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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

May 17, 2011
June 6, 2012
April 2011
December 2020   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT01356537 on ClinicalTrials.gov Archive Site
Number (per infusion) and severity of infusion-related side effects [ Time Frame: baseline compared to 12 months ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
Home Therapy With VPRIV in Gaucher's Disease
Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease

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.
Not Provided
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Probability Sample

Patients with proven Gaucher's Disease foreseen for home treatment with VPRIV (Velaglucerase alfa) at German Gaucher centers
Gaucher's Disease
Not Provided
Gaucher's Disease under VPRIV
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
50
June 2021
December 2020   (final data collection date for primary outcome measure)

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.
Both
2 Years and older
No
Not Provided
Germany
 
NCT01356537
Shire/CS03
No
Shire Human Genetic Therapies, Inc.
Shire Human Genetic Therapies, Inc.
Not Provided
Principal Investigator: Eugen Mengel, Dr. Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
Shire Human Genetic Therapies, Inc.
June 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP