Gaucher DiseaseNCT04094181
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
An observational study of Gaucher Disease, run by Shire at 1 site in Canada.
Trial summary
The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Gaucher Disease
- How many people
- 2 participants
- Where
- 1 siteCanada
- Study dates
- 19 Sep 2019to 17 Nov 2021
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants included in the study will have a diagnosis of Type 1 GD and be in enzyme/substrate replacement therapy other than VPRIV, for at least 6 months prior to enrolment in the study, or have been previously treated with an ERT/SRT other than VPRIV for a minimum of 6 months prior to transitioning to VPRIV.
Who may be able to join
- Participant with GD1 currently being treated with an ERT/SRT other than VPRIV for at least 6 months before baseline enrolment; or participant previously treated with another ERT/ SRT for at least 6 months prior to transitioning to VPRIV..
- Participant or legally authorized representative has provided written informed consent.
Who cannot join
- In the opinion of the investigator, participant is at high risk of non-compliance.
- In the opinion of the investigator, participant is unsuitable in any other way to participate in this study.
- Participant is pregnant.
Trial locations and contacts
1 site in 1 country.
Canada1 site
University of Alberta/Medical Genetics Clinic
Edmonton, Alberta, T6G 2H7
About this study
Official title: VPRIV® Non-Interventional Study in Patients Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)
The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this study started.
In this study, data on VPRIV will be collected from the medical records of participants who already transitioned to VPRIV before this study started. Other participants will join this study when they transition to VPRIV. All participants will be followed to allow for 12 months of observation from time of transition to VPRIV.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.
- 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 |
|---|---|---|
| VPRIV Participants | Participants who has been transitioned from ERTs/SRTs to VPRIV, the data will be collected retrospectively from time of transition until the point at which participant begins in this study and then will be followed up prospectively for 12 months. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Number of participants transitioning from other ERTs/SRTs to VPRIV with AEs will be reported.
Measured: Baseline up to 12 months
Secondary outcomes (2)
Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker
Change in use of Lyso-Gb1 by participants following the transition from other ERTs/SRTs to velaglucerase alfa (VPRIV) will be assessed.
Measured: Baseline, Month 12
Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at Month 12
Gaucher disease questionnaire (PRO) scores will be calculated for each adult participant. The questionnaire asks how much the participant is affected by the Gaucher disease. A total summated score is calculated and ranges from 0 to 100 with higher scores indicating more severe impairment and worse quality of life.
Measured: Baseline, Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2019
- Study start19 Sep 2019
- Primary completion17 Nov 2021
- Study completion17 Nov 2021
Why it stopped: The decision was taken after careful consideration relating to recruitment challenges that could impact the scientific value of the study.
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
- Case only
- Time perspective
- Other
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04094181
- Sponsor's protocol ID
- SHP669-405
- Other IDs
- EUPAS42338
- Status last verified
- Jan 2024
- First submitted
- 17 Sep 2019
- Last updated
- 2 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- VPRIV® Non-Interventional Study in Patients Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)
- ClinicalTrials.gov ID
- NCT04094181
- Acronym / sponsor ID
- SHP669-405
- Conditions
- Gaucher Disease
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The decision was taken after careful consideration relating to recruitment challenges that could impact the scientific value of the study.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 19 Sep 2019 (actual)
- Primary completion
- 17 Nov 2021 (actual)
- Study completion
- 17 Nov 2021 (actual)
- First posted
- 18 Sep 2019 (actual)
- 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 main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this study started.
In this study, data on VPRIV will be collected from the medical records of participants who already transitioned to VPRIV before this study started. Other participants will join this study when they transition to VPRIV. All participants will be followed to allow for 12 months of observation from time of transition to VPRIV.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VPRIV Participants | – | –Participants who has been transitioned from ERTs/SRTs to VPRIV, the data will be collected retrospectively from time of transition until the point at which participant begins in this study and then will be followed up prospectively for 12 months. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants included in the study will have a diagnosis of Type 1 GD and be in enzyme/substrate replacement therapy other than VPRIV, for at least 6 months prior to enrolment in the study, or have been previously treated with an ERT/SRT other than VPRIV for a minimum of 6 months prior to transitioning to VPRIV.
Key inclusion criteria (2 listed)
- Participant with GD1 currently being treated with an ERT/SRT other than VPRIV for at least 6 months before baseline enrolment; or participant previously treated with another ERT/ SRT for at least 6 months prior to transitioning to VPRIV..
- Participant or legally authorized representative has provided written informed consent.
Key exclusion criteria (3 listed)
- In the opinion of the investigator, participant is at high risk of non-compliance.
- In the opinion of the investigator, participant is unsuitable in any other way to participate in this study.
- Participant is pregnant.
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
Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV
Time frame: Baseline up to 12 months
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Number of participants transitioning from other ERTs/SRTs to VPRIV with AEs will be reported.
2 secondary outcomes
Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker
Time frame: Baseline, Month 12
Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at Month 12
Time frame: Baseline, Month 12
Results and safety
The study was stopped early (reason given: The decision was taken after careful consideration relating to recruitment challenges that could impact the scientific value of the study.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2024.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: No. De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
Related trials for Gaucher Disease
RecruitingObservational study
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Neurological Regression, Myoclonus
RecruitingObservational study
Parkinson's Disease in Type 1 Gaucher Disease
Parkinson Disease (PD), Type 1 Gaucher Disease
RecruitingObservational study
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
Gaucher Disease Type I, Oxidative Stress
Not yet recruitingPhase 1/2
4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
Neuroblastoma
Enrolling by invitationObservational study
Genetic Studies of Lysosomal Storage Disorders
Lysosomal Storage Disorders, Gaucher Disease
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
Shire
1 site in 1 country: 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 1 site in Canada.
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 2 inclusion and 3 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: stopped early, as of the sponsor's last update on 2 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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 was stopped early (reason given: The decision was taken after careful consideration relating to recruitment challenges that could impact the scientific value of the study.). Results are not available in the source record.
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?
Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV (time frame: Baseline up to 12 months). 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 2 Feb 2024.