Ignite Creation Date:
2025-12-25 @ 1:45 AM
Ignite Modification Date:
2025-12-31 @ 2:22 PM
Study NCT ID:
NCT00004294
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
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:
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 ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| UPITTS-GAUCHER | None | None | View |