CClinicalTrials.gg
Sign In
Status unknownNCT02520934Updated Mar 29, 2019

Miglustat on Gaucher Disease Type IIIB

An interventional study of Miglustat and ERT in Gaucher Disease, sponsored by National Taiwan University Hospital. Status unknown at 1 site in Taiwan. Open to participants aged 6 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2019-03-29.

Sponsored by National Taiwan University Hospital · Not applicable, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Mar 2019), so the status shown — last known as Active, not recruiting — may be out of date.
Phase
Not applicable
Study type
Interventional
Enrollment
19
Allocation
Non-randomized
Ages
6 Years and older
Sex
All
01

Study summary

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

Read the detailed description

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

02

Conditions studied

  • Gaucher Disease

Browse trials for

Keywords

  • Enzyme Replacement Therapy
  • Miglustat
  • Gaucher Disease
03

In context

Gaucher Disease

171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.

This study's enrollment of 19 is close to the median of 20 across 98 interventional studies indexed under Gaucher Disease.

Browse Gaucher Disease studies →

Lead sponsor

National Taiwan University Hospital is the lead sponsor of 2,563 studies on the registry; 569 are open to participants now.

Of its 11 completed or terminated interventional studies of FDA-regulated products, 2 (18%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
Yes

Eligibility criteria

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)
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
19 participants (actual)

Study arms

  • Experimental
    Case_Miglustat

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

    Drug: Miglustat · Drug: ERT

  • No intervention
    Control

    Patients will be tested for their pupil cycle time.

Interventions

  • DrugMiglustat

    combine miglustat and ERT to see if neurologic manifestations can be improved

    Also known as: Zavesca

  • DrugERT

    enzyme replacement therapy

06

What researchers measure

Primary outcomes

  1. Improve in Purdue Pegboard test speed

    Time frame: 24 months

07

Study locations

1 site
  • National Taiwan University Hospital
    Taipei, 10041, Taiwan
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 29, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT02520934
Lead sponsor
National Taiwan University Hospital
Collaborators
Actelion
Responsible party
Sponsor
First posted
Aug 13, 2015
Start date
Jul 2015
Primary completion
Dec 2017
Completion
Dec 2019 (estimated)
Last update
Mar 29, 2019

Study contacts

Yin-Hsiu Chien, M.D., Ph.D.
principal investigator · National Taiwan University Hospital

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Mar 2019. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Gaucher Disease→

Other studies from this sponsor

National Taiwan University Hospital

Start the discussion