Health Condition 1: null- Gaucher disease
Conditions
Sponsors
Shire Human Genetic Therapies Inc
inVentiv International Pharma Services Pvt Ltd
Eligibility
Inclusion criteria
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
Exclusion criteria: 1. Patients currently enrolled in ongoing blinded or open-label clinical trials (drugs or devices) (includes all blinded trials).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| â?¢ To characterize the clinical course of the patient population with Gaucher disease of any type. â?¢ To characterize the population of patients affected with Gaucher disease who are receiving treatment. â?¢ To monitor the safety and effectiveness of VPRIV® (velaglucerase alfa) enzyme replacement therapy. â?¢ 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
| Measure | Time frame |
|---|---|
| - 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 Timepoint: None | — |
Countries
India
Contacts
Public ContactDr Sumit Goyal
inVentiv Health Clinical
Outcome results
None listed