drugset / Trial / NCT00004294
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
Phase 1
Completed
15 enrolled
National Center for Research Resources (NCRR)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · collabUniversity of Pittsburgh · collab
Treatment
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.
Timeline
- Start
- 1999-11
- Primary completion
- —
- Completion
- —
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | R-GC | Unknown | — | — |