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
- -
A Global Prospective Observational Registry of Patients With Pompe Disease
- Conditions
- Interventions
- First Posted Date
- 2023-11-07
- Last Posted Date
- 2024-10-30
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 500
- Registration Number
- NCT06121011
- Locations
- 🇺🇸
Cincinnati Children's Hospital, Cincinnati, Ohio, United States
🇩🇪Universitätsklinikum Gießen und Marburg GmhH, Marburg, Germany
🇺🇸University of Arkansas Medical Science, Little Rock, Arkansas, United States
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
- Conditions
- Interventions
- First Posted Date
- 2021-03-22
- Last Posted Date
- 2024-11-06
- 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
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
- Conditions
- 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
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
- Conditions
- Interventions
- First Posted Date
- 2019-10-24
- Last Posted Date
- 2024-11-15
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 110
- Registration Number
- NCT04138277
- Locations
- 🇺🇸
University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
🇺🇸University of California, Irvine, Irvine, California, United States
🇺🇸University of Florida Clinical Research Center, Gainesville, Florida, United States
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
- Conditions
- Interventions
- 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
Safety and Efficacy of Miglustat in Chinese NPC Patients
- First Posted Date
- 2019-04-10
- Last Posted Date
- 2022-05-25
- 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
- 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)
- 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
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
- Conditions
- Interventions
- First Posted Date
- 2018-11-02
- Last Posted Date
- 2023-09-11
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 125
- Registration Number
- NCT03729362
- Locations
- 🇺🇸
The Ohio State University Wexner Medical Center, Columbus, Ohio, United States
🇫🇷Hôpital Salengro, Lille, France
🇫🇷Hôpital Pasteur, Nice, France
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
- First Posted Date
- 2016-02-05
- Last Posted Date
- 2024-08-23
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 32
- Registration Number
- NCT02675465
- Locations
- 🇺🇸
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), Fairfax, Virginia, United States
🇺🇸Emory University Division of Medical Genetics, Decatur, Georgia, United States
🇬🇧University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center, Birmingham, United Kingdom
Drug Development Updates
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