NCT00319046

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

Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 42 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	Miglustat	Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.)

Timeline

• Start date: 2006-02-01
• Primary completion: 2010-06-01
• Completion: 2010-07-01
• First posted: 2006-04-27
• Last updated: 2025-02-04
• Results posted: 2012-05-23