Status:
COMPLETED
Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Gaucher Disease
Eligibility:
All Genders
Brief Summary
The purpose of this study is to learn more about Gaucher disease. The information we collect from medical histories and a blood sample from people with Gaucher disease may help us pinpoint certain thi...
Detailed Description
This study is designed to evaluate the ex vivo response to pharmacological chaperone therapy by testing blood samples from previously treated and untreated patients with Gaucher disease. The study wil...
Eligibility Criteria
Inclusion
- Willing and able to provide written informed consent by subject or legal guardian
- Male or female of any age
- Confirmed diagnosis of Gaucher disease with known genotype
- Clinically stable and either treatment naïve or on a stable dose of enzyme replacement therapy and/or substrate reduction therapy for at least 6 months prior to study entry
- Available medical records for collection of retrospective clinical information
Exclusion
- Received any investigational product within 30 days prior to study entry
- Other significant disease or be otherwise unsuitable for the study, as determined by the investigator
Key Trial Info
Start Date :
July 1 2006
Trial Type :
OBSERVATIONAL
End Date :
March 1 2007
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT00351156
Start Date
July 1 2006
End Date
March 1 2007
Last Update
August 19 2010
Active Locations (7)
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1
University of California - San Francisco
San Francisco, California, United States, 94143
2
University Research Foundation for Lysosomal Storage Diseases, Inc.
Coral Springs, Florida, United States, 33065
3
Emory University Lysosomal Storage Disease Center
Decatur, Georgia, United States, 30033
4
National Institute of Neurological Disorders and Stroke, NIH
Bethesda, Maryland, United States, 20892