Miglustat on Gaucher Disease Type IIIB

Gaucher Disease Clinical Trial

Official title:

Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02520934
• Other study ID #: 201503076MIPD
• Status: Active, not recruiting
• Phase: N/A
• Start date: July 2015
• Est. completion date: December 2019

Study information

• Verified date: March 2019
• Source: National Taiwan University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 19
• Est. completion date: December 2019
• Est. primary completion date: December 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 6 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

Drug:
• Miglustat
combine miglustat and ERT to see if neurologic manifestations can be improved
• ERT
enzyme replacement therapy

Locations

Country	Name	City	State
Taiwan	National Taiwan University Hospital	Taipei

Sponsors (2)

Lead Sponsor	Collaborator
National Taiwan University Hospital	Actelion

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improve in Purdue Pegboard test speed		24 months