Status:
COMPLETED
Analysis of the Nervous System in Patients With Fabry's Disease
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Fabry's Disease
Eligibility:
All Genders
Brief Summary
Fabry's disease a genetic disorder (X-linked recessive) due to the absence of the enzyme ceramidetrihexosidase. The disease is characterized by abnormal collections of glycolipids in cells (histiocyte...
Detailed Description
OBJECTIVE: The purpose of this protocol is to study the natural history of Fabry disease in the different organ systems, understand its pathogenesis, and develop adequate clinical outcome measures for...
Eligibility Criteria
- INCLUSION CRITERIA:
All patients with Fabry's disease will be considered as potential candidates for this study.
EXCLUSION CRITERIA:
All candidates must be serologically nonreactive for human immunodeficiency (AIDS) virus. HIV positive patients will be excluded because of the effects of the latter illness on the nervous system.
Patients with Fabry's disease will be excluded from participation if they have additional illnesses such as cancer, diabetes or vasculitis that could potentially involve the nervous system.
The general health and well being of each candidate must be sufficient to allow for a modest amount of blood drawing, collection of appropriate laboratory specimens and performance of necessary roentgenograpic and magnetic resonance (MR) imaging studies. In addition, each candidate must be able to return to the National Institutes of Health (NIH) annually for monitoring of clinical and laboratory parameters.
Key Trial Info
Start Date :
May 11 1995
Trial Type :
OBSERVATIONAL
End Date :
March 3 2008
Estimated Enrollment :
325 Patients enrolled
Trial Details
Trial ID
NCT00001491
Start Date
May 11 1995
End Date
March 3 2008
Last Update
July 2 2017
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892