201. アンジェルマン症候群 Angelman syndrome Clinical trials / Disease details
臨床試験数 : 25 / 薬物数 : 40 - (DrugBank : 11) / 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 |
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1 | NCT05630066 (ClinicalTrials.gov) | June 1, 2023 | 9/11/2022 | Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype | A Phase IIa Multicenter, Open-Label, 12-Week Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype | Angelman Syndrome | Drug: 60 mg QD Alogabat;Drug: 40 mg QD Alogabat;Drug: 7 mg QD Alogabat;Drug: Part 2 Adult Alogabat High Dose (aged 15-17);Drug: Alogabat | Hoffmann-La Roche | NULL | Not yet recruiting | 5 Years | 17 Years | All | 56 | Phase 2 | NULL |