Ignite Creation Date:
2024-05-06 @ 1:48 AM
Last Modification Date:
2024-10-26 @ 11:10 AM
Study NCT ID:
NCT01906541
Status:
UNKNOWN
Last Update Posted:
2013-08-02
First Post:
2013-07-15
Brief Title:
Gene Therapy for X-CGD
Sponsor:
Hubert Serve Prof MD
Organization:
Johann Wolfgang Goethe University Hospital
Study Overview
Official Title:
A Phase III Gene Therapy Trial for X-CGD With a SIN Gammaretroviral Vector
Status:
UNKNOWN
Status Verified Date:
2013-08
Last Known Status:
RECRUITING
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 inherited immune defect which is caused by the inability of phagocytic cells to produce reactive oxygen species due to a defect in the gp91phox subunit of the NADPH oxidase complex X-CGD patients suffer from recurrent and life-threatening infections and severe hyperinflammatory complications
The only curative treatment for X-CGD is allogenic hematopoietic stem cell transplantation but this procedure implies severe risks and many patients lack an appropriate donor Therefore alternative curative approaches are urgently needed In this study patients will be treated with gene-corrected autologous CD34 cells using a SIN gammaretroviral vector for ex-vivo gene-therapy
The only curative treatment for X-CGD is allogenic hematopoietic stem cell transplantation but this procedure implies severe risks and many patients lack an appropriate donor Therefore alternative curative approaches are urgently needed In this study patients will be treated with gene-corrected autologous CD34 cells using a SIN gammaretroviral vector for ex-vivo gene-therapy
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