201. アンジェルマン症候群
[臨床試験数:19,薬物数:30(DrugBank:7),標的遺伝子数:22,標的パスウェイ数:20]
Searched query = "Angelman syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
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 | Active, not recruiting | 4 Years | 17 Years | All | 20 | Phase 1;Phase 2 | United States |