The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa.
DEFI-RDEB is a multi-center, intra-patient randomized, controlled, open-label, Phase 3 study of FCX-007 for the treatment of persistent non-healing wounds in approximately 20 RDEB subjects. Each subject will serve as his/her own control. Each subject's target wounds will be paired then randomized to receive FCX-007 (treatment wound) or remain untreated (control wound). Up to three target wound pairs will be identified for each subject. FCX-007 will be administered to one, two or three treatment wounds, as applicable; the corresponding paired control wounds will not be treated. Subjects who receive at least one FCX-007 administration will participate in a separate 15-year long-term safety follow-up study.
- Male or female ≥2 years of age at the Screening visit.
- Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
- At least two eligible persistent non-healing wound sites identified.
- Please click the link below for exclusion criteria.