Trial record 16 of 103 for:    Gaucher Disease

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01356537
Recruitment Status : Recruiting
First Posted : May 19, 2011
Last Update Posted : February 3, 2017
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
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease
Study Start Date : April 2011
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : June 2021

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

Contact: Shire Contact

Landeskrankenhaus Bregenz Recruiting
Bregenz, Austria, A-6900
Contact: Martina Huemer, MD    +43 (0)5574 401-6507   
Principal Investigator: Martina Huemer, MD         
Principal Investigator: Yildiz Yildiz, MD         
Paracelsus Medizinische Privatuniversität Salzburg Recruiting
Salzburg, Austria, A-5020
Contact: Florian B. Lagler, MD    43 662 442002 1224   
Principal Investigator: Florian B. Lagler, MD         
AKH, Allgemeines Krankenhaus der Stadt Wien Recruiting
Wien, Austria, A-1090
Contact: Dorothea Möslinger, MD    +43-1-40400-3232   
Principal Investigator: Vassiliki Konstantopoulou, MD         
Medical University of Vienna Recruiting
Wien, Austria, A-1090
Contact: Thomas Stulnig, MD    +43 (0)1 40400 4368   
Principal Investigator: Thomas Stulnig, MD         
Medizinische Universität Wien Recruiting
Wien, Austria, A-1090
Contact: Prof. Thomas Stulnig, MD    +43 1 40400 4368   
Principal Investigator: Thomas Stulnig, MD         
Endocrinology in the center Withdrawn
Erlangen, Germany, D-91054
Facharzt für Chirurgie und Orthopädie Withdrawn
Hamburg, Germany, D-22607
Gastroenterological FOCUS PRACTICE Dr. med. Hans Werner Karch Withdrawn
Kirn, Germany, 55606
Gemeinschaftspraxis für Hämatologie und Onkologie Recruiting
Köln, Germany, 51103
Contact: Tatjana Gruner-Ewald    +49 221 989 44717   
Principal Investigator: Achim Rothe, MD         
Universitätsklinikum Mainz Recruiting
Mainz, Germany, 55131
Contact: Eugen Mengel, Dr.    +49 6131 172781   
Principal Investigator: Eugen Mengel, MD         
Group Practice, Drs Klaus Michels and Michels Alexander Withdrawn
Mühlhausen, Germany, D-92360
Klinikum rechts der Isar Recruiting
München, Germany, D-81675
Contact: Claudia Regenbogen, MD    +49-089-4140-5644   
Principal Investigator: Claudia Regenbogen, MD         
Albrecht-Kossel-Institut für Neuroregeneration (AKos) Recruiting
Rostock, Germany, D-18147
Contact: Arndt Rolfs, MD    +49 381 494 95 40   
Principal Investigator: Arndt Rolfs, MD         
Helios Klinikum Schwerin Recruiting
Schwerin, Germany, D-19055
Contact: CA Prof. Peter Clemens, MD    +49-385 5202660   
Principal Investigator: Peter Clemens, MD         
Universitätsklinikum Ulm Recruiting
Ulm, Germany, D-89075
Contact: Michael Leichsenring, MD    0731 - 500 57143   
Principal Investigator: Prof. Michael Leichsenring, MD         
Praxis für Innere Medizin Withdrawn
Wallgau, Germany, D-82499
Sponsors and Collaborators
Principal Investigator: Shire Study Physician 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: February 3, 2017
Last Verified: February 2017

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
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors