Principal Investigator: Nicola Longo
Keywords: Gaucher , VPRIV , GOS Department: Pediatric Genetics
IRB Number: 00062414 Co Investigator: Nicola Longo
Specialty: Pediatric Genetics
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Carrie Bailey

Brief Summary

The Gaucher Outcome Survey (GOS) is a long-term observational survey being developed in conjunction with leading international experts on Gaucher disease. Patient participation in GOS will be voluntary and GOS will be open to patients with Gaucher disease of any phenotype. Patients who are naïve to treatment, patients who are currently or have been previously treated with VPRIV, as well as patients who have been exposed to or are currently receiving other treatments for Gaucher disease may be included. It is designed to gain a better understanding of the clinical course of the disease and its response to VPRIV therapy, thereby improving the clinical management of patients affected by Gaucher disease. 



The objectives of the Survey are: 

To characterize the clinical course of the patient population with Gaucher disease of any type. 

To characterize the population of patients affected with Gaucher disease who are receiving treatment. 

To monitor the safety and effectiveness of VPRIV® (velaglucerase alfa) enzyme replacement therapy. 

To collect data on the long-term safety and effectiveness of VPRIV treatment in patients with Gaucher disease who are new to therapy or who have been switched from another agent. 

To enhance the understanding of the response of VPRIV in patients with Gaucher disease. 

To collect data on the impact of treatment on the health-related quality of life for patients with Gaucher disease. 

To provide a platform for the collection of data on the clinical experience with VPRIV and to serve as a database for evidence-based management of Gaucher disease. 

To collect ongoing information on safety and effectiveness in support of regulatory and pharmacovigilance obligations 

Inclusion Criteria


1. Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease. 

2. Written informed consent obtained from the patient, and/or patient‟s parent(s), and/or legal representative. Assent, if old enough to grant, will be obtained from all patients under the age of 18 years (For patients in the UK only, informed consent should be obtained for patients age 16 or older, assent for under 16). 

Exclusion Criteria


Patients currently enrolled in ongoing blinded or open-label clinical trials (drugs or devices) (includes all blinded trials).