Clinical Trial: Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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

Brief 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.

Detailed Summary:
Sponsor: Actelion

Current Primary Outcome:

• Liver Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]
  Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging
• Percent Change in Liver Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]
  Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging

Original Primary Outcome: Percent change from baseline to Month 24 in liver volume

Current Secondary Outcome:

• Spleen Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]
  Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging
• Percent Change in Spleen Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]
  Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging

Original Secondary Outcome: Percent change from baseline to Month 24 in spleen volume

Information By: Actelion

Dates:
Date Received: April 26, 2006
Date Started: February 2006
Date Completion:
Last Updated: May 24, 2012
Last Verified: May 2012