Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-25 @ 1:45 AM
Ignite Modification Date:
2025-12-25 @ 1:45 AM
NCT ID:
NCT00004294
Brief 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.
Detailed Description:
PROTOCOL OUTLINE: Patients undergo autologous transplantation using peripheral blood stems cells (PBSC) stimulated with filgrastim (G-CSF) and transduced with a retroviral vector containing the human glucocerebrosidase gene (R-GC). Patients may receive up to 4 transplants if a deficient glucocerebrosidase level is found in peripheral leukocytes 1 month following transplantation.
The G-CSF-stimulated PBSC are enriched for CD34-positive stem cells and transduced with the R-GC vector. Stem cells with normal gene activity are selected for transplantation.
Patients are followed every month for 6 months, every 6 months for 18 months, and then annually thereafter.
Study:
NCT00004294
Study Brief:
Protocol Section:
NCT00004294