Cipaglucosidase alfa (ATB200) ( DrugBank: Cipaglucosidase alfa )
1 disease
| 告示番号 | 疾患名(ページ内リンク) | 臨床試験数 | 
|---|---|---|
| 256 | 筋型糖原病 | 1 | 
256. 筋型糖原病
臨床試験数 : 180 / 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 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 |