Status:
COMPLETED
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Gaucher's Disease
Eligibility:
All Genders
Phase:
PHASE1
Brief Summary
Gaucher's disease is a lysosomal storage disease resulting from glycocerebroside GLUCOCEREBROSIDE (1) accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may o...
Detailed Description
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 pa...
Eligibility 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.
Key Trial Info
Start Date :
January 1 1988
Trial Type :
INTERVENTIONAL
End Date :
April 1 2002
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT00001234
Start Date
January 1 1988
End Date
April 1 2002
Last Update
March 4 2008
Active Locations (1)
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1
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States, 20892