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

NCT00319046 ↗

Phase 3 Completed 42 enrolled Actelion

NaSingle-groupOpen-labelTreatment

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.

Timeline

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

Outcome

Met primary endpoint

paper The upper 95% confidence limit of mean percent change in liver volume from baseline to end of treatment was below the non-inferiority margin of 10% (-1.1%; 95%CI -6.0, 3.9%). PMID 23270487 ↗

paper The primary efficacy endpoint was met; overall there was no change in liver volume during 24 months of miglustat therapy. PMID 23270487 ↗

Publications

Results Cox TM, Amato D, Hollak CE, Luzy C, Silkey M, Giorgino R, Steiner RD; Miglustat Maintenance Study Group. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study. Orphanet J Rare Dis. 2012 Dec 27;7:102. doi: 10.1186/1750-1172-7-102.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Miglustat	Small molecule	100 mg	Oral