256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
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 | NCT04848779 (ClinicalTrials.gov) | June 10, 2021 | 29/3/2021 | A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients Upto 6 Months of Age With Infantile-onset Pompe Disease (IOPD) | A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients =6 Months of Age With Infantile-onset Pompe Disease (IOPD) | Glycogen Storage Disease Type II | Drug: Alglucosidase alfa GZ419829 (Myozyme) | Sanofi | NULL | Recruiting | N/A | 6 Months | All | 16 | Belgium;Germany;Italy;Taiwan |