Home Therapy With VPRIV in Gaucher's Disease
Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease
1 other identifier
observational
34
2 countries
11
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started May 2011
Longer than P75 for all trials
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 17, 2011
CompletedFirst Posted
Study publicly available on registry
May 19, 2011
CompletedStudy Start
First participant enrolled
May 20, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
September 30, 2017
CompletedMarch 17, 2021
March 1, 2021
6.4 years
May 17, 2011
March 15, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire
comparison of baseline to 12 months value
Secondary Outcomes (1)
Number (per infusion) and severity of infusion-related side effects
baseline compared to 12 months
Study Arms (1)
Gaucher's Disease under VPRIV
Eligibility Criteria
Patients with proven Gaucher's Disease foreseen for home treatment with VPRIV (Velaglucerase alfa) at German Gaucher centers
You may qualify if:
- 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 / be lawful. Representative has consented in writing to participate in this study.
You may not qualify if:
- The patient is participating in a clinical trial with a medicinal product
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (11)
Landeskrankenhaus Bregenz
Bregenz, A-6900, Austria
Paracelsus Medizinische Privatuniversität Salzburg
Salzburg, A-5020, Austria
AKH, Allgemeines Krankenhaus der Stadt Wien
Vienna, A-1090, Austria
Medical University of Vienna
Vienna, A-1090, Austria
Medizinische Universität Wien
Vienna, A-1090, Austria
Gemeinschaftspraxis für Hämatologie und Onkologie
Cologne, 51103, Germany
Universitätsklinikum Mainz
Mainz, 55131, Germany
Klinikum rechts der Isar
München, D-81675, Germany
Albrecht-Kossel-Institut für Neuroregeneration (AKos)
Rostock, D-18147, Germany
Helios Klinikum Schwerin
Schwerin, D-19055, Germany
Universitätsklinikum Ulm
Ulm, D-89075, Germany
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 17, 2011
First Posted
May 19, 2011
Study Start
May 20, 2011
Primary Completion
September 30, 2017
Study Completion
September 30, 2017
Last Updated
March 17, 2021
Record last verified: 2021-03
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.