).
Study participation is free and includes treatment at medical centers recognized for world-leading care of EB. Reasonable costs of travel and meals will also be reimbursed.
- Diagnosis of RDEB
- Age 6 years or older
- Positive expression of type VII collagen protein in the skin
- Two RDEB type VII collagen mutations
- ≥40 cm2 of chronically wounded areas on the trunk and/or extremities deemed suitable by the investigator for EB-101 treatment
- ≥2 matched eligible wound sites ≥20 cm2; present for ≥6 months, and stage 2 wound