Genetic: UX701 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
171Wilson disease1

171. Wilson disease


Clinical trials : 68 Drugs : 79 - (DrugBank : 17) / Drug target genes : 3 - Drug target pathways : 28
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PhaseCountries
1NCT04884815
(ClinicalTrials.gov)
September 27, 20217/5/2021Clinical Study of UX701 AAV-Mediated 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/AAll90Phase 1/Phase 2United States