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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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02991144 (ClinicalTrials.gov)	July 31, 2017	9/12/2016	Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamy ...	A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AA ...	Ornithine Transcarbamylase (OTC) Deficiency	Genetic: scAAV8OTC;Drug: Reactive Corticosteroid Taper Regimen;Drug: Prophylactic Corticosteroid Tap ...	Ultragenyx Pharmaceutical Inc	NULL	Completed	18 Years	N/A	All	16	Phase 1/Phase 2	United States;Canada;Spain;United Kingdom

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