Survey of the Gaucher Disease Outcome to use the data collected to better understand the course of the disease, how it is treated and possibly improve how patients are cared for.
Not Applicable
- Conditions
- Health Condition 1: null- Gaucher disease
- Registration Number
- CTRI/2013/12/004218
- Lead Sponsor
- Shire Human Genetic Therapies Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Other (Terminated)
- Sex
- Not specified
- Target Recruitment
- 500
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 patientâ??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
Exclusion Criteria
1. Patients currently enrolled in ongoing blinded or open-label clinical trials (drugs or devices) (includes all blinded trials).
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method � To characterize the clinical course of the patient population with Gaucher disease of any type. <br/ ><br>� To characterize the population of patients affected with Gaucher disease who are receiving treatment. <br/ ><br>� To monitor the safety and effectiveness of VPRIV® (velaglucerase alfa) enzyme replacement therapy. <br/ ><br>� 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.Timepoint: None
- Secondary Outcome Measures
Name Time Method - To enhance the understanding of the response of VPRIV in patients with Gaucher disease. <br/ ><br>- To collect data on the impact of treatment on the health-related quality of life for patients with Gaucher disease. <br/ ><br>- 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. <br/ ><br>-To collect ongoing information on safety and effectiveness in support of regulatory and pharmacovigilance obligations <br/ ><br>Timepoint: None