Back to resultsMiglustat on Gaucher Disease Type IIIB
Primary Purpose
Gaucher Disease
Status
Unknown status
Phase
Not Applicable
Locations
Taiwan
Study Type
Interventional
Intervention
Miglustat
ERT
Sponsored by
About this trial
This is an interventional treatment trial for Gaucher Disease focused on measuring Enzyme Replacement Therapy, Miglustat, Gaucher Disease
Eligibility Criteria
Case_Miglustat
Inclusion Criteria:
- Confirmed diagnosis of Gaucher Disease: blood test shown lack of beta- glucocerebrosidase, and found L444P homozygous on GBA gene.
- Aged 6 years old or above.
- 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:
- History of tremor and abnormal extremities perception ( pain, numbness, tingle etc.)
- Abnormal kidney function.
- 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).
- Allergic to Miglustat.
Control_normal Inclusion Criteria
- Age 6-18 years
- No significant physical, mental, or psychiatric problems
Exclusion criteria
1. Children with eye disease (not include myopia, hyperopia, Astigmatism)
Sites / Locations
- National Taiwan University Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
Case_Miglustat
Control
Arm Description
Besides regular ERT, patients in this group also need to take Miglustat for 24 months.
Patients will be tested for their pupil cycle time.
Outcomes
Primary Outcome Measures
Improve in Purdue Pegboard test speed
Secondary Outcome Measures
Full Information
NCT ID
NCT02520934
First Posted
August 6, 2015
Last Updated
March 28, 2019
Sponsor
National Taiwan University Hospital
Collaborators
Actelion
1. Study Identification
Unique Protocol Identification Number
NCT02520934
Brief Title
Miglustat on Gaucher Disease Type IIIB
Official Title
Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB
Study Type
Interventional
2. Study Status
Record Verification Date
March 2019
Overall Recruitment Status
Unknown status
Study Start Date
July 2015 (undefined)
Primary Completion Date
December 2017 (Actual)
Study Completion Date
December 2019 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Taiwan University Hospital
Collaborators
Actelion
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease
Detailed Description
understand if Miglustat (glucosylceramide synthase inhibitor) could improve neuropathy in patients with Gaucher disease
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease
Keywords
Enzyme Replacement Therapy, Miglustat, Gaucher Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Case_Miglustat
Arm Type
Experimental
Arm Description
Besides regular ERT, patients in this group also need to take Miglustat for 24 months.
Arm Title
Control
Arm Type
No Intervention
Arm Description
Patients will be tested for their pupil cycle time.
Intervention Type
Drug
Intervention Name(s)
Miglustat
Other Intervention Name(s)
Zavesca
Intervention Description
combine miglustat and ERT to see if neurologic manifestations can be improved
Intervention Type
Drug
Intervention Name(s)
ERT
Intervention Description
enzyme replacement therapy
Primary Outcome Measure Information:
Title
Improve in Purdue Pegboard test speed
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Case_Miglustat
Inclusion Criteria:
Confirmed diagnosis of Gaucher Disease: blood test shown lack of beta- glucocerebrosidase, and found L444P homozygous on GBA gene.
Aged 6 years old or above.
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:
History of tremor and abnormal extremities perception ( pain, numbness, tingle etc.)
Abnormal kidney function.
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).
Allergic to Miglustat.
Control_normal Inclusion Criteria
Age 6-18 years
No significant physical, mental, or psychiatric problems
Exclusion criteria
1. Children with eye disease (not include myopia, hyperopia, Astigmatism)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien, M.D., Ph.D.
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10041
Country
Taiwan
12. IPD Sharing Statement
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Miglustat on Gaucher Disease Type IIIB
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