Ignite Creation Date:
2024-05-06 @ 2:20 AM
Last Modification Date:
2024-10-26 @ 11:17 AM
Study NCT ID:
NCT02022696
Status:
COMPLETED
Last Update Posted:
2017-09-27
First Post:
2013-12-20
Brief Title:
Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34 Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector
Sponsor:
National Human Genome Research Institute NHGRI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34 Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector
Status:
COMPLETED
Status Verified Date:
2017-09-21
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 clinical gene transfer study that aims to verify the safety and efficacy of the use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase ADA gene into the hematopoietic progenitors of patients affected with severe combined immunodeficiency due to ADA deficiency The EFS-ADA vector expresses the human ADA cDNA under the control of the elongation factor alpha short promoter EFS In addition this protocol will examine the effects of the ADA gene transfer on the immune system of treated patients Patients with ADA deficiency and ineligible for matched sibling allogeneic bone marrow transplantation are eligible to participate in the study To increase engraftment and selected advantage or gene-corrected cells busulfan will be used as a cytoreductive agent Enzyme replacement PEG-ADA will be discontinued 30 days after infusion of gene-corrected cells CD34 hematopoietic progenitors will be isolated from the patient bone marrow peripheral blood or cord blood exposed to lentiviral vector-mediated gene transfer and re-infused into the patient through a peripheral vein Clinical immunological and molecular follow-up studies will assess safety toxicity and efficacy of the procedure
Detailed Description:
This is a clinical gene transfer study that aims to verify the safety and efficacy of the use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase ADA gene into the hematopoietic progenitors of patients affected with severe combined immunodeficiency due to ADA deficiency The EFS-ADA vector expresses the human ADA cDNA under the control of the elongation factor alpha short promoter EFS In addition this protocol will examine the effects of the ADA gene transfer on the immune system of treated patients Patients with ADA deficiency and ineligible for matched sibling allogeneic bone marrow transplantation are eligible to participate in the study To increase engraftment and selected advantage or gene-corrected cells busulfan will be used as a cytoreductive agent Enzyme replacement PEG-ADA will be discontinued 30 days after infusion of gene-corrected cells CD34 hematopoietic progenitors will be isolated from the patient bone marrow peripheral blood or cord blood exposed to lentiviral vector-mediated gene transfer and re-infused into the patient through a peripheral vein Clinical immunological and molecular follow-up studies will assess safety toxicity and efficacy of the procedure
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 14-HG-0038 | None | None | None |