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

  
1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05630066
(ClinicalTrials.gov)
June 1, 20239/11/2022Study 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 GenotypeA 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 GenotypeAngelman SyndromeDrug: 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: AlogabatHoffmann-La RocheNULLNot yet recruiting5 Years17 YearsAll56Phase 2NULL