Ignite Creation Date:
2024-05-06 @ 8:44 AM
Last Modification Date:
2024-10-26 @ 12:03 PM
Study NCT ID:
NCT02800070
Status:
COMPLETED
Last Update Posted:
2024-04-17
First Post:
2016-04-15
Brief Title:
Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease
Sponsor:
University Health Network Toronto
Organization:
University Health Network Toronto
Study Overview
Official Title:
Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive CD34 Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease
Status:
COMPLETED
Status Verified Date:
2024-04
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:
This is a first-in-human study for the treatment of Fabry disease Eligible patients will have an autologous stem cell transplantation using CD34 cells that are transduced with the lentivirus vector containing the human alpha-gal A gene The researchers of this study would like to see if the re-introduction of transduced cells will help increase the levels of alpha-gal A enzyme levels and to determine the safety and toxicity of autologous stem cell transplantation using CD34 cells transduced with lentivirus vector containing the alpha-gal A gene This studys objective is to determine the safety and toxicity of lentivirus alpha-gal A transduced CD34 cells in adult males with Fabry disease
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