171. ウィルソン病 Wilson disease Clinical trials / Disease details


臨床試験数 : 79 薬物数 : 77 - (DrugBank : 17) / 標的遺伝子数 : 6 - 標的パスウェイ数 : 30

  
1 trial found
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1NCT04884815
(ClinicalTrials.gov)
September 27, 20217/5/2021Study of UX701 Gene Transfer for the Treatment of Wilson DiseaseA Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson DiseaseWilson DiseaseGenetic: UX701;Other: Placebo;Drug: oral corticosteroids;Drug: Placebo for oral corticosteroidsUltragenyx Pharmaceutical IncNULLRecruiting18 YearsN/AAll78Phase 1/Phase 2United States;Canada;Spain