Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 1 study, sponsored by National Center for Research Resources (NCRR).
NCT00004294 is a Phase 1 study that has completed, run by National Center for Research Resources (NCRR). The registered enrollment target is 15 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
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 |
What the finished NCT00004294 record still lists
NCT00004294 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower).
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 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00004294's enrollment target sits among peer trials
15 1798th of 2000 higher than 157 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01889381 · 15 participants · Phase 2
Human Craniomaxillofacial Allotransplantation
- NCT02530073 · 15 participants · NA
Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) for CDH
- NCT02535936 · 15 participants · NA
Cortical Plasticity in Spastic Diplegia After Selective Dorsal Rhizotomy
- NCT02918474 · 15 participants · NA
Decision Making Tool in Supporting Decision Making in Contralateral Prophylactic Mastectomy in Patients With Newly Diagnosed Breast Cancer
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia