Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Completed
Conditions studied: Gaucher Disease
In brief
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 things that are different between people who have Gaucher disease and people who do not have Gaucher disease. This information may be useful in the future to help find new treatments for Gaucher disease.
Key facts
- Study ID
- NCT00351156
- Run by
- Amicus Therapeutics
- People needed
- 50
- Starts
- 2006-07-01
- Expected to finish
- 2007-03-01
- Last updated by the study team
- 2010-08-19
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- University of California - San Francisco — San Francisco, California, United States
- University Research Foundation for Lysosomal Storage Diseases, Inc. — Coral Springs, Florida, United States
- Emory University Lysosomal Storage Disease Center — Decatur, Georgia, United States
- National Institute of Neurological Disorders and Stroke, NIH — Bethesda, Maryland, United States
- New York University School of Medicine, Neurogenetics Department — New York, New York, United States
- Lysosomal Disease Center, Cincinnati Children's Hospital — Cincinnati, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Full record on ClinicalTrials.gov
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