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

Completed · Phase 1

Conditions studied: Gaucher's Disease

In brief

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.

Key facts

Study ID

NCT00004294

Run by

National Center for Research Resources (NCRR)

People needed

15

Starts

1999-11-01

Last updated by the study team

2005-06-24

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of Pittsburgh — Pittsburgh, Pennsylvania, United States

Full record on ClinicalTrials.gov

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