Phase I Study of Lentiviral-mediated COL7A1 Gene-modified Autologous Fibroblasts in Adults With Recessive Dystrophic Epidermolysis Bullosa.

NCT02493816 Phase 1 COMPLETED

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe form of blistering skin disease caused by mutations in COL7A1 gene. This study aims to assess the safety of intradermal injections of gene-modified autologous fibroblasts in 5-10 adults with RDEB.

Details

Lead sponsor	King's College London
Phase	Phase 1
Status	COMPLETED
Enrolment	5
Start date	2015-09
Completion	2018-03

Conditions

• Recessive Dystrophic Epidermolysis Bullosa

Interventions

• Gene-modified autologous fibroblasts

Primary outcomes

• Adverse events (AEs), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs) at each visit over 12 months' follow up period. — 12 months

Countries

United Kingdom