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The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means the participant will be treated according to the clinic's standard practice. The study sponsor will not be involved in how participants are treated with VPRIV, will provide instructions on how the clinic will record what happens during the study.
VPRIV is a type of enzyme replacement therapy (also known as ERT). Before starting the study, participants must either have switched from substrate reduction therapies (SRT) to VPRIV or switched from other enzyme replacement therapies to SRT then finally to VPRIV. During this time, medical data will be collected from the participants' medical records.
During the study, participants will be treated with VPRIV according to their clinic's standard practice. VPRIV is given by a slow injection into the vein, also known as an infusion. This will happen in the clinic or at home.
The study will record if blood levels of specific substances remain stable or improve during the switch to treatment with VPRIV. Some of these substances will show if organs such as the liver or spleen are working well. Others are blood cells that help blood to clot, known as platelets. Another is a substance in a red blood cell used to carry oxygen around the body, known as hemoglobin.
Participants will use a digital tool so they can be more involved in decision making in their treatment. The digital tool is a mobile phone app, in which each participant can log their daily activities, their general health and wellbeing, and other key information.
Medical data will also be collected from the participants' charts during this time.
Health problems of the participants will be recorded during the study to check if there were any side effects from VPRIV treatment.
Participants will be in this study for up to 12 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A: Prospective | Experimental | Participants with Gaucher's Disease 1 (GD1) transitioning from SRTs to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) in a real-world setting among adults 18 and older will be studied by observing standard patient care and by reviewing the results of tests and assessments that would be performed as part of their routine treatment. |
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| Arm B: Retrospective | Experimental | Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) will be assessed retrospectively after switch to ERT for up to 12 months. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Digital Engagement Application (GD App) | Other | This is a chart review (prospective) data analysis study to describe the effect of the treatment change on the clinical parameters and patient reported outcomes (PROs) with the use of a digital engagement application (GD app) (to evaluate participants clinical engagement, shared decision making, emotional wellbeing, activities of daily living, goal attainment and Gaucher Disease specific measures). |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12 | Hemoglobin concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of hemoglobin concentration level less than (<) 1.5 gram per deciliter (g/dl) decrease level. Number of participants with clinically stable hemoglobin from baseline up to Month 12 were reported. | From Baseline up to Month 12 |
| Number of Participants With Clinically Stable Platelet Count From Baseline in Platelet Count up to Month 12 | Platelet count concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of platelet count < 25 percent (%) decrease levels. Number of participants with clinically stable platelet count from baseline up to Month 12 were reported. | From Baseline up to Month 12 |
| Number of Participants With Clinically Stable Liver Volume From Baseline up to Month 12 | Clinical stability from baseline was calculated using prespecified threshold of liver volume < 20 % increase, was assessed using ultrasound or Magnetic Resonance Image (MRI). Number of participants with clinically stable liver volume from baseline up to Month 12 were reported. | From Baseline up to Month 12 |
| Number of Participants With Clinically Stable Spleen Volume From Baseline up to Month 12 | Clinical stability from baseline was calculated using prespecified threshold of spleen volume < 25 % increase was assessed using ultrasound or MRI. Number of participants with clinically stable spleen volume from baseline up to Month 12 were reported. | From Baseline up to Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was defined as any untoward medical occurrence in a clinical investigation participant administered with a drug; it does not necessarily have to have a causal relationship with the treatment. An SAE was any event that resulted in: death; was life-threatening; required inpatient hospitalization or resulted in prolongation of existing hospitalization; persistent or significant disability/incapacity; was a congenital anomaly/birth defect or a medically important event. AEs included both SAEs, and non-serious AEs. |
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Inclusion Criteria:
Participant eligibility is determined according to the following criteria prior to entry into the study:
Exclusion Criteria:
Any participant who meets any of the following criteria will not qualify for entry into the study:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Lysosomal and Rare Disorders Research and Treatment Center | Fairfax | Virginia | 22030 | United States |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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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).
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A total of 4 participants were enrolled in the study. This is an interventional, retrospective and prospective study to describe the effect of the treatment change on the clinical parameters and patient reported outcomes (PROs) with the use of a digital engagement application.
The study was conducted at 3 sites in United States from 22 April 2021 to 16 February 2023.
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| ID | Title | Description |
|---|---|---|
| FG000 | Arm A: Prospective | Participants with Gaucher's Disease 1 (GD1) who were treated with substrate reduction therapy (SRT) for at least 3 months before being switched to enzyme replacement therapy (ERT) velaglucerase alfa (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline). All participants were treated in accordance with physician treatment plan (standard clinical practice). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 3, 2022 | Nov 15, 2023 |
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| No Intervention | Other | This is a chart review (retrospective) data analysis study to describe the effect of the treatment change on the clinical parameters. |
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| From start of the study up to Month 12 |
| FG001 | Arm B: Retrospective | Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice). |
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| NOT COMPLETED |
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Analysis population included all eligible participants whose medical charts were retrieved and observed in this study. Only 4 participants were enrolled in this study. Based on the low enrolment number, "Overall Arm" data was collected and reported in order to protect and maintain participant privacy/confidentiality.
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| ID | Title | Description |
|---|---|---|
| BG000 | All GD1 Participants | All participants with GD1 who were treated with SRT for at least 3 months before being switched to ERT (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline); and participants transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice). |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12 | Hemoglobin concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of hemoglobin concentration level less than (<) 1.5 gram per deciliter (g/dl) decrease level. Number of participants with clinically stable hemoglobin from baseline up to Month 12 were reported. | Analysis population included all eligible participants whose medical charts were retrieved and observed in this study. | Posted | Count of Participants | Participants | From Baseline up to Month 12 |
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| Primary | Number of Participants With Clinically Stable Platelet Count From Baseline in Platelet Count up to Month 12 | Platelet count concentration level was assessed from the blood sample. Clinical stability from baseline was calculated using prespecified threshold of platelet count < 25 percent (%) decrease levels. Number of participants with clinically stable platelet count from baseline up to Month 12 were reported. | Analysis population included all eligible participants whose medical charts were retrieved and observed in this study. | Posted | Count of Participants | Participants | From Baseline up to Month 12 |
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| Primary | Number of Participants With Clinically Stable Liver Volume From Baseline up to Month 12 | Clinical stability from baseline was calculated using prespecified threshold of liver volume < 20 % increase, was assessed using ultrasound or Magnetic Resonance Image (MRI). Number of participants with clinically stable liver volume from baseline up to Month 12 were reported. | Data for this outcome measure could not be collected and analyzed as no participants were evaluable for the specified parameter. | Posted | From Baseline up to Month 12 |
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| Primary | Number of Participants With Clinically Stable Spleen Volume From Baseline up to Month 12 | Clinical stability from baseline was calculated using prespecified threshold of spleen volume < 25 % increase was assessed using ultrasound or MRI. Number of participants with clinically stable spleen volume from baseline up to Month 12 were reported. | Data for this outcome measure could not be collected and analyzed as no participants were evaluable for the specified parameter. | Posted | From Baseline up to Month 12 |
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| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was defined as any untoward medical occurrence in a clinical investigation participant administered with a drug; it does not necessarily have to have a causal relationship with the treatment. An SAE was any event that resulted in: death; was life-threatening; required inpatient hospitalization or resulted in prolongation of existing hospitalization; persistent or significant disability/incapacity; was a congenital anomaly/birth defect or a medically important event. AEs included both SAEs, and non-serious AEs. | Analysis population included all eligible participants whose medical charts were retrieved and observed in this study. | Posted | Count of Participants | Participants | From start of the study up to Month 12 |
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From start of the study up to Month 12
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm A: Prospective | Participants with GD1 who were treated with SRT for at least 3 months before being switched to ERT (VPRIV) were followed-up prospectively for 12 months from the switch from SRT to ERT (baseline). All participants were treated in accordance with physician treatment plan (standard clinical practice). | 0 | 2 | 0 | 2 | 0 | 2 |
| EG001 | Arm B: Retrospective | Participants with GD1 transitioning from SRT to ERT (VPRIV) or ERT to SRT and then to ERT (VPRIV) previously (within the past 5 years at the time of enrollment) were followed-up retrospectively for up to 12 months before being switched from SRT to ERT (VPRIV). All participants were treated in accordance with physician treatment plan (standard clinical practice). | 0 | 2 | 0 | 2 | 0 | 2 |
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If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 2, 2022 | Dec 20, 2023 | SAP_002.pdf |
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| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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