Ignite Creation Date:
2024-05-05 @ 11:40 PM
Last Modification Date:
2024-10-26 @ 10:37 AM
Study NCT ID:
NCT01380990
Status:
COMPLETED
Last Update Posted:
2021-09-16
First Post:
2011-06-23
Brief Title:
Lentiviral LV Gene Therapy for Adenosine Deaminase ADA Deficiency
Sponsor:
Great Ormond Street Hospital for Children NHS Foundation Trust
Organization:
Great Ormond Street Hospital for Children NHS Foundation Trust
Study Overview
Official Title:
Phase III Historical Controlled Open-label Non-randomised Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34 Cells From ADA-deficient Individuals
Status:
COMPLETED
Status Verified Date:
2021-08
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 historically controlled non-randomized Phase III clinical trial to assess the safety and efficacy of autologous transplantation of CD34 hematopoietic stemprogenitor cells HSPCs obtained from infants affected by ADA-SCID following transduction of the HSPCs with a lentiviral vector LV carrying the human ADA complementary DNA cDNA under the control of the elongation factor 1 alpha shortened EFS promoter Subjects treated in the trial receive the infusion of autologous transduced cells following marrow cytoreduction with busulfan The outcomes are compared to those observed in a historical control group of patients who received an allogeneic hematopoietic stem cell transplant HSCT
This Phase III clinical trial will be performed at Great Ormond Street Hospital GOSH London United Kingdom
This Phase III clinical trial will be performed at Great Ormond Street Hospital GOSH London United Kingdom
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None