GTX-102 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
201アンジェルマン症候群1

201. アンジェルマン症候群


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.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
1NCT04259281
(ClinicalTrials.gov)
February 24, 202031/1/2020A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeA 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 SyndromeDrug: GTX-102GeneTX Biotherapeutics, LLCUltragenyx Pharmaceutical IncRecruiting4 Years17 YearsAll20Phase 1/Phase 2United States;Canada;United Kingdom