Ignite Creation Date:
2024-05-06 @ 1:37 AM
Last Modification Date:
2024-10-26 @ 11:06 AM
Study NCT ID:
NCT01852071
Status:
COMPLETED
Last Update Posted:
2022-08-03
First Post:
2013-05-07
Brief Title:
Autologous CD34 Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened EFS Lentiviral Vector Encoding for the Human ADA Gene
Sponsor:
University of California Los Angeles
Organization:
University of California Los Angeles
Study Overview
Official Title:
Autologous Transplantation of Bone Marrow CD34 StemProgenitor Cells After Addition of a Normal Human ADA Complementary DNA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency ADA-SCID
Status:
COMPLETED
Status Verified Date:
2022-08
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:
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells CD34 cells from the bone marrow BM of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector The level of gene transfer in blood cells and immune function will be measured as endpoints
Detailed Description:
The study is open to twenty 20 infants and children diagnosed with ADA-deficient SCID who did not have a medically eligible human leukocyte antigen HLA-identical sibling donor for bone marrow transplantation The EFS-ADA lentiviral vector with the human ADA cDNA will be used to transduce autologous CD34 cells from the bone marrow of these subjects The subjects will receive 4 mgkg busulfan prior to re-infusion of their gene-modified cells Safety is the primary endpoint During the follow-up phase the investigators aim to determine whether the cells could engraft and produce mature cells that contain and express the corrected ADA gene in the absence of pegademase bovine PEG-ADA enzyme replacement therapy ERT which will be withheld at Day 30 following transplant Efficacy studies to evaluate the level of immune reconstitution will be performed in the first and second years of the study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| U01AI100801 | NIH | None | None |
| 2P01HL073104 | NIH | None | None |
| 0910-1006 | OTHER | OBA-RAC | httpsreporternihgovquickSearch2P01HL073104 |