Ignite Creation Date:
2024-05-06 @ 7:12 AM
Last Modification Date:
2024-10-26 @ 11:46 AM
Study NCT ID:
NCT02493816
Status:
COMPLETED
Last Update Posted:
2019-09-24
First Post:
2015-05-21
Brief Title:
Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa
Sponsor:
Kings College London
Organization:
Kings College London
Study Overview
Official Title:
Phase I Study of Lentiviral-mediated COL7A1 Gene-modified Autologous Fibroblasts in Adults With Recessive Dystrophic Epidermolysis Bullosa
Status:
COMPLETED
Status Verified Date:
2018-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
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
This is an open-label single-centre phase I study with primary objective to evaluate the adverse and serious adverse events over 12 months follow-up period Secondary objectives include 1 analysis of type VII collagen C7 expression and morphology of anchoring fibrils in the injected areas of the skin 2 analysis of immune response to newly expressed C7
Each study participant will receive three intradermal injections of COL7A1 gene-modified autologous fibroblasts on Day 0 only Each subject will undergo an initial screening including a physical examination and assessment of disease severity Blood analyses and skin biopsies will be performed at various time points as per the monitoring schedule over 12 months
This is an open-label single-centre phase I study with primary objective to evaluate the adverse and serious adverse events over 12 months follow-up period Secondary objectives include 1 analysis of type VII collagen C7 expression and morphology of anchoring fibrils in the injected areas of the skin 2 analysis of immune response to newly expressed C7
Each study participant will receive three intradermal injections of COL7A1 gene-modified autologous fibroblasts on Day 0 only Each subject will undergo an initial screening including a physical examination and assessment of disease severity Blood analyses and skin biopsies will be performed at various time points as per the monitoring schedule over 12 months
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None