Miglustat (AT2221) ( DrugBank: Miglustat )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 256 | Muscle glycogenosis | 2 |
256. Muscle glycogenosis
Clinical trials : 180 / Drugs : 153 - (DrugBank : 30) / Drug target genes : 31 - Drug target pathways : 134
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04808505 (ClinicalTrials.gov) | August 2022 | 10/3/2021 | Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | An Open-label Study to Evaluate the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years | Glycogen Storage Disease Type II Infantile Onset | Biological: Cipaglucosidase alfa (ATB200);Drug: Miglustat (AT2221) | Amicus Therapeutics | NULL | Recruiting | N/A | 17 Years | All | 22 | Phase 3 | United States |
| 2 | JPRN-JapicCTI-194887 | 17/9/2019 | 26/07/2019 | ATB200/AT2221phase3 double blind randomized study | A phase 3 double-blind randomized study to assess the efficacy and safety of intravenous ATB200 co-administered with oral AT2221 in adult subjects with late- onset pompe disease compared with alglucosidase alfa/placebo | Pompe disease | Intervention name : Recombinant human acid alfa-glucosidase (ATB200) INN of the intervention : Recombinant human acid alfa-glucosidase Dosage And administration of the intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after AT2221(Miglustat) administration every 2 weeks Intervention name : Miglustat (AT2221) INN of the intervention : Miglustat Dosage And administration of the intervention : Subjects weighing >= 50 kg, 260mg (4 oral capsules of AT2221) 1hour prior to ATB200 infusion every 2 weeks. Subjects weighing >= 40 kg to < 50 kg, 195 mg (3 oral capsules) 1 hour prior to ATB200 infusion every 2 weeks. Control intervention name : Alglucosidase Alfa (Genetical Recombination) INN of the control intervention : Alglucosidase Alfa (Genetical Recombination) Dosage And administration of the control intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after placebo administration every 2 weeks Control intervention name : Plecebo INN of the control intervention : - Dosage And administration of the control intervention : - | Amicus Therapeutics, Inc./CMIC Co., Ltd. | NULL | complete | 18 | BOTH | 110 | Phase 3 | Japan, Asia except Japan, North America, South America, Europe, Oceania |