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NCT00004294 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study, sponsored by National Center for Research Resources (NCRR).
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
NCT00004294 is a Phase 1 study that has completed, run by National Center for Research Resources (NCRR). The registered enrollment target is 15 participants.
The verdict
NCT00004294, a Phase 1 study, has completed, sponsored by National Center for Research Resources (NCRR).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
Study 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.
Interventions
- GENETIC human glucocerebrosidase gene into autologous peripheral blood stem cells
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 1999-11 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00004294
The ClinicalTrials.gov registry entry for NCT00004294 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Center for Research Resources (NCRR), which has 94 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which human glucocerebrosidase gene into autologous peripheral blood stem cells is the first listed.
NCT00004294 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00004294 about?
NCT00004294 is a clinical study titled "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". 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 trans...
What is the current status of trial NCT00004294?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 1999-11.
What interventions are being tested in trial NCT00004294?
The interventions under investigation include: human glucocerebrosidase gene into autologous peripheral blood stem cells (GENETIC).
Who is sponsoring clinical trial NCT00004294?
This trial is sponsored by National Center for Research Resources (NCRR), which has 94 total clinical trials registered on ClinicalTrials.gov.
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