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: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.
1 / 19 trial found
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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 IncActive, not recruiting4 Years17 YearsAll20Phase 1;Phase 2United States