251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details


臨床試験数 : 54 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28

  
1 trial found
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1NCT02991144
(ClinicalTrials.gov)
July 31, 20179/12/2016Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) DeficiencyA Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC DeficiencyOrnithine Transcarbamylase (OTC) DeficiencyGenetic: scAAV8OTC;Drug: Reactive Corticosteroid Taper Regimen;Drug: Prophylactic Corticosteroid Taper RegimenUltragenyx Pharmaceutical IncNULLCompleted18 YearsN/AAll16Phase 1/Phase 2United States;Canada;Spain;United Kingdom