Home Therapy With VPRIV in Gaucher's Disease

Completed

• Conditions: Gaucher Disease

• Registration Number: NCT01356537
• Lead Sponsor: Shire

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-05-17
• Study First Submitted QC: 2011-05-17
• Study First Posted: 2011-05-19
• Study Start: 2011-05-20
• Primary Completion: 2017-09-30
• Study Completion: 2017-09-30
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-15
• Last Update Posted: 2021-03-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

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

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire	comparison of baseline to 12 months value

Secondary Outcome Measures

Name	Time	Method
Number (per infusion) and severity of infusion-related side effects	baseline compared to 12 months

Trial Locations

Locations (11)

Gemeinschaftspraxis für Hämatologie und Onkologie; 🇩🇪 Köln, Germany

Universitätsklinikum Mainz; 🇩🇪 Mainz, Germany

Albrecht-Kossel-Institut für Neuroregeneration (AKos); 🇩🇪 Rostock, Germany

Helios Klinikum Schwerin; 🇩🇪 Schwerin, Germany

Klinikum rechts der Isar; 🇩🇪 München, Germany

Universitätsklinikum Ulm; 🇩🇪 Ulm, Germany

Landeskrankenhaus Bregenz; 🇦🇹 Bregenz, Austria

Medical University of Vienna; 🇦🇹 Wien, Austria

Paracelsus Medizinische Privatuniversität Salzburg; 🇦🇹 Salzburg, Austria

AKH, Allgemeines Krankenhaus der Stadt Wien; 🇦🇹 Wien, Austria

Medizinische Universität Wien; 🇦🇹 Wien, Austria