Ignite Creation Date:
2024-05-06 @ 11:58 AM
Last Modification Date:
2024-10-26 @ 12:52 PM
Study NCT ID:
NCT03645460
Status:
RECRUITING
Last Update Posted:
2024-07-08
First Post:
2018-07-17
Brief Title:
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector Ivlv-ADA
Sponsor:
Shenzhen Geno-Immune Medical Institute
Organization:
Shenzhen Geno-Immune Medical Institute
Study Overview
Official Title:
Gene Therapy Via Intravenous Injection of Lentiviral Vector Ivlv-ADA for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency ADA-SCID
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
Recruiting
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 Phase III trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency ADA-SCID using a self-inactivating lentiviral vector LV ivlv-ADA to functionally correct the genetic defect The primary objectives are to evaluate the safety and efficacy of the direct intravenous iv LV gene therapy protocol
Detailed Description:
This clinical trial will evaluate safety and efficiency of an improved LV system for delivering a therapeutic gene to patients with severe combined immunodeficiency SCID due to a defective adenosine deaminase ADA gene This gene encodes for the adenosine deaminase enzyme which is essential for the proper growth and function of infection-fighting white blood cells called T and B lymphocytes Patients who lack this enzyme are vulnerable to frequent and severe infections
ADA-SCID patients are normally rescued by a bone marrow transplant BMT from a matched healthy donor However matched donors are difficult to find and donor BMT is associated with high risk This trial aims to treat ADA-SCID via direct intravenous iv injection of a safety and efficiency improved self-inactivating LV carrying a functional ADA gene ivlv-ADA to correct the genetic defect By direct iv injection of ivlv-ADA the defective immune cells and blood stem cells in the body can be modified to exhibit ADA activity and correct the immunodeficiency
The primary objectives are to evaluate the safety of the improved ivlv-ADA the iv LV gene transfer clinical protocol and the efficacy of immune recovery in patients to overcome frequent infections present at the time of treatment We will assess the in vivo lentiviral gene transfer efficiency and the long-term effect of this gene transfer procedure
ADA-SCID patients are normally rescued by a bone marrow transplant BMT from a matched healthy donor However matched donors are difficult to find and donor BMT is associated with high risk This trial aims to treat ADA-SCID via direct intravenous iv injection of a safety and efficiency improved self-inactivating LV carrying a functional ADA gene ivlv-ADA to correct the genetic defect By direct iv injection of ivlv-ADA the defective immune cells and blood stem cells in the body can be modified to exhibit ADA activity and correct the immunodeficiency
The primary objectives are to evaluate the safety of the improved ivlv-ADA the iv LV gene transfer clinical protocol and the efficacy of immune recovery in patients to overcome frequent infections present at the time of treatment We will assess the in vivo lentiviral gene transfer efficiency and the long-term effect of this gene transfer procedure
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