Ignite Creation Date:
2024-05-05 @ 7:03 PM
Last Modification Date:
2024-10-26 @ 9:42 AM
Study NCT ID:
NCT00598481
Status:
COMPLETED
Last Update Posted:
2024-01-29
First Post:
2008-01-10
Brief Title:
ADA Gene Transfer Into Hematopoietic StemProgenitor Cells for the Treatment of ADA-SCID
Sponsor:
Fondazione Telethon
Organization:
Fondazione Telethon
Study Overview
Official Title:
ADA Gene Transfer Into Hematopoietic StemProgenitor Cells for the Treatment of ADA-SCID
Status:
COMPLETED
Status Verified Date:
2023-11
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:
Gene-ADA
Brief Summary:
This is a phase III protocol to evaluate the safety and efficacy of ADA gene transfer into hematopoietic stemprogenitor cells for the treatment of adenosine deaminase ADA-deficiency This condition is an autosomal recessive form of Severe Combined Immunodeficiency SCID characterized by impaired immune responses recurrent infections failure to thrive and systemic toxicity due to accumulation of purine metabolites Transplants from an human leukocyte-antigen HLA-identical sibling donor is the treatment of choice but available for a minority of patients The use of alternative bone marrow donors or enzyme replacement therapy is associated with important drawbacks The drug product studied in this protocol consists of autologous cluster of differentiation CD34 hematopoietic stemprogenitor cells engineered ex vivo with a retroviral vector encoding the therapeutic gene ADA The engineered CD34 cells are infused following a nonmyeloablative conditioning with busulfan to make space in the bone marrow The study objectives are a to evaluate the safety and the clinical efficacy of gene therapy in the absence of enzyme replacement therapy b to evaluate the biological activity engraftment ADA expression of ADA transduced CD34 cells and their hematopoietic progeny c to evaluate the immunological reconstitution and purine metabolism after gene therapy
Detailed Description:
The safety of the study will be evaluated by description of all adverse events and adverse drug reactions
The study is aimed at reaching the minimum sample size of ten patients
The study is aimed at reaching the minimum sample size of ten patients
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 |
|---|---|---|---|
| 15386-PRE21 | OTHER | IRCCS San Raffaele | None |