Clinical trialNCT00517153CompletedPhase 2Interventional

Miglustat in Niemann-Pick Type C Disease

SponsorActelionEnrollment29Niemann-Pick Type C Disease

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Claudia Chiriboga, Assoc. Prof. MD
Columbia University
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

No facility list is in our current data snapshot for this study. The registry record on ClinicalTrials.gov ↗ carries any published locations. Why? →

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Miglustat →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 31 Jan 2002
Primary completion: 30 Sept 2006
Completion: 31 Jan 2008
Last update posted: 4 Feb 2025
Registered enrollment: 29
Registry id: NCT00517153

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 11 Nov 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.