An observational study in Gaucher Disease, sponsored by Shire. Completed at 2 sites in India. Per ClinicalTrials.gov, last updated 2023-06-07.
Sponsored by Shire · Observational
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.
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's enrollment of 21 is below the median of 60 across 63 observational studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Shire is the lead sponsor of 346 studies on the registry; 2 are open to participants now.
Of its 47 completed or terminated interventional studies of FDA-regulated products, 47 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
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.
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).
Time frame: 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.
Time frame: Baseline up to approximately 12 months
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline up to approximately 12 months
Plan to share: 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.
Supporting information: Study protocol, Sap, Icf, Csr
This study is completed, as verified in Jun 2023. You cannot join it, but the record below documents what was studied.
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