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No subjects accrued and no potential subjects were identified..
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| Name | Class |
|---|---|
| Texas Scottish Rite Hospital for Children | OTHER |
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The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Gaucher Type 1 or 3 | Other | Velaglucerase alfa IV 60 units/kg every other week for duration of the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Velaglucerase alfa | Drug | Enzyme replacement therapy |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa. | Baseline pre-intervention and yearly thereafter for 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa. | Baseline pre intervention and yearly thereafter for 3 years | |
| Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Raphael Schiffmann, M.D.,M.H.Sc. | Baylor Research Institute/Institute of Metabolic Disease | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Baylor Research Institute | Dallas | Texas | 75226 | United States |
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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 |
|---|---|
| D005962 | Glucosylceramidase |
| ID | Term |
|---|---|
| D005959 | Glucosidases |
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
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| Baseline pre intervention and yearly thereafter for 3 years |
| Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa. | Baseline pre-intervention and yearly thereafter for 3 years |
| Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa. | Baseline pre-intervention and yearly thereafter for 3 years. |
| 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 |
| D045762 |
| Enzymes and Coenzymes |