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 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

Clinical Trials

A Global Prospective Observational Registry of Patients With Pompe Disease

Recruiting

Conditions: Pompe Disease

Interventions: Other: Untreated
Biological: Alglucosidase alfa or Avalglucosidase alfa
Biological: Cipaglucosidase alfa
Drug: Miglustat

First Posted Date: 2023-11-07

Last Posted Date: 2025-04-23

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 500

Registration Number: NCT06121011

Locations: 🇩🇪
Ruhr-Universität Bochum im St. Josef-Hospital, Bochum, Germany
🇩🇪
SphinCS, Institute of Clinical Science in LSD, Hochheim, Germany
🇩🇪
Universitätsklinikum Gießen und Marburg GmhH, Marburg, Germany

and more 21 locations

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease

Phase 1

Active, not recruiting

Conditions: Batten Disease

Interventions: Drug: Miglustat 100Mg Oral Capsule

First Posted Date: 2021-12-30

Last Posted Date: 2024-04-26

Lead Sponsor: Beyond Batten Disease Foundation

Target Recruit Count: 6

Registration Number: NCT05174039

Locations: 🇺🇸
Texas Children Hospital, Houston, Texas, United States

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Phase 3

Recruiting

Conditions: Glycogen Storage Disease Type II Infantile Onset

Interventions: Biological: Cipaglucosidase alfa
Drug: Miglustat

First Posted Date: 2021-03-22

Last Posted Date: 2025-03-18

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 36

Registration Number: NCT04808505

Locations: 🇺🇸
University of Florida Clinical Research Center, Gainesville, Florida, United States
🇺🇸
The Emory Clinic, Atlanta, Georgia, United States
🇺🇸
Duke University Early Phase Research Unit, Durham, North Carolina, United States

and more 14 locations

Testing Miglustat Administration in Subjects With Spastic Paraplegia 11

Phase 2

Completed

Conditions: Hereditary Spastic Paraparesis

Interventions: Drug: Miglustat 100 MG

First Posted Date: 2021-02-24

Last Posted Date: 2022-04-11

Lead Sponsor: IRCCS Fondazione Stella Maris

Target Recruit Count: 10

Registration Number: NCT04768166

Locations: 🇮🇹
IRCCS Fondazione Stella Maris, Pisa, PI, Italy

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Conditions: Pompe Disease Infantile-Onset

First Posted Date: 2020-03-31

Last Posted Date: 2024-08-16

Lead Sponsor: Amicus Therapeutics

Registration Number: NCT04327973

Locations: 🇺🇸
UCSF Benioff Children's Hospital Oakland, Oakland, California, United States
🇺🇸
UF Health Shands Hospital, Gainesville, Florida, United States
🇮🇹
University Hospital of Padova, Padova, Italy

and more 1 locations

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Phase 3

Completed

Conditions: Pompe Disease (Late-onset)

Interventions: Drug: AT2221
Biological: ATB200

First Posted Date: 2019-10-24

Last Posted Date: 2025-03-20

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 119

Registration Number: NCT04138277

Locations: 🇭🇺
Semmelweis University, Budapest, Hungary
🇭🇺
University of Pécs, Pécs, Hungary
🇭🇺
University of Szeged, Szeged, Hungary

and more 57 locations

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Phase 3

Recruiting

Conditions: Pompe Disease (Late-onset)

Interventions: Biological: Cipaglucosidase Alfa
Drug: Miglustat

First Posted Date: 2019-04-11

Last Posted Date: 2024-07-01

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 22

Registration Number: NCT03911505

Locations: 🇺🇸
Woodruff Memorial Research Building, Atlanta, Georgia, United States
🇺🇸
UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States

and more 20 locations

Safety and Efficacy of Miglustat in Chinese NPC Patients

Phase 4

Completed

Conditions: Niemann-Pick Disease, Type C

Interventions: Drug: Miglustat

First Posted Date: 2019-04-10

Last Posted Date: 2025-03-30

Lead Sponsor: Actelion

Target Recruit Count: 17

Registration Number: NCT03910621

Locations: 🇨🇳
Xin Hua Hospital, Shanghai Jiao Tong University, Shanghai, China
🇨🇳
Peking University first Hospital, Beijing, China

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Conditions: Pompe Disease

First Posted Date: 2019-03-07

Last Posted Date: 2024-08-16

Lead Sponsor: Amicus Therapeutics

Registration Number: NCT03865836

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Phase 3

Recruiting

Conditions: GM2 Gangliosidosis
Supportive Care

Interventions: Drug: Miglustat

First Posted Date: 2019-01-30

Last Posted Date: 2024-04-16

Lead Sponsor: Tehran University of Medical Sciences

Target Recruit Count: 30

Registration Number: NCT03822013

Locations: 🇮🇷
Kashan University Of Medical Sciences, Kashan, Isfahan, Iran, Islamic Republic of
🇮🇷
Tehran University Of Medical Sciences, Tehran, Iran, Islamic Republic of
🇮🇷
Mashhad University Of Medical Sciences, Mashhad, Khorasan, Iran, Islamic Republic of

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