Niemann-Pick Disease Type C Clinical Trial
Official title:
Application of Miglustat in Patients With Niemann-Pick Type C
| Verified date | November 2012 |
| Source | National Taiwan University Hospital |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Taiwan: Department of Health |
| Study type | Interventional |
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
| Status | Completed |
| Enrollment | 5 |
| Est. completion date | December 2010 |
| Est. primary completion date | December 2010 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | N/A and older |
| Eligibility |
Inclusion Criteria: - Confirm diagnosis of Niemann-Pick C disease - Symptomatic including motor or mental symptoms Exclusion Criteria: - Unknown severe diarrhea for more than 7 day - Allergy to miglustat |
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Taiwan | National Taiwan University Hospital | Taipei |
| Lead Sponsor | Collaborator |
|---|---|
| National Taiwan University Hospital |
Taiwan,
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 — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Swallowing | videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue | 12th month | No |
| Secondary | Mental | IQ test or Mini-Mental Status Examination if a full IQ test can not be performed | 12th month | No |