Niemann-Pick Disease Type CPhase 3NCT01760564
Application of Miglustat in Patients With Niemann-Pick Type C
A Phase 3 study testing Miglustat in people with Niemann-Pick Disease Type C, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Niemann-Pick Disease Type C
- What is being tested?
- Miglustat
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteTaiwan
- Study dates
- Jan 2008to Dec 2010
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirm diagnosis of Niemann-Pick C disease
- Symptomatic including motor or mental symptoms
Who cannot join
- Unknown severe diarrhea for more than 7 day
- Allergy to miglustat
Trial locations and contacts
1 site in 1 country.
About this study
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
Read the detailed description
Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.
- Conditions
- Niemann-Pick Disease Type C
- Being tested
- Miglustat
- Keywords
- Niemann-Pick disease type C, miglustat
- Medical subject headings
- Niemann-Pick Disease, Type C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MiglustatDrug
Also called Zavesca
miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.
| Study group | Role | What they receive |
|---|---|---|
| Miglustat | Experimental | miglustat 200mg tid Receives: Drug: Miglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Swallowing
videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue
Measured: 12th month
Secondary outcomes (1)
Mental
IQ test or Mini-Mental Status Examination if a full IQ test can not be performed
Measured: 12th month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2013 (estimated)
- Study startJan 2008
- Primary completionDec 2010
- Study completionDec 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Niemann-Pick Disease Type CStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Whu-Liang Hwu, MD, PhD, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01760564
- Sponsor's protocol ID
- 200802043M
- Status last verified
- Nov 2012
- First submitted
- 2 Jan 2013
- Last updated
- 4 Jan 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Chien YH, Peng SF, Yang CC, Lee NC, Tsai LK, Huang AC, Su SC, Tseng CC, Hwu WL. Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. J Inherit Metab Dis. 2013 Jan;36(1):129-37. doi: 10.1007/s10545-012-9479-9. Epub 2012 Apr 5.
PubMed 22476655
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Application of Miglustat in Patients With Niemann-Pick Type C
- ClinicalTrials.gov ID
- NCT01760564
- Acronym / sponsor ID
- 200802043M
- Conditions
- Niemann-Pick Disease Type C
- Keywords
- Niemann-Pick disease type C, miglustat
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- Jan 2008
- Primary completion
- Dec 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 4 Jan 2013 (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.
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
Detailed description
Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Miglustat | Experimental | Drug: Miglustatmiglustat 200mg tid |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Confirm diagnosis of Niemann-Pick C disease
- Symptomatic including motor or mental symptoms
Key exclusion criteria (2 listed)
- Unknown severe diarrhea for more than 7 day
- Allergy to miglustat
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: Zavescamiglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect. | Drug | Miglustat |
Primary outcome
Swallowing
Time frame: 12th month
videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue
1 secondary outcome
Mental
Time frame: 12th month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2013.
- Results publication: PubMed 22476655Chien YH, Peng SF, Yang CC, Lee NC, Tsai LK, Huang AC, Su SC, Tseng CC, Hwu WL. Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. J Inherit Metab Dis. 2013 Jan;36(1):129-37. doi: 10.1007/s10545-012-9479-9. Epub 2012 Apr 5.
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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.
Whu-Liang Hwu, MD, PhD 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 Phase 3 study testing Miglustat in people with Niemann-Pick Disease Type C, 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 2 inclusion and 2 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: finished, as of the sponsor's last update on 4 Jan 2013. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Swallowing (time frame: 12th month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01760564, which also lists 1 linked publication.
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 4 Jan 2013.