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 | NCT03019406 (ClinicalTrials.gov) | October 12, 2017 | 20/12/2016 | A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa | An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response | Glycogen Storage Disease Type II-Pompe's Disease | Drug: avalglucosidase alfa GZ402666;Drug: alglucosidase alfa GZ419829 | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 6 Months | 17 Years | All | 22 | Phase 2 | United States;France;Japan;Taiwan;United Kingdom |