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Home Therapy With VPRIV in Gaucher's Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2016 by Shire
Information provided by (Responsible Party):
Shire Identifier:
First received: May 17, 2011
Last updated: May 9, 2016
Last verified: May 2016
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.

Gaucher Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease

Resource links provided by NLM:

Further study details as provided by Shire:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 50
Study Start Date: April 2011
Estimated Study Completion Date: June 2021
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Gaucher's Disease under VPRIV


Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01356537

Contact: Shire Contact

Landeskrankenhaus Bregenz Akademische Lehrabteilung der medizinischen Universität Innsbruck Not yet recruiting
Bregenz, Austria, A-6900
Contact: Martina Huemer, MD    +43 (0)5574 401-6507   
Contact: Yildiz Yildiz, MD    0043(0)5574-401-6402   
Paracelsus Medical University Salzburg, Institute for congenital metabolic diseases Recruiting
Salzburg, Austria, A-5020
Contact: Florian B. Lagler, MD    43 662 442002 1224   
AKH, Allgemeines Krankenhaus der Stadt Wien Recruiting
Wien, Austria, A-1090
Contact: Dorothea Möslinger, MD    +43-1-40400-3232   
Medical University of Vienna Recruiting
Wien, Austria, A-1090
Contact: Thomas Stulnig, MD    +43 (0)1 40400 4368   
Endocrinology in the center Recruiting
Erlangen, Germany, D-91054
Contact: Beate Schöfl-Siegert, MD    +49 211 38546590      
Surgery and Orthopedics Community Dr. Buchholz & Partners Recruiting
Hamburg, Germany, D-22607
Contact: Martin Buchholz, MD    +49-40 899008-0      
Dr. med. Hans-Wolfgang Scheuer Recruiting
Kirn, Germany, 55606
Contact: Hans-Wolfgang Scheuer, MD    +49 6752 5191      
Gastroenterological FOCUS PRACTICE Dr. med. Hans Werner Karch Terminated
Kirn, Germany, 55606
Facharzt für Allgemeinmedizin Not yet recruiting
Kriftel, Germany, 65830
Contact: Friedlieb Braun, MD    +49 6192-42210      
Gemeinschaftspraxis für Hämatologie und Onkologie / Ambulante Tumortherapie Not yet recruiting
Köln, Germany, 51103
Contact: Achim Rothe, MD    0049 221/872 047      
Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin Recruiting
Mainz, Germany, 55131
Contact: Eugen Mengel, Dr.    +49 6131 172781 or 175754   
Group Practice, Drs Klaus Michels and Michels Alexander Recruiting
Mühlhausen, Germany, D-92360
Contact: Alexander Michels, MD    +49-9185 438      
lysoTUM Munich Department of Nephrology of the Second Department of Internal Medicine Recruiting
München, Germany, D-81675
Contact: Claudia Regenbogen, MD    +49-089-4140-5644   
Universitätsklinikum Münster Klinik f. Allgemeine Pädiatrie Not yet recruiting
Münster, Germany, 48149
Contact: Thorsten Marquardt, MD   
Albrecht-Kossel-Institut für Neuroregeneration (AKos) Recruiting
Rostock, Germany, D-18147
Contact: Arndt Rolfs, MD    +49 381 494 95 40   
Helios Klinikum Schwerin, Department of Child and Adolescent Medicine Recruiting
Schwerin, Germany, D-19055
Contact: CA Prof. Peter Clemens, MD    +49-385 5202660   
University Clinic of Ulm, Clinic for Children - Pediatric and Adolescent Medicine Recruiting
Ulm, Germany, 89075
Contact: Michael Leichsenring, MD    0731 - 500 57143   
Sponsors and Collaborators
Principal Investigator: Shire Study Physician Shire
  More Information

Responsible Party: Shire Identifier: NCT01356537     History of Changes
Other Study ID Numbers: Shire/CS03 
Study First Received: May 17, 2011
Last Updated: May 9, 2016
Health Authority: Germany: Ethics Commission

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 processed this record on December 08, 2016