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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02520934 |
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Date of registration:
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06/08/2015 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Miglustat on Gaucher Disease Type IIIB
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Scientific title:
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Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB |
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Date of first enrolment:
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July 2015 |
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Target sample size:
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19 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT02520934 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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N/A
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Countries of recruitment
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Taiwan
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Contacts
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Name:
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Yin-Hsiu Chien, M.D., Ph.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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National Taiwan University Hospital |
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Key inclusion & exclusion criteria
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Case_Miglustat
Inclusion Criteria:
1. Confirmed diagnosis of Gaucher Disease: blood test shown lack of beta-
glucocerebrosidase, and found L444P homozygous on GBA gene.
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.
Exclusion Criteria:
1. History of tremor and abnormal extremities perception ( pain, numbness, tingle etc.)
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.
Control_normal Inclusion Criteria
1. Age 6-18 years
2. No significant physical, mental, or psychiatric problems
Exclusion criteria
1. Children with eye disease (not include myopia, hyperopia, Astigmatism)
Age minimum:
6 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Gaucher Disease
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Intervention(s)
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Drug: Miglustat
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Drug: ERT
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Primary Outcome(s)
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Improve in Purdue Pegboard test speed
[Time Frame: 24 months]
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Secondary ID(s)
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201503076MIPD
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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