Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004294
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Brief Title:
Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
Sponsor:
National Center for Research Resources NCRR
Organization:
National Center for Research Resources NCRR
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2003-12
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:
OBJECTIVES I Transfer the human glucocerebrosidase GC gene into peripheral blood stem cells PBSC obtained from patients with type I Gaucher disease using a retroviral vector
II Transplant the autologous transduced PBSC in these patients III Measure the carriage and expression of the transferred gene and its duration in peripheral blood leukocytes
IV Assess the clinical effects of transplanting genetically corrected PBSC
II Transplant the autologous transduced PBSC in these patients III Measure the carriage and expression of the transferred gene and its duration in peripheral blood leukocytes
IV Assess the clinical effects of transplanting genetically corrected PBSC
Detailed Description:
PROTOCOL OUTLINE Patients undergo autologous transplantation using peripheral blood stems cells PBSC stimulated with filgrastim G-CSF and transduced with a retroviral vector containing the human glucocerebrosidase gene R-GC Patients may receive up to 4 transplants if a deficient glucocerebrosidase level is found in peripheral leukocytes 1 month following transplantation
The G-CSF-stimulated PBSC are enriched for CD34-positive stem cells and transduced with the R-GC vector Stem cells with normal gene activity are selected for transplantation
Patients are followed every month for 6 months every 6 months for 18 months and then annually thereafter
The G-CSF-stimulated PBSC are enriched for CD34-positive stem cells and transduced with the R-GC vector Stem cells with normal gene activity are selected for transplantation
Patients are followed every month for 6 months every 6 months for 18 months and then annually thereafter
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UPITTS-GAUCHER | None | None | None |