Clincosm LogoSign in Get a demo

Miglustat on Gaucher Disease Type IIIB

Sponsor
National Taiwan University Hospital (Other)
Overall Status
Unknown status
CT.gov ID
NCT02520934
Collaborator
Actelion (Industry)
19
Enrollment
1
Location
2
Arms
53
Duration (Months)
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease

Condition or Disease Intervention/Treatment Phase
N/A

Detailed Description

understand if Miglustat (glucosylceramide synthase inhibitor) could improve neuropathy in patients with Gaucher disease

Study Design

Study Type:
Interventional
Actual Enrollment :
19 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB
Study Start Date :
Jul 1, 2015
Actual Primary Completion Date :
Dec 1, 2017
Anticipated Study Completion Date :
Dec 1, 2019

Arms and Interventions

Arm Intervention/Treatment
Experimental: Case_Miglustat

Besides regular ERT, patients in this group also need to take Miglustat for 24 months.

Drug: Miglustat
combine miglustat and ERT to see if neurologic manifestations can be improved
Other Names:
  • Zavesca

    • Drug: ERT
    enzyme replacement therapy
    No Intervention: Control

    Patients will be tested for their pupil cycle time.

    Outcome Measures

    Primary Outcome Measures

    1. Improve in Purdue Pegboard test speed [24 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    Yes

    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)

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 National Taiwan University Hospital Taipei Taiwan 10041

    Sponsors and Collaborators

    • National Taiwan University Hospital
    • Actelion

    Investigators

    • Principal Investigator: Yin-Hsiu Chien, M.D., Ph.D., National Taiwan University Hospital

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    National Taiwan University Hospital
    ClinicalTrials.gov Identifier:
    NCT02520934
    Other Study ID Numbers:
    • 201503076MIPD
    First Posted:
    Aug 13, 2015
    Last Update Posted:
    Mar 29, 2019
    Last Verified:
    Mar 1, 2019
    Keywords provided by National Taiwan University Hospital
    Enzyme Replacement Therapy
    Miglustat
    Gaucher Disease
    Additional relevant MeSH terms:
    Gaucher Disease
    Miglustat

    Study Results

    No Results Posted as of Mar 29, 2019