Home Therapy With VPRIV in Gaucher's Disease

Shire

Status: Completed

Conditions: Gaucher Disease

Study type: Observational

Funder types: Industry

Identifiers

NCT01356537

Shire/CS03

Details and patient eligibility

About

The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Gaucher's Disease receiving their enzyme replacement therapy with VPRIV (Velaglucerase alfa)at their home setting compared to receiving the infusions at the clinic or at doctor's practice.

Enrollment: 34 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female patients with a confirmed diagnosis of Gaucher disease type 1
• Age> 2 years
• patients who have at least 3 infusions (6 weeks) at least 5-year or 5-6 infusions (10-12 weeks) at 2 - to 4-year patients have received VPRIV ® and tolerate well
• The patient is compliant, the previous VPRIV ® infusions were / performed approximately every 2 weeks in the center during office visits
• The patient was already before inclusion in this study for a home infusion therapy and has consented to (or their legal representative)
• The patient / be lawful. Representative has consented in writing to participate in this study.

Exclusion criteria

• The patient is participating in a clinical trial with a medicinal product

Trial design

34 participants in 1 patient group

Gaucher's Disease under VPRIV