Miglustat
- Generic Name
- Miglustat
- Brand Names
- Opfolda, Zavesca, Miglustat Dipharma, Miglustat Gen.Orph, Yargesa
- Drug Type
- Small Molecule
- Chemical Formula
- C10H21NO4
- 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 approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.
- 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 patients with Niemann-Pick disease type C (NP-C).
- Associated Conditions
- Mild, moderate Gaucher Disease, Type 1
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
- Conditions
- GM2 GangliosidosesTay-SachsSandhoff Disease
- First Posted Date
- 2008-05-06
- Last Posted Date
- 2008-05-06
- Lead Sponsor
- Children's National Research Institute
- Target Recruit Count
- 10
- Registration Number
- NCT00672022
- Locations
- 🇺🇸
Children's National Medical Center, Washington, District of Columbia, United States
Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
- 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
- First Posted Date
- 2007-08-16
- Last Posted Date
- 2025-02-04
- 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
- 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
- First Posted Date
- 2006-04-27
- Last Posted Date
- 2025-03-25
- 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
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
- Conditions
- Niemann Pick Diseases
- First Posted Date
- 2006-04-20
- Last Posted Date
- 2017-07-02
- Lead Sponsor
- National Eye Institute (NEI)
- Target Recruit Count
- 30
- Registration Number
- NCT00316498
- Locations
- 🇺🇸
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
🇺🇸Columbia University, New York, New York, United States
🇬🇧Oxford GlycoSciences Ltd., Abingdon, United Kingdom
Glycosphingolipid Inhibition and Spermatogenesis in Man: A Pilot Study (MIG 2)
- First Posted Date
- 2005-09-19
- Last Posted Date
- 2008-09-19
- Lead Sponsor
- University of Washington
- Target Recruit Count
- 8
- Registration Number
- NCT00194649
- Locations
- 🇺🇸
University of Washington, Seattle, Washington, United States
OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
- Conditions
- Gaucher Disease
- First Posted Date
- 2002-07-10
- Last Posted Date
- 2017-07-02
- Target Recruit Count
- 30
- Registration Number
- NCT00041535
- Locations
- 🇬🇧
Institute of Child Health, London, United Kingdom
🇺🇸National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
Phase II Study of the Safety and Surrogate Marker Efficacy of Butyldeoxynojirimycin (SC-48334) and AZT in Symptomatic HIV-1 Infected Patients With 200 - 500 CD4+ Cells/mm3. (NOTE: Asymptomatic HIV-1 Infected Patients Also Eligible)
- Conditions
- HIV Infections
- First Posted Date
- 2001-08-31
- Last Posted Date
- 2005-06-24
- Lead Sponsor
- G D Searle
- Registration Number
- NCT00002079
- Locations
- 🇺🇸
West Paces Clinical Research Incoporated, Atlanta, Georgia, United States
🇺🇸Stratogen of South Florida, Miami Beach, Florida, United States
🇺🇸Dr Daniel Barbero, Fort Worth, Texas, United States
Initial Phase II Efficacy and Safety Study of SC-48334 Administered in Combination With Low-Dose Zidovudine (AZT) to Symptomatic HIV-1 Infected Patients With = or > 200 to = or < 500 CD4+ Cells/mm3
- Conditions
- HIV Infections
- First Posted Date
- 2001-08-31
- Last Posted Date
- 2005-06-24
- Lead Sponsor
- G D Searle
- Target Recruit Count
- 120
- Registration Number
- NCT00001993
- Locations
- 🇺🇸
Rush Presbyterian - Saint Luke's Med Ctr, Chicago, Illinois, United States
🇺🇸Univ of Miami School of Medicine, Miami, Florida, United States
🇺🇸Infectious Diseases Research Clinic / Indiana Univ Hosp, Indianapolis, Indiana, United States
Drug Development Updates
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