256. Muscle glycogenosis Clinical trials / Disease details
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 | 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 | |
| 2 | 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 |