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 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
-

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)

Phase 2
Completed
Conditions
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

and more 6 locations

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

Phase 2
Completed
Conditions
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

and more 3 locations

Phase I Rising Dose Tolerability Study of SC-48334 in Patients With Acquired Immunodeficiency Syndrome (AIDS) and Advanced AIDS Related Complex

Phase 1
Completed
Conditions
First Posted Date
2001-08-31
Last Posted Date
2005-06-24
Lead Sponsor
G D Searle
Target Recruit Count
48
Registration Number
NCT00000692
Locations
🇺🇸

Julio Arroyo, West Columbia, South Carolina, United States

🇺🇸

Univ of Miami School of Medicine, Miami, Florida, United States

🇺🇸

Stanford Univ School of Medicine, Stanford, California, United States

and more 3 locations
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