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CompletedNCT00517153Updated Feb 4, 2025

Miglustat in Niemann-Pick Type C Disease

A Phase 2 interventional study of miglustat in Niemann-Pick Type C Disease, sponsored by Actelion. Completed. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2025-02-04.

Sponsored by Actelion · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
29
Allocation
Randomized
Ages
4 Years and older
Sex
All
01

Study summary

This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a non-treatment group. Both groups will follow an identical visit schedule.

02

Conditions studied

  • Niemann-Pick Type C Disease

Keywords

  • Zavesca
  • miglustat
  • Niemann-Pick Type C
  • Actelion
03

In context

Niemann-Pick Disease, Type C

55 studies on the registry are indexed under Niemann-Pick Disease, Type C; 11 are open to participants now.

This study's enrollment of 29 is above the median of 25 across 38 interventional studies indexed under Niemann-Pick Disease, Type C.

Browse Niemann-Pick Disease, Type C studies →

Lead sponsor

Actelion is the lead sponsor of 140 studies on the registry; 1 is open to participants now.

Of its 27 completed or terminated interventional studies of FDA-regulated products, 24 (89%) have results posted.

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

04

Who can participate

Ages eligible
4 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Patients with Niemann-Pick Type C disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
  2. Patients who can ingest a capsule.
  3. Patients who are above the age of four (4) years of age.

Exclusion criteria

Exclusion Criteria:

  1. Patients younger than 18 years who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
  2. Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
  3. Sexually active and fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment.
  4. Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
  5. Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
  6. Patients suffering from clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 3 months of Screening Visit, or who have a history of significant gastrointestinal disorders.
  7. Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
  8. Patients who in the opinion of the Investigator (for whatever reason) are thought to be unsuitable for the study.
  9. Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m2 (CrCl \<70).
  10. Patients younger than four (4) years of age.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
29 participants (actual)

Study arms

  • Experimental
    1

    OGT-918 - Zavesca (miglustat)

    Drug: miglustat

  • No intervention
    2

    Standard treatment

Interventions

  • Drugmiglustat

    Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT 918 three times daily initially for twelve months. An additional sub-study of up to 12 patients aged under 12 years will receive a lower dose of OGT 918 according to their BSA

    Also known as: Zavesca

06

What researchers measure

Primary outcomes

  1. The primary endpoints will be two parameters - Horizontal Saccadic α and Horizontal Saccadic β - which are estimated for each patient from their saccadic eye movement data

    Time frame: Baseline to Month 12

Secondary outcomes

  1. Highest amplitude for which a velocity measurement is obtained

    Time frame: Baseline to Month 12

07

Study locations

No study locations are listed for this record.

08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 4, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00517153
Lead sponsor
Actelion
First posted
Aug 16, 2007
Start date
Jan 2002
Primary completion
Sep 2006
Completion
Jan 2008
Last update
Feb 4, 2025

Study contacts

Claudia Chiriboga, Assoc. Prof. MD
principal investigator · Columbia University

Oversight

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

Not currently enrolling

This study is completed, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.

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