A Screening Study Evaluating Disease Status of Gaucher Type I Patients
A screening study to identify and characterize the disease status of patients with Gaucher Type 1 disease for potential inclusion in a subsequent phase 3 clinical study.
Gaucher Disease, Type 1
|Official Title:||A Screening Protocol to Characterize the Disease Status of Gaucher Type 1 Patients for Potential Inclusion in a Subsequent Phase 3 Clinical Study|
Blood sample/Urine sample
|Study Start Date:||December 2008|
|Study Completion Date:||December 2009|
|Primary Completion Date:||December 2009 (Final data collection date for primary outcome measure)|
The primary objective of this screening study is to identify and characterize the disease status of patients with Gaucher Type 1 disease for potential inclusion in a subsequent Phase 3 clinical study which will evaluate the efficacy and safety of Genz-112638.
This will be a multi-center, multi-national screening study to assess the disease status of patients with Gaucher Type 1 disease utilizing criteria to be eligible for enrollment in a subsequent phase 3 clinical study. The assessment performed in this study are standard of care for the evaluation of symptoms of Gaucher Type 1 disease. No study drug will be administered in this screening study.
|United States, New York|
|The Mount Sinai School of Medicine, Department of Genetics and Genomic Sciences|
|New York, New York, United States, 10029|
|Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGI)|
|Lucknow, India, 226014|
|Jaslok Hospital and Research Center|
|Mumbai, India, 400026|
|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|