Gaucher DiseaseNCT02520934
Miglustat on Gaucher Disease Type IIIB
A study testing Miglustat and ERT in people with Gaucher Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease Read more about this study
- Who is it for?
- Ages 6 years and olderHealthy volunteers can take part
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- Miglustat and ERT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteTaiwan
- Study dates
- Jul 2015to Dec 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
- Controlnormal Inclusion Criteria
- Age 6-18 years
- No significant physical, mental, or psychiatric problems
- 1. Children with eye disease (not include myopia, hyperopia, Astigmatism)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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
Read the detailed description
understand if Miglustat (glucosylceramide synthase inhibitor) could improve neuropathy in patients with Gaucher disease
- Conditions
- Gaucher Disease
- Being tested
- Miglustat, ERT
- Keywords
- Enzyme Replacement Therapy, Miglustat, Gaucher Disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MiglustatDrug
Also called Zavesca
combine miglustat and ERT to see if neurologic manifestations can be improved
ERTDrug
enzyme replacement therapy
| Study group | Role | What they receive |
|---|---|---|
| Case_Miglustat | Experimental | Besides regular ERT, patients in this group also need to take Miglustat for 24 months. Receives: Drug: Miglustat; Drug: ERT |
| Control | No intervention | Patients will be tested for their pupil cycle time. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improve in Purdue Pegboard test speed
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2015 (estimated)
- Study startJul 2015
- Primary completionDec 2017
- Study completionDec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Actelion
- Responsible party
- Sponsor
- Investigators
- Yin-Hsiu Chien, M.D., Ph.D., Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02520934
- Sponsor's protocol ID
- 201503076MIPD
- Status last verified
- Mar 2019
- First submitted
- 6 Aug 2015
- Last updated
- 29 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB
- ClinicalTrials.gov ID
- NCT02520934
- Acronym / sponsor ID
- 201503076MIPD
- Conditions
- Gaucher Disease
- Keywords
- Enzyme Replacement Therapy, Miglustat, Gaucher Disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 19 participants (actual)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Collaborators
- Actelion
- Study start
- Jul 2015
- Primary completion
- Dec 2017 (actual)
- Study completion
- Dec 2019 (estimated)
- First posted
- 13 Aug 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Case_Miglustat | Experimental | Drug: Miglustat, Drug: ERTBesides regular ERT, patients in this group also need to take Miglustat for 24 months. |
| Control | No intervention | –Patients will be tested for their pupil cycle time. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
- Controlnormal Inclusion Criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MiglustatAlso: Zavescacombine miglustat and ERT to see if neurologic manifestations can be improved | Drug | Case_Miglustat |
| ERTenzyme replacement therapy | Drug | Case_Miglustat |
Primary outcome
Improve in Purdue Pegboard test speed
Time frame: 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yin-Hsiu Chien, M.D., Ph.D. principal investigator
National Taiwan University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Miglustat and ERT in people with Gaucher Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improve in Purdue Pegboard test speed (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2019.