Gaucher DiseaseNCT01356537
Home Therapy With VPRIV in Gaucher's Disease
An observational study of Gaucher Disease, run by Shire at 11 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Gaucher Disease
- How many people
- 34 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 20 May 2011to 30 Sep 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with proven Gaucher's Disease foreseen for home treatment with VPRIV (Velaglucerase alfa) at German Gaucher centers
Who may be able to join
- 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.
Who cannot join
- The patient is participating in a clinical trial with a medicinal product
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
Germany6 sites
Gemeinschaftspraxis für Hämatologie und Onkologie
Cologne, 51103
Universitätsklinikum Mainz
Mainz, 55131
Klinikum rechts der Isar
München, D-81675
Albrecht-Kossel-Institut für Neuroregeneration (AKos)
Rostock, D-18147
Helios Klinikum Schwerin
Schwerin, D-19055
Universitätsklinikum Ulm
Ulm, D-89075
Austria5 sites
Landeskrankenhaus Bregenz
Bregenz, A-6900
Paracelsus Medizinische Privatuniversität Salzburg
Salzburg, A-5020
AKH, Allgemeines Krankenhaus der Stadt Wien
Vienna, A-1090
Medical University of Vienna
Vienna, A-1090
Medizinische Universität Wien
Vienna, A-1090
About this study
Official title: 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.
- Conditions
- Gaucher Disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Gaucher's Disease under VPRIV | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire
Measured: comparison of baseline to 12 months value
Secondary outcomes (1)
Number (per infusion) and severity of infusion-related side effects
Measured: baseline compared to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2011 (estimated)
- Study start20 May 2011
- Primary completion30 Sep 2017
- Study completion30 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01356537
- Sponsor's protocol ID
- Shire/CS03
- Status last verified
- Mar 2021
- First submitted
- 17 May 2011
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease
- ClinicalTrials.gov ID
- NCT01356537
- Acronym / sponsor ID
- Shire/CS03
- Conditions
- Gaucher Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 20 May 2011 (actual)
- Primary completion
- 30 Sep 2017 (actual)
- Study completion
- 30 Sep 2017 (actual)
- First posted
- 19 May 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gaucher's Disease under VPRIV | – | – |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with proven Gaucher's Disease foreseen for home treatment with VPRIV (Velaglucerase alfa) at German Gaucher centers
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (1 listed)
- The patient is participating in a clinical trial with a medicinal product
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
1 secondary outcome
Number (per infusion) and severity of infusion-related side effects
Time frame: baseline compared to 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gaucher Disease, run by Shire at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Germany, Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2021.