Home Therapy With VPRIV in Gaucher's Disease

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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Landeskrankenhaus Bregenz, Bregenz, Austria

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire

Time frame: comparison of baseline to 12 months value

Secondary outcomes

Number (per infusion) and severity of infusion-related side effects

Time frame: baseline compared to 12 months

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease

Important dates

Study start: 2011
Primary completion: 2017
Study completion: 2017
First posted: May 19, 2011
Registry last updated: Mar 17, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed