Miglustat on Gaucher Disease Type IIIB | NCT02520934

Studies
Miglustat on Gaucher Disease Type IIIB

NCT02520934

Sponsor

National Taiwan University Hospital

Status

Unknown status

Last Update Posted

Mar 29, 2019Actual

Enrollment

19Actual

Phase

Not Applicable

Conditions

Gaucher Disease

Interventions

Miglustat
ERT

Countries

Taiwan

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2019

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Active, not recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2015

Primary Completion Date

Dec 2017Actual

Completion Date

Dec 2019Estimated

First Submitted Date

Aug 6, 2015

First Submission Date that Met QC Criteria

Aug 11, 2015

First Posted Date

Aug 13, 2015Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 28, 2019

Last Update Posted Date

Mar 29, 2019Actual

Name	Class
Actelion	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

19Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Case_Miglustat	Experimental	Besides regular ERT, patients in this group also need to take Miglustat for 24 months.	Drug: Miglustat Drug: ERT
Control	No Intervention	Patients will be tested for their pupil cycle time.

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Miglustat	Drug	combine miglustat and ERT to see if neurologic manifestations can be improved	Case_Miglustat

Measure	Description	Time Frame
Improve in Purdue Pegboard test speed		24 months

Eligibility Module

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)

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Yin-Hsiu Chien, M.D., Ph.D.	National Taiwan University Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
National Taiwan University Hospital		Taipei		10041	Taiwan

Condition Browse Module

MeSH Terms

ID	Term
D005776	Gaucher Disease

Ancestor Terms

ID	Term
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic

Browse Leaves

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Browse Branches

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