337. Homocystinuria Clinical trials / Disease details


Clinical trials : 13 Drugs : 13 - (DrugBank : 4) / Drug target genes : 2 - Drug target pathways : 23

  
1 trial found
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1NCT01793090
(ClinicalTrials.gov)
January 20138/2/2013EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological ImpairmentPhase 2, Double-Blind, Placebo Controlled Clinical Trial of EPI-743 in Subjects With Cobalamin C DefectMethylmalonic Aciduria and Homocystinuria,Cblc Type;Genetic Disease;RetinopathyDrug: Epi-743;Other: Placebo supplementationBambino Gesù Hospital and Research InstituteCatholic University of the Sacred HeartCompleted1 Year20 YearsAll30Phase 2Italy