Table of contents
Trial overview
The main clinical trial in the source data is NCT04186650, a Phase 1/2 interventional study that is authorised and includes 3 participants.[1]
It is studying recessive dystrophic epidermolysis bullosa (RDEB), a rare skin disease, in adults.[1]
The intervention is a skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vector.[1]
Who is being studied
This trial is designed for adults with RDEB.[1]
The source data does not give more detailed inclusion or exclusion rules, so only the adult RDEB population can be confirmed from the trial record.[1]
What the trial measures
The main goal is to evaluate safety after grafting the genetically corrected autologous skin equivalent.[1]
The primary outcome includes adverse events, serious adverse events, adverse reactions, and serious adverse reactions during the first 12 months after grafting.[1]
These safety outcomes are also checked during a planned long-term follow-up period lasting 5 years in total.[1]
Trial phase and design
This is a Phase 1/2 study, which means it is in an early stage of clinical research.[1]
Early-phase studies usually focus first on safety, and this trial follows that pattern by measuring treatment-related harms over time.[1]
The study is interventional, meaning the research team gives a treatment as part of the trial rather than only observing what happens naturally.[1]
Follow-up plan
The brief summary says safety is checked at M1, M2, M3, M6, and M12 after grafting.[1]
After that, participants continue in a regular 5-year follow-up with visits at M18, M24, M30, M36, M42, M48, M54, and M60.[1]
This long follow-up helps researchers watch for later safety problems that may not appear soon after treatment.[1]
