256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
| 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 | NCT03687333 (ClinicalTrials.gov) | December 4, 2018 | 24/9/2018 | Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment | A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment | Glycogen Storage Disease Type II | Drug: ALGLUCOSIDASE ALFA (MYOZYME) | Genzyme, a Sanofi Company | NULL | Completed | N/A | 12 Months | All | 10 | Phase 4 | China |