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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UNKNOWN
NA
19 participants
INTERVENTIONAL
2015-07-31
2019-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Case_Miglustat
Besides regular ERT, patients in this group also need to take Miglustat for 24 months.
Miglustat
combine miglustat and ERT to see if neurologic manifestations can be improved
ERT
enzyme replacement therapy
Control
Patients will be tested for their pupil cycle time.
No interventions assigned to this group
Interventions
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Miglustat
combine miglustat and ERT to see if neurologic manifestations can be improved
ERT
enzyme replacement therapy
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Aged 6 years old or above.
3. Already have regular ERT (30-120 IU/kg/ every 2 weeks) at least a year; dosage and frequency of ERT had not been changed in recent 3 months.
1. Age 6-18 years
2. No significant physical, mental, or psychiatric problems
Exclusion Criteria
2. Abnormal kidney function.
3. Pregnant or plan to have a baby ( potentially pregnant patient need to be transferred to gynecologist for the test and promise to have proper contraception measures).
4. Allergic to Miglustat.
1\. Children with eye disease (not include myopia, hyperopia, Astigmatism)
6 Years
ALL
Yes
Sponsors
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Actelion
INDUSTRY
National Taiwan University Hospital
OTHER
Responsible Party
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Principal Investigators
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Yin-Hsiu Chien, M.D., Ph.D.
Role: PRINCIPAL_INVESTIGATOR
National Taiwan University Hospital
Locations
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National Taiwan University Hospital
Taipei, , Taiwan
Countries
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Other Identifiers
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201503076MIPD
Identifier Type: -
Identifier Source: org_study_id
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