LCAT-trial-24 weeks: Protocol for a clinical study to evaluate the safety of regenerative medicine and gene therapy by the autologous transplantation of human lecithin:cholesterol acyltransferase gene-transduced human pre-adipocytes

•Familial LCAT deficiency is an orphan disease without any effective treatment.•We have been developing a novel adipocyte-based ex vivo gene therapy that enables life-long enzyme replacement.•This clinical study was designed to assess the dose-response and the safety in a limited number of orphan disease patients.•The protocol includes a preclinical tumorigenicity test in immunodeficient (NSG) mice as one of the primary endpoints.