Phase 3 Study of EB 101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa
- Clinical diagnosis of RDEB AND confirmation of RDEB diagnosis by immunofluorescence and electron microscopy AND confirmation of RDEB by genetic testing (determined in the "Characteristics" study)
- Age 6 or older
- Presence of some but deficient type VII collagen (determined in the "Characteristics" study)
- At least 40 cm2 (a little smaller than a playing card) areas of chronically wounded areas (present for at least 6 months) on the trunk and/or extremities. Wounds must be open (stage 2).
- Able to undergo anesthesia
- Stable pain regimen for 30 days
- CANNOT have:
- Any additional illnesses that would complicate participation
- Any antibodies to type VII collagen associated antigens (determined in the "Characteristics" study)
- Systemic infection
- Evidence or history of squamous cell carcinoma in the area to be grafted
- Any chemical or biological study product to treat RDEB in the past 3 months
About the clinical trial:
This clinical trial will analyze how effectively sheets of skin made from the participant's own skin cells promote wound healing in patients with RDEB.
This trial will analyze how effectively sheets of skin made from patients' own skin cells, called keratinoyctes, that have been genetically engineered to correctly produce the protein type VII collagen that is missing or dysfunctional in RDEB patients' skin. After the patients' keratinocytes are corrected so that they can produce type VII collagen, they are grown into sheets of cells that look like a plastic film, about the size of a playing card. Then, up to 6 of these sheets of corrected skin cells will be transplanted back onto the patients' skin where they have wounds.
Participants are monitored for 6 months as part of the study and will be asked to enroll in a long term follow up study to evaluate wound healing.
This study will require several trips to Stanford and will require blood tests and skin biopsies. It will also require general anesthesia while grafts are being placed in the operating room, and a one-week hospital stay to immobilize the grafted areas. We will pay travel and study-related expenses.
Study Coordinator: Irene Bailey (650) 721-7149 firstname.lastname@example.org
Link on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT04227106
Previous Phase 1/2a trial on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT01263379
This study is sponsored by Abeona Therapeutics.
The initial results of our Phase 1 study were published in the Journal of the American Medical Association in November 2016. Here is a link to the article.
Siprashvili Z, Nguyen NT, Gorell ES, Loutit K, Khuu P, Furukawa LK, Lorenz HP, Leung TH, Keene DR, Rieger KE, Khavari P, Lane AT, Tang JY, Marinkovich MP. Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients with Recessive Dystrophic Epidermolysis Bullosa. Journal of the American Medical Association. Nov. 2016. 316(17): 1808-1817. https://www.ncbi.nlm.nih.gov/pubmed/27802546