NCT01760564

Brief Summary

To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2008

Typical duration for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2008

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2010

Completed
2.1 years until next milestone

First Submitted

Initial submission to the registry

January 2, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 4, 2013

Completed
Last Updated

January 4, 2013

Status Verified

November 1, 2012

Enrollment Period

2.9 years

First QC Date

January 2, 2013

Last Update Submit

January 3, 2013

Conditions

Niemann-Pick Disease Type C

Keywords

Niemann-Pick disease type Cmiglustat

Outcome Measures

Primary Outcomes (1)

  • Swallowing

    videofluoroscopic swallowing study (VFSS), or NP-C functional disability rating scale if VFSS can not be performed due to safety issue

    12th month

Secondary Outcomes (1)

  • Mental

    12th month

Study Arms (1)

Miglustat

EXPERIMENTAL

miglustat 200mg tid

Drug: Miglustat

Interventions

MiglustatDRUG

miglustat 200mg tid for adult. For children surface area correction will be made. A lower dose may be used initially to decrease side effect.

Also known as: Zavesca
Miglustat

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Confirm diagnosis of Niemann-Pick C disease
  • Symptomatic including motor or mental symptoms

You may not qualify if:

  • Unknown severe diarrhea for more than 7 day
  • Allergy to miglustat

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan

Location

Related Publications (1)

  • 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.

    PMID: 22476655RESULT

MeSH Terms

Conditions

Niemann-Pick Disease, Type C

Interventions

miglustat

Condition Hierarchy (Ancestors)

Niemann-Pick DiseasesSphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHistiocytosis, Non-Langerhans-CellHistiocytosisLymphatic DiseasesHemic and Lymphatic DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Whu-Liang Hwu, MD, PhD

    National Taiwan University Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 2, 2013

First Posted

January 4, 2013

Study Start

January 1, 2008

Primary Completion

December 1, 2010

Study Completion

December 1, 2010

Last Updated

January 4, 2013

Record last verified: 2012-11

Locations

TW(1)