CLINICAL TRIALS PROFILE FOR ZAVESCA

All Clinical Trials for ZAVESCA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00319046 ↗	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.
NCT00418847 ↗	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.
NCT00418847 ↗	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.
NCT00517153 ↗	Miglustat in Niemann-Pick Type C Disease	Completed	Actelion	Phase 2	2002-01-01	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.
NCT00537602 ↗	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.
NCT00672022 ↗	Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses	Completed	Actelion	Phase 3	2004-07-01	We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical symptoms.
NCT00672022 ↗	Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses	Completed	Children's National Research Institute	Phase 3	2004-07-01	We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical symptoms.

Clinical Trial Conditions for ZAVESCA

Condition Name

Condition Name for ZAVESCA
Intervention	Trials
Pompe Disease	2
Hypersensitivity Reaction	1
Niemann-Pick Disease Type C	1
Niemann-Pick Disease, Type C	1

Condition MeSH

Condition MeSH for ZAVESCA
Intervention	Trials
Niemann-Pick Disease, Type A	3
Tay-Sachs Disease	3
Gangliosidoses, GM2	3
Gangliosidoses	3

Clinical Trial Locations for ZAVESCA

Trials by Country

Trials by Country for ZAVESCA
Location	Trials
United States	10
Iran, Islamic Republic of	3
Taiwan	3
Canada	3
Spain	2

Trials by US State

Trials by US State for ZAVESCA
Location	Trials
Florida	2
New York	2
District of Columbia	2
Wisconsin	1
Oregon	1

Clinical Trial Progress for ZAVESCA

Clinical Trial Phase

Clinical Trial Phase for ZAVESCA
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	4
Phase 2	3

Clinical Trial Status

Clinical Trial Status for ZAVESCA
Clinical Trial Phase	Trials
Completed	7
Terminated	2
Recruiting	1

Clinical Trial Sponsors for ZAVESCA

Sponsor Name

Sponsor Name for ZAVESCA
Sponsor	Trials
Actelion	8
University of Florida	2
National Taiwan University Hospital	2

Sponsor Type

Sponsor Type for ZAVESCA
Sponsor	Trials
Other	10
Industry	9