Miglustat on Gaucher Disease Type IIIB

March 28, 2019 updated by: National Taiwan University Hospital

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

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

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Interventional

Enrollment (Actual)

19

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Taiwan
      • Taipei, Taiwan, 10041
        • National Taiwan University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Improve in Purdue Pegboard test speed
Time Frame: 24 months
24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Taiwan University Hospital

Collaborators

Actelion

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2015

Primary Completion (Actual)

December 1, 2017

Study Completion (Anticipated)

December 1, 2019

Study Registration Dates

First Submitted

August 6, 2015

First Submitted That Met QC Criteria

August 11, 2015

First Posted (Estimate)

August 13, 2015

Study Record Updates

Last Update Posted (Actual)

March 29, 2019

Last Update Submitted That Met QC Criteria

March 28, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 201503076MIPD

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Gaucher Disease

Clinical Trials on Miglustat

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Papua New Guinea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe