Gaucher DiseaseNCT04429984
Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
An observational study of Gaucher Disease, run by Shire at 2 sites in India.
Trial summary
The main aim of this study is to measure the safety and to find out the effects of VPRIV in participants with Gaucher disease using both retrospective and prospective data when used in the post-marketing setting and to collect genetic mutation data from participants with Gaucher disease. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Gaucher Disease
- How many people
- 21 participants
- Where
- 2 sitesIndia
- Study dates
- 28 Jul 2021to 22 Apr 2023
- 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 with confirmed type 1 Gaucher disease irrespective of any age or gender who have been prescribed VPRIV therapy according to the investigator's judgment will be included in this study.
Who may be able to join
- Participants with type 1 Gaucher disease prescribed VPRIV according to the investigator's judgment and current Indian Prescribing information (PI) are eligible for this study.
- Participants or legally authorized representative must provide written informed consent to participate.
Who cannot join
- Participants will be excluded from this study if the participant met any of the contraindications included in the current Indian PI for VPRIV.
Trial locations and contacts
2 sites in 1 country.
India2 sites
Amrita Institute of Medical Sciences & Research Centre (AIMS)
Kochi, Kerala, 682041
All India Institute of Medical Sciences
New Delhi, 110029
About this study
Official title: A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India
The main aim of this study is to measure the safety and to find out the effects of VPRIV in participants with Gaucher disease using both retrospective and prospective data when used in the post-marketing setting and to collect genetic mutation data from participants with Gaucher disease.
This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study.
When the participants start the study, they will visit the study clinic close to approximately 12 months.
- 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: All Participants | Participants with type 1 Gaucher disease who as part of standard or routine clinical practice, have already received VPRIV retrospectively and will further receive VPRIV therapy according to the investigator's judgment for approximately 12 months will be observed prospectively in this PMS study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs)
An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP).
Measured: Baseline up to approximately 12 months
Number of Participants With Adverse Drug Reactions (ADRs)
An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
Measured: Baseline up to approximately 12 months
Secondary outcomes (10)
Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data
Hemoglobin concentration will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
Measured: Baseline up to approximately 12 months
Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data
Platelet count will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
Measured: Baseline up to approximately 12 months
Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data
Spleen size will be assessed using ultrasound or Magnetic Resonance Image (MRI) in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
Measured: Baseline up to approximately 12 months
Change From Baseline in Liver Size Using Both Retrospective and Prospective Data
Liver size will be assessed using ultrasound or MRI in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is first time participants were dosed on CAP.
Measured: Baseline up to approximately 12 months
Treatment History Based on Previous VPRIV Treatment
Presence of treatment history will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
Measured: At Baseline
Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment
Hemoglobin concentration will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
Measured: Baseline up to approximately 12 months
Change From Baseline in Platelet Count Based on Previous VPRIV Treatment
Platelet count data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
Measured: Baseline up to approximately 12 months
Change From Baseline in Spleen Size Based on Previous VPRIV Treatment
Spleen size date will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
Measured: Baseline up to approximately 12 months
Change From Baseline in Liver Size Based on Previous VPRIV Treatment
Liver size data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP.
Measured: Baseline up to approximately 12 months
Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment
An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A SAE is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious AEs will be collected using retrospective data of the previous VPRIV treatment. Baseline is defined as measurement from first time participants was dosed on CAP. Data will be assessed retrospectively based on participant records.
Measured: Baseline up to approximately 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2020
- Study start28 Jul 2021
- Primary completion22 Apr 2023
- Study completion22 Apr 2023
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
- Other
- Enrolment
- 21 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
- NCT04429984
- Sponsor's protocol ID
- SHP669-406
- Status last verified
- Jun 2023
- First submitted
- 9 Jun 2020
- Last updated
- 7 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- 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 7 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India
- ClinicalTrials.gov ID
- NCT04429984
- Acronym / sponsor ID
- SHP669-406
- Conditions
- Gaucher Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 28 Jul 2021 (actual)
- Primary completion
- 22 Apr 2023 (actual)
- Study completion
- 22 Apr 2023 (actual)
- First posted
- 12 Jun 2020 (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 measure the safety and to find out the effects of VPRIV in participants with Gaucher disease using both retrospective and prospective data when used in the post-marketing setting and to collect genetic mutation data from participants with Gaucher disease.
This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study.
When the participants start the study, they will visit the study clinic close to approximately 12 months.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VPRIV: All Participants | – | –Participants with type 1 Gaucher disease who as part of standard or routine clinical practice, have already received VPRIV retrospectively and will further receive VPRIV therapy according to the investigator's judgment for approximately 12 months will be observed prospectively in this PMS study. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with confirmed type 1 Gaucher disease irrespective of any age or gender who have been prescribed VPRIV therapy according to the investigator's judgment will be included in this study.
Key inclusion criteria (2 listed)
- Participants with type 1 Gaucher disease prescribed VPRIV according to the investigator's judgment and current Indian Prescribing information (PI) are eligible for this study.
- Participants or legally authorized representative must provide written informed consent to participate.
Key exclusion criteria (1 listed)
- Participants will be excluded from this study if the participant met any of the contraindications included in the current Indian PI for VPRIV.
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)
Time frame: Baseline up to approximately 12 months
An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP).
Number of Participants With Adverse Drug Reactions (ADRs)
Time frame: Baseline up to approximately 12 months
An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.
10 secondary outcomes
Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data
Time frame: Baseline up to approximately 12 months
Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data
Time frame: Baseline up to approximately 12 months
Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data
Time frame: Baseline up to approximately 12 months
Change From Baseline in Liver Size Using Both Retrospective and Prospective Data
Time frame: Baseline up to approximately 12 months
Treatment History Based on Previous VPRIV Treatment
Time frame: At Baseline
Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment
Time frame: Baseline up to approximately 12 months
Change From Baseline in Platelet Count Based on Previous VPRIV Treatment
Time frame: Baseline up to approximately 12 months
Change From Baseline in Spleen Size Based on Previous VPRIV Treatment
Time frame: Baseline up to approximately 12 months
Change From Baseline in Liver Size Based on Previous VPRIV Treatment
Time frame: Baseline up to approximately 12 months
Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment
Time frame: Baseline up to approximately 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 7 Jun 2023.
- To obtain more information on the study, click here/on this linkLink 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
Shire
2 sites 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 2 sites in India.
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 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 7 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in India. 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?
Number of Participants With Adverse Events (AEs) (time frame: Baseline up to approximately 12 months). The study lists 2 primary outcomes in all. 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 7 Jun 2023.