In short
Clinical trials are studying AUTOLOGOUS KERATINOCYTES AND FIBROBLASTS TRANSDUCED WITH A RETROVIRAL VECTOR ENCODING COL7A1 CDNA in people with recessive dystrophic epidermolysis bullosa (RDEB). These trials mainly look at safety after grafting genetically corrected skin equivalents and follow patients over time to watch for side effects and longer-term outcomes.
Key points
- Clinical trials of AUTOLOGOUS KERATINOCYTES AND FIBROBLASTS TRANSDUCED WITH A RETROVIRAL VECTOR ENCODING COL7A1 CDNA are studying treatment for recessive dystrophic epidermolysis bullosa (RDEB). The available trial is a Phase 1/2 interventional study in adults with RDEB. It is mainly designed to evaluate safety after grafting genetically corrected autologous skin equivalents. The main outcomes are adverse events, serious adverse events, adverse reactions, and serious adverse reactions. Participants are followed for 12 months after grafting and then for a total follow-up period of 5 years.
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.
It is studying recessive dystrophic epidermolysis bullosa (RDEB), a rare skin disease, in adults.
The intervention is a skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vector.
Who is being studied
This trial is designed for adults with RDEB.
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.
What the trial measures
The main goal is to evaluate safety after grafting the genetically corrected autologous skin equivalent.
The primary outcome includes adverse events, serious adverse events, adverse reactions, and serious adverse reactions during the first 12 months after grafting.
These safety outcomes are also checked during a planned long-term follow-up period lasting 5 years in total.
Trial phase and design
This is a Phase 1/2 study, which means it is in an early stage of clinical research.
Early-phase studies usually focus first on safety, and this trial follows that pattern by measuring treatment-related harms over time.
The study is interventional, meaning the research team gives a treatment as part of the trial rather than only observing what happens naturally.
Follow-up plan
The brief summary says safety is checked at M1, M2, M3, M6, and M12 after grafting.
After that, participants continue in a regular 5-year follow-up with visits at M18, M24, M30, M36, M42, M48, M54, and M60.
This long follow-up helps researchers watch for later safety problems that may not appear soon after treatment.