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Miglustat in Niemann-Pick Type C Disease

NCT00517153 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2025-02-04

No results posted yet for this 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.

Conditions

  • Niemann-Pick Type C Disease

Interventions

DRUG

miglustat

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

Sponsors & Collaborators

  • Actelion

    lead INDUSTRY

Principal Investigators

  • Claudia Chiriboga, Assoc. Prof. MD · Columbia University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2002-01-31
Primary Completion
2006-09-30
Completion
2008-01-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00517153 on ClinicalTrials.gov