Ignite Creation Date:
2024-05-05 @ 7:02 PM
Last Modification Date:
2024-10-26 @ 9:42 AM
Study NCT ID:
NCT00599781
Status:
COMPLETED
Last Update Posted:
2008-01-24
First Post:
2008-01-08
Brief Title:
Gene Therapy for ADA-SCID
Sponsor:
IRCCS San Raffaele
Organization:
IRCCS San Raffaele
Study Overview
Official Title:
Treatment of ADA-SCID by Gene Therapy on Somatic Cells
Status:
COMPLETED
Status Verified Date:
2007-12
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 study investigated the safety and efficacy of different gene therapy approaches for Severe Combined Immunodeficiency SCID caused by the deficiency of adenosine deaminase ADA enzyme This is a severe condition that can be cured by HLA-matched sibling donor bone marrow transplantation Patients were enrolled if no HLA-identical sibling donor was available and the patient showed evidence of failure of enzyme replacement therapy or this treatment was not a long-term available option The aim of the study was to evaluate the safety and efficacy of the procedure and to identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after retroviral vector mediated ADA gene transfer
Detailed Description:
This is mono-centric non-randomized non-controlled open label phase I-II trial that evaluated the safety and efficacy of ADA gene transfer into somatic cells for the treatment of ADA-SCID
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