Study evaluates long-term effects of FCX-007 in recessive dystrophic epidermolysis bullosa
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Researchers will analyze the safety and efficacy of gene-corrected fibroblasts for the treatment of recessive dystrophic epidermolysis bullosa, according to a presentation at the South Beach Symposium Medical Dermatology Summit.
“RDEB is a debilitating genetic disorder caused by mutations of the COL7A1 gene responsible for synthesis of collagen 7 (COL7). Mutations may result in reduced or absent COL7, a primary component of the anchoring fibrils in the basement membrane and, consequently, lead to separation of the epidermis and dermis,” Maria Yonchek, BSN, RN, of Castle Creek Biosciences, and colleagues wrote. “A previous phase 1/2 study demonstrated that replacing the mutated COL7A1 gene with the wild type gene is effective in healing wounds associated with RDEB at 12 weeks following initial treatment.”
In a phase 3 study, researchers will examine the efficacy, durability of response and long-term safety of FCX-007 for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Patients will first undergo skin biopsies for fibroblast isolation, followed by a 12-week photographic observational period. Up to three pairs of wounds of will be identified, with one in each pair randomly assigned to receive treatment with FCX-007 intradermally on day 1 and at week 12. The other wound in each pair will serve as a control.
Efficacy endpoints will include wound closure, response durability, change in wound surface area and wound pain; safety assessments will include adverse events, replication-competent lentivirus, COL7 antibody and clinical testing. Patients who receive at least one treatment injection will be followed for 15 years.
“RDEB is a serious and debilitating disorder which currently has no FDA-approved treatment,” Yonchek and colleagues wrote. “The safety and efficacy of local injection of autologous gene-corrected COL7-expressing fibroblasts for the treatment of wounds associated with RDEB will be evaluated in this late-phase clinical study.”
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Yonchek M, et al. A phase 3 randomized trial to determine the safety and efficacy of FCX-007 for the treatment of wounds associated with recessive dystrophic epidermolysis bullosa (RDEB). Presented at: South Beach Symposium Medical Dermatology Summit; Feb. 5-7, 2021 (virtual meeting).
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