Miglustat

Generic Name: Miglustat

Brand Names: Opfolda, Zavesca, Miglustat Dipharma, Miglustat Gen.Orph, Yargesa

Drug Type: Small Molecule

Chemical Formula: C_₁₀H_₂₁NO_₄

CAS Number: 72599-27-0

Unique Ingredient Identifier: ADN3S497AZ

Background: Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is now the first and only approv...

Indication: For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric pat...

Associated Conditions: Mild, moderate Gaucher Disease, Type 1

Associated Therapies: -

Clinical Trials

Miglustat on Gaucher Disease Type IIIB

Not Applicable

Conditions: Gaucher Disease

Interventions: Drug: Miglustat
Drug: ERT

First Posted Date: 2015-08-13

Last Posted Date: 2019-03-29

Lead Sponsor: National Taiwan University Hospital

Target Recruit Count: 19

Registration Number: NCT02520934

Locations: 🇨🇳
National Taiwan University Hospital, Taipei, Taiwan

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Phase 4

Terminated

Conditions: GM1 Gangliosidoses
GM2 Gangliosidoses
Tay-Sachs Disease
Sandhoff Disease

Interventions: Drug: miglustat
Other: Ketogenic Diet

First Posted Date: 2014-01-08

Last Posted Date: 2021-04-14

Lead Sponsor: University of Minnesota

Target Recruit Count: 16

Registration Number: NCT02030015

Locations: 🇺🇸
University of Minnesota, Minneapolis, Minnesota, United States

Application of Miglustat in Patients With Niemann-Pick Type C

Phase 3

Completed

Conditions: Niemann-Pick Disease Type C

Interventions: Drug: Miglustat

First Posted Date: 2013-01-04

Last Posted Date: 2013-01-04

Lead Sponsor: National Taiwan University Hospital

Target Recruit Count: 5

Registration Number: NCT01760564

Locations: 🇨🇳
National Taiwan University Hospital, Taipei, Taiwan

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Completed

Conditions: Pompe Disease

Interventions: Drug: Rituximab
Drug: Miglustat

First Posted Date: 2011-10-14

Last Posted Date: 2017-12-08

Lead Sponsor: University of Florida

Target Recruit Count: 11

Registration Number: NCT01451879

Locations: 🇺🇸
University of Florida, Gainesville, Florida, United States

Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?

Phase 2

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: Placebo
Drug: Miglustat

First Posted Date: 2009-07-24

Last Posted Date: 2011-08-10

Lead Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Target Recruit Count: 10

Registration Number: NCT00945347

Locations: 🇧🇪
Cliniques Universitaires St Luc (Université Catholique de Louvain) 10 avenue Hippocrate, Brussels, Belgium

Miglustat in Cystic Fibrosis

Phase 2

Completed

Conditions: Cystic Fibrosis

Interventions: Drug: miglustat
Drug: placebo

First Posted Date: 2008-08-27

Last Posted Date: 2014-03-07

Lead Sponsor: Actelion

Target Recruit Count: 11

Registration Number: NCT00742092

Locations: 🇧🇪
Universite Catholique de Louvain, Brussels, Belgium

Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Phase 2

Terminated

Conditions: Cystic Fibrosis

Interventions: Drug: miglustat
Drug: placebo

First Posted Date: 2007-10-01

Last Posted Date: 2010-02-12

Lead Sponsor: Actelion

Target Recruit Count: 6

Registration Number: NCT00537602

Locations: 🇪🇸
Corporacio Parc Tauli / Parc Tauli Hospital, Barcelona, Spain

Miglustat in Niemann-Pick Type C Disease

Phase 2

Completed

Conditions: Niemann-Pick Type C Disease

Interventions: Drug: miglustat

First Posted Date: 2007-08-16

Last Posted Date: 2010-03-26

Lead Sponsor: Actelion

Target Recruit Count: 29

Registration Number: NCT00517153

Locations: 🇺🇸
Columbia University Medical Center, New York, New York, United States

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

Phase 2

Completed

Conditions: Gangliosidoses GM2

Interventions: Drug: miglustat

First Posted Date: 2007-01-05

Last Posted Date: 2016-05-19

Lead Sponsor: The Hospital for Sick Children

Target Recruit Count: 5

Registration Number: NCT00418847

Locations: 🇨🇦
The Hospital for Sick Children, Toronto, Ontario, Canada

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

Phase 3

Completed

Conditions: Gaucher Disease Type 1

Interventions: Drug: Miglustat

First Posted Date: 2006-04-27

Last Posted Date: 2018-11-21

Lead Sponsor: Actelion

Target Recruit Count: 42

Registration Number: NCT00319046

Locations: 🇺🇸
NYU School of Medicine, New York, New York, United States
🇦🇺
Royal Melbourne Hospital, Victoria, Australia
🇦🇺
Royal Brisbane and Women's Hospital, Queensland, Australia

and more 17 locations

Drug Development Updates

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