Ignite Creation Date:
2024-05-06 @ 8:31 AM
Last Modification Date:
2024-10-26 @ 12:01 PM
Study NCT ID:
NCT02757911
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-04-06
First Post:
2016-04-20
Brief Title:
Gene Therapy for X-linked Chronic Granulomatous Disease
Sponsor:
Genethon
Organization:
Genethon
Study Overview
Official Title:
A Phase III Non Randomized Monocentric 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:
None
Brief Summary:
X-linked chronic granulomatous disease X-CGD is a rare genetic disorder which affects boys It is a primary immunodeficiency disorder which results from an inability of the white blood cells called phagocytic cells or phagocytes to kill invading bacteria and fungi These cells have difficulty forming the free radicals most importantly the superoxide radical due to defective phagocyte NADPH oxidase complex which are important in the killing of ingested pathogens In X-CGD which accounts for two thirds of CGD patients the defect lies in a gene which makes up a critical part of the NADPH-oxidase complex the catalytic subunit gp91-phox protein 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