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Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

NCT00319046 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 42

Last updated 2025-02-04

Study results available
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Summary

Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy, safety and tolerability of miglustat in maintaining diseases stability after a switch from ERT.

Conditions

  • Gaucher Disease Type 1

Interventions

DRUG

Miglustat

Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.)

Sponsors & Collaborators

  • Actelion

    lead INDUSTRY

Principal Investigators

  • Timothy Cox, Prof · University of Cambridge

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-02-01
Primary Completion
2010-06-01
Completion
2010-07-01

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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 NCT00319046 on ClinicalTrials.gov