Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells
N/A
N/A
Both
Gaucher's Disease
Trial Information
Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells
This protocol was developed in order to obtain bone marrow stem cells for ex vivo
transduction with retroviruses containing the human glucocerebrosidase gene. We continue to
enter a small number of patients to this protocol each year. Studies with the bone marrow
hematopoietic progenitor cells have enabled us to identify the most effective retroviral
construct currently available in order to carry out gene therapy trials in patients with
Gaucher's disease. The data revealed that a comparatively simple retroviral construct
containing human glucocerebrosidase cDNA driven by the MoLV promoter is highly effective.
We have obtained approval and initiated a Phase I safety and gene marking investigation in
patients with Type I Gaucher's Disease.
Inclusion Criteria
Normal and patient volunteers.
Individuals with platelet counts less than 40,000/ul, PT greater than 15 seconds, or PTT
greater than 40 seconds will not undergo bone marrow aspiration.
Individuals with hematologic disorders other than Gaucher Disease, Fabry Disease, or mild
iron deficiency will not undergo bone marrow aspiration.
HIV positive individuals will be excluded from participating.
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety Study, Primary Purpose: Treatment
Authority:
United States: Federal Government
Study ID:
880019
NCT ID:
NCT00001234
Start Date:
January 1988
Completion Date:
April 2002
Related Keywords:
- Gaucher's Disease
- Bone Marrow
- Retroviral-Mediated Gene Transfer
- Hematopoietic Progenitor Cell
- Gaucher Disease
Name | Location |
|---|---|
| National Institute of Neurological Disorders and Stroke (NINDS) | Bethesda, Maryland 20892 |
