Ignite Creation Date:
2024-05-06 @ 1:37 AM
Last Modification Date:
2024-10-26 @ 11:07 AM
Study NCT ID:
NCT01855685
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-04-06
First Post:
2013-05-14
Brief Title:
Gene Therapy for X-linked Chronic Granulomatous Disease X-CGD
Sponsor:
Genethon
Organization:
Genethon
Study Overview
Official Title:
A Phase III Non Randomized Multicenter Open-label Study of Autologous CD34 Cells Transduced With the G1XCGD Lentiviral Vector in Patients With X-linked Chronic Granulomatous Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-04
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:
CGD
Brief Summary:
X-linked chronic granulomatous disease X-CGD is a rare genetic disorder which affects boys It is caused by an error in a gene that makes part of the immune system The basic defect lies in specialised white blood cells called phagocytic cells or phagocytes which are responsible for protection against infection by destroying invading bacteria and fungi They do this by pouring large amounts of substances similar to bleach onto these organisms In CGD there is a defect in the system that makes the bleach called the NADPH-oxidase In X-CGD which accounts for two thirds of patients the defect lies in a gene which makes up a critical part of the NADPH-oxidase known as gp91-phox and the cells cannot make bleach-like substances Therefore they kill bacteria and fungi poorly and the patients suffer from severe and recurrent infections This also results in inflammation which can damage parts of the body such as the lung and gut
In many cases patients can be adequately protected from infection by constant intake of antibiotics However in others severe life-threatening infections break through In some cases inflammation in the bowel or urinary systems results in blockages which cannot be treated with antibiotics and which may require the use of other drugs such as steroids Development of curative treatments for CGD is therefore of great importance
In many cases patients can be adequately protected from infection by constant intake of antibiotics However in others severe life-threatening infections break through In some cases inflammation in the bowel or urinary systems results in blockages which cannot be treated with antibiotics and which may require the use of other drugs such as steroids Development of curative treatments for CGD is therefore of great importance
Detailed Description:
None
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