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| ID | Type | Description | Link |
|---|---|---|---|
| TAK-669-4019 | Other Identifier | Takeda |
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The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
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. 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.
When the participants start the study, they will visit the study clinic every 6 months after their first visit.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Standard of Care (SoC) | Neonatal and pediatric participants who has been on ERT (VPRIV) will be followed up for 36 months from the time of treatment initiation as per SOC. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Standard of Care | Other | Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Hemoglobin (Hb) Level | Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed. | From start of ERT initiation up to 5 years of age |
| Percent Change From Baseline in Platelet Count Increase | Percent change from baseline for platelet count increase will be assessed. | From start of ERT initiation up to 5 years of age |
| Percent Change From Baseline in Liver Volume | Percent change from baseline in liver volume will be assessed. | From start of ERT initiation up to 5 years of age |
| Percent Change From Baseline for Spleen Volume | Percent change from baseline for spleen volume will be assessed. | From start of ERT initiation up to 5 years of age |
| Percentage of Participants With Growth Normalization | Percentage of participants with growth normalization will be assessed. | From start of ERT initiation up to 5 years of age |
| Percentage of Participants With Improvement in Bone Disease | Percentage of participants with improvement in bone disease will be assessed. | From start of ERT initiation up to 5 years of age |
| Percentage of Participants With Improvement in Thrombocytopenia | Percentage of participants with improvement in thrombocytopenia will be assessed. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. AEs include SAEs, non-serious AEs. |
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Inclusion Criteria:
Exclusion Criteria:
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Neonatal and pediatric participants included have a diagnosis of GD type I and III and are currently being treated with total ERT (VPRIV) for less than or equal to (<=) 36 months from the time of treatment initiation.
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lysosomal & Rare Disorders Research & 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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| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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| From start of ERT initiation up to 5 years of age |
| From start of ERT initiation up to 5 years of age |
| 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 |