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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

NCT00004294 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2005-06-24

No results posted yet for this 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.

Conditions

  • Gaucher's Disease

Interventions

GENETIC

human glucocerebrosidase gene into autologous peripheral blood stem cells

Sponsors & Collaborators

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    collaborator NIH

  • University of Pittsburgh

    collaborator OTHER

  • National Center for Research Resources (NCRR)

    lead NIH

Principal Investigators

  • John Barranger · University of Pittsburgh

Study Design

Purpose
TREATMENT

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1999-11-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00004294 on ClinicalTrials.gov