The VIITAL™ Study: A Phase 3 Clinical Trial of EB-101
A multi-center, randomized clinical trial assessing 10-15 patients with RDEB treated with autologous, gene-corrected cell therapy EB-101 compared to intra-patient untreated wounds.
Enrollment expected to begin in mid-2019.
Phase 1/2 Clinical Trial of EB-101
A single-center randomized clinical trial assessing autologous, gene-corrected cell therapy EB-101 for wound healing in patients with RDEB.
Visit Clinical Trials.gov for more information.
Active, Not Recruiting
Who is eligible to participate in the study?
Key criteria for eligible patients include the following. A full list of inclusion and exclusion criteria are available here.
- Diagnosis of RDEB
- Age 13 years or older
- RDEB type VII collagen mutations
- Confirmed carrier parents
What will be required of patients and their families?
- A pre-biopsy visit
- Visits 14 days and 4, 12, 25, and 52 weeks after cells are transplanted back into the patient
- Additional visits are required for eligible patients receiving long-term follow-up care
About the Study
Recessive Dystrophic Epidermolysis Bullosa, or RDEB, is caused by a defect in the COL7A1 gene resulting in the inability to produce Type VII collagen, which plays an important role in anchoring the dermal and epidermal layers of the skin. The study involves gene transfer into a majority of cells in the outer layer of skin, using an ex vivo approach. Investigators will biopsy skin tissue, grow and multiply the cells, and then insert the correct type VII collagen gene. Inserting the gene may provide the cells with the properties to grow and develop normal skin. This process of inserting the correct gene, or "gene transfer," uses a vector virus. The vector is made so that it only delivers the gene and it should not spread to other parts of the body, which investigators monitor during the study. After cells have received gene transfer, they are grown into a skin patches of cells that look like a plastic film, before being transplanted back onto the patient. After treatment, patients are followed for 24 months to assess safety and efficacy.
Stanford University School of Medicine
The safety and efficacy of the investigational use of this product has not been determined. There is no guarantee that the investigational use listed will be filed with and/or approved for marketing by a regulatory agency.