Home Therapy With VPRIV in Gaucher's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01356537
Recruitment Status : Completed
First Posted : May 19, 2011
Last Update Posted : November 29, 2018
Information provided by (Responsible Party):

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.

Condition or disease
Gaucher Disease

Study Type : Observational
Actual Enrollment : 34 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease
Actual Study Start Date : May 20, 2011
Actual Primary Completion Date : September 30, 2017
Actual Study Completion Date : September 30, 2017

Gaucher's Disease under VPRIV

Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients with proven Gaucher's Disease foreseen for home treatment with VPRIV (Velaglucerase alfa) at German Gaucher centers

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01356537

Landeskrankenhaus Bregenz
Bregenz, Austria, A-6900
Paracelsus Medizinische Privatuniversität Salzburg
Salzburg, Austria, A-5020
AKH, Allgemeines Krankenhaus der Stadt Wien
Wien, Austria, A-1090
Medical University of Vienna
Wien, Austria, A-1090
Medizinische Universität Wien
Wien, Austria, A-1090
Gemeinschaftspraxis für Hämatologie und Onkologie
Köln, Germany, 51103
Universitätsklinikum Mainz
Mainz, Germany, 55131
Klinikum rechts der Isar
München, Germany, D-81675
Albrecht-Kossel-Institut für Neuroregeneration (AKos)
Rostock, Germany, D-18147
Helios Klinikum Schwerin
Schwerin, Germany, D-19055
Universitätsklinikum Ulm
Ulm, Germany, D-89075
Sponsors and Collaborators
Study Director: Study Director Shire

Responsible Party: Shire Identifier: NCT01356537     History of Changes
Other Study ID Numbers: Shire/CS03
First Posted: May 19, 2011    Key Record Dates
Last Update Posted: November 29, 2018
Last Verified: November 2018

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders