303. アッシャー症候群 Usher syndrome Clinical trials / Disease details


臨床試験数 : 10 薬物数 : 11 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

  
1 trial found
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1NCT05158296
(ClinicalTrials.gov)
December 8, 202123/11/2021Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision DisordersDrug: Ultevursen;Other: Sham-procedureProQR TherapeuticsNULLActive, not recruiting12 YearsN/AAll81Phase 2/Phase 3United States;Germany;Netherlands;United Kingdom