201. アンジェルマン症候群 Angelman syndrome Clinical trials / Disease details
臨床試験数 : 24 / 薬物数 : 35 - (DrugBank : 9) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 20
| 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 | NCT04259281 (ClinicalTrials.gov) | February 24, 2020 | 31/1/2020 | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome | A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS) | Angelman Syndrome | Drug: GTX-102 | GeneTX Biotherapeutics, LLC | Ultragenyx Pharmaceutical Inc | Recruiting | 4 Years | 17 Years | All | 20 | Phase 1/Phase 2 | United States;Canada;United Kingdom |