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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1NCT05092685
(ClinicalTrials.gov)
February 1, 202228/9/2021Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEnPhase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency.Ornithine Transcarbamylase DeficiencyGenetic: AAVLK03hOTCUniversity College, LondonNULLNot yet recruitingN/A16 YearsAll12Phase 1/Phase 2NULL