Ignite Creation Date:
2024-05-05 @ 11:08 PM
Last Modification Date:
2024-10-26 @ 10:29 AM
Study NCT ID:
NCT01263379
Status:
COMPLETED
Last Update Posted:
2023-08-22
First Post:
2010-12-15
Brief Title:
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Sponsor:
Abeona Therapeutics Inc
Organization:
Abeona Therapeutics Inc
Study Overview
Official Title:
A Phase 12A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa RDEB Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets LEAES
Status:
COMPLETED
Status Verified Date:
2023-08
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:
This trial will create a skin graft which the investigators call LEAES using the patients own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen The corrected cells will be transplanted back to the patient
Detailed Description:
The research project involves gene transfer into keratinocytes which are the majority of the cells in the outer layer of skin In this gene transfer trial we plan to biopsy some skin tissue grow the cells in a skin cell culture sterile dishes with special fluid that allows cells to grow and multiply and then infect the cells with a virus that we have genetically engineered to insert the correct type VII collagen gene The cells should then make type VII collagen
The process of inserting the correct type VII collagen gene into cells is called gene transfer The virus used is called a retrovirus The virus is made so that it only delivers the type VII collagen gene and it should not spread to other parts of the body During the study we will check for growth of the virus
After cells have received gene transfer we will grow the cells in culture into a sheet of cells that look like a plastic film We plan to graft the sheet to wounds Grafting means we will take cells from the culture and stitch them to the patients skin
The process of inserting the correct type VII collagen gene into cells is called gene transfer The virus used is called a retrovirus The virus is made so that it only delivers the type VII collagen gene and it should not spread to other parts of the body During the study we will check for growth of the virus
After cells have received gene transfer we will grow the cells in culture into a sheet of cells that look like a plastic film We plan to graft the sheet to wounds Grafting means we will take cells from the culture and stitch them to the patients skin
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R01AR055914 | NIH | None | None |
| RAC Protocol 0701-827 | OTHER | None | None |
| eProtocol 14563 | OTHER | Stanford Institutional Review Board | httpsreporternihgovquickSearchR01AR055914 |