Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Completed
Actelion
Phase 3
2006-02-01
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.
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Completed
Actelion
Phase 2
2004-07-01
The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat,
OGT918) when given as single and multiple doses in juvenile patients with GM2 gangliosidosis.
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Completed
The Hospital for Sick Children
Phase 2
2004-07-01
The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat,
OGT918) when given as single and multiple doses in juvenile patients with GM2 gangliosidosis.
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.
Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
Terminated
Actelion
Phase 2
2007-11-01
Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane
conductance regulator (CFTR). The purpose of the study is to investigate the effects of
miglustat on CFTR function in cystic fibrosis patients.
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