Status:
COMPLETED
Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Type 2 Gaucher Disease
Eligibility:
MALE
Up to 5 years
Brief Summary
This study will use an experimental method of delivering the enzyme glucocerebrosidase directly into the brain of a patient with Gaucher disease to treat disease symptoms. Patients with Gaucher diseas...
Detailed Description
Objective. To overcome the previous delivery limitations and to provide enzyme to the deficient sites in the central nervous system (CNS) of a Type 2 Gaucher disease patient, we will investigate the u...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA
- The patient is eligible because he has Type 2 Gaucher disease.
- EXCLUSION CRITERIA
- The patient may not be eligible to receive CED of glucocerebrosidase if he:
- Is not healthy enough to undergo surgery or general anesthesia.
- Has an uncorrectable bleeding disorder.
- Is not able to undergo magnetic resonance (MR)-imaging.
Exclusion
Key Trial Info
Start Date :
October 22 2005
Trial Type :
OBSERVATIONAL
End Date :
November 3 2006
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT00244582
Start Date
October 22 2005
End Date
November 3 2006
Last Update
July 2 2017
Active Locations (1)
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1
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States, 20892