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Home Therapy With VPRIV in Gaucher's Disease

This study is currently recruiting participants.
Verified June 2012 by Shire Human Genetic Therapies, Inc.
Sponsor:
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT01356537
First received: May 17, 2011
Last updated: June 6, 2012
Last verified: June 2012
History of Changes
  Purpose

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
Gaucher's 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:

U.S. FDA Resources

Further study details as provided by Shire Human Genetic Therapies, Inc.:

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)
Groups/Cohorts
Gaucher's Disease under VPRIV

  Eligibility

Ages Eligible for Study:   2 Years and older
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

Criteria

Inclusion Criteria:

  • Male or female patient with proven diagnosis of Gaucher's Disease Type 1
  • Age > 2 years
  • At least 5-year old patients having received at least 3 infusions (6 weeks) OR at least 2- to 4-year old patients having received at least 5 - 6 infusions (10 - 12 weeks) of VPRIV® AND well tolerated the infusions
  • Patient is compliant
  • Patient has been selected for home therapy and has already accepted home therapy with VPRIV before inclusion in this study
  • Patient/legal representative has given written informed consent to participation in this study.

Exclusion Criteria:

• The patient is participating in a clinical trial with a medicinal product.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01356537

Locations
Germany
Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin Recruiting
Mainz, Germany, 55131
Contact: Eugen Mengel, Dr.     +49 6131 172 ext -781     mengel@kinder.klinik.uni-mainz.de    
Principal Investigator: Eugen Mengel, Dr.            
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
Principal Investigator: Eugen Mengel, Dr. Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
  More Information

No publications provided

Responsible Party: Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier: NCT01356537     History of Changes
Other Study ID Numbers: Shire/CS03
Study First Received: May 17, 2011
Last Updated: June 6, 2012
Health Authority: Germany: Local Ethics Committee

Additional relevant MeSH terms:
Gaucher Disease
Sphingolipidoses
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 19, 2013