Ignite Creation Date:
2024-05-06 @ 5:48 PM
Last Modification Date:
2024-10-26 @ 2:35 PM
Study NCT ID:
NCT05432310
Status:
RECRUITING
Last Update Posted:
2024-05-16
First Post:
2022-06-04
Brief Title:
Autologous Mobilized Peripheral Blood CD34 Hematopoietic Stem and Progenitor Cells HSPC Transduced With the Elongation Factor Alpha Short Promoter EFS - Adenosine Deaminase ADA Gene EFS-ADA Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency ADA SCID
Sponsor:
University of California Los Angeles
Organization:
University of California Los Angeles
Study Overview
Official Title:
Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34 Hematopoietic Stem and Progenitor Cells Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency
Status:
RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
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 mobilized peripheral blood mPB of ADA-deficient SCID infants and children following human ADA gene 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 complementary DNA cDNA will be used to transduce autologous CD34 cells from Granulocyte Colony Stimulating Factor G-CSFPlerixafor mobilized Peripheral Blood mPB of these subjects The subjects will receive pharmacokinetically-adjusted busulfan reduced intensity conditioning prior to re-infusion of their gene-modified cells Overall survival at two years 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 pegylated adenosine deaminase PEG-ADA enzyme replacement therapy ERT which will be withheld starting on Day 30 following transplant Efficacy studies to evaluate the level of immune reconstitution will be performed in the two years of the study Patients will be asked to enroll into a long-term follow-up study to reach a total of 15 years follow-up after gene therapy
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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