Application of Miglustat in Patients With Niemann-Pick Type C
NCT ID: NCT01760564
Last Updated: 2013-01-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
5 participants
INTERVENTIONAL
2008-01-31
2010-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Miglustat
miglustat 200mg tid
Miglustat
miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.
Interventions
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Miglustat
miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Symptomatic including motor or mental symptoms
Exclusion Criteria
* Allergy to miglustat
ALL
No
Sponsors
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National Taiwan University Hospital
OTHER
Responsible Party
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Principal Investigators
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Whu-Liang Hwu, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
National Taiwan University Hospital
Locations
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National Taiwan University Hospital
Taipei, , Taiwan
Countries
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References
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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.
Other Identifiers
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200802043M
Identifier Type: -
Identifier Source: org_study_id
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