Home Therapy With VPRIV in Gaucher's Disease

Gaucher Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01356537
• Other study ID #: Shire/CS03
• Status: Completed
• Start date: May 20, 2011
• Est. completion date: September 30, 2017

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: September 30, 2017
• Est. primary completion date: September 30, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Gaucher Disease

Locations

Country	Name	City	State
Austria	Landeskrankenhaus Bregenz	Bregenz
Austria	Paracelsus Medizinische Privatuniversität Salzburg	Salzburg
Austria	AKH, Allgemeines Krankenhaus der Stadt Wien	Wien
Austria	Medical University of Vienna	Wien
Austria	Medizinische Universität Wien	Wien
Germany	Gemeinschaftspraxis für Hämatologie und Onkologie	Köln
Germany	Universitätsklinikum Mainz	Mainz
Germany	Klinikum rechts der Isar	München
Germany	Albrecht-Kossel-Institut für Neuroregeneration (AKos)	Rostock
Germany	Helios Klinikum Schwerin	Schwerin
Germany	Universitätsklinikum Ulm	Ulm

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

Austria,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Number (per infusion) and severity of infusion-related side effects		baseline compared to 12 months

External Links

•   To obtain more information on the study, click here/on this link.