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To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Miglustat | Experimental | miglustat 200mg tid |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Miglustat | Drug | miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Swallowing | videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue | 12th month |
| Measure | Description | Time Frame |
|---|---|---|
| Mental | IQ test or Mini-Mental Status Examination if a full IQ test can not be performed | 12th month |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Whu-Liang Hwu, MD, PhD | National Taiwan University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Taiwan University Hospital | Taipei | Taiwan |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22476655 | Result | Chien YH, Peng SF, Yang CC, Lee NC, Tsai LK, Huang AC, Su SC, Tseng CC, Hwu WL. Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. J Inherit Metab Dis. 2013 Jan;36(1):129-37. doi: 10.1007/s10545-012-9479-9. Epub 2012 Apr 5. |
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| ID | Term |
|---|---|
| D052556 | Niemann-Pick Disease, Type C |
| ID | Term |
|---|---|
| D009542 | Niemann-Pick Diseases |
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
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| ID | Term |
|---|---|
| C059896 | miglustat |
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|
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D015616 | Histiocytosis, Non-Langerhans-Cell |
| D015614 | Histiocytosis |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic 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 |