Oral prednisone    (DrugBank: Prednisone)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
251尿素サイクル異常症1

251. 尿素サイクル異常症 [臨床試験数:42,薬物数:45(DrugBank:10),標的遺伝子数:4,標的パスウェイ数:29
Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 42 trial found
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PhaseCountries
1NCT02991144
(ClinicalTrials.gov)
January 20179/12/2016Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset 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: Oral prednisoneUltragenyx Pharmaceutical IncNULLRecruiting18 YearsN/AAll18Phase 1/Phase 2United States;Canada;Spain;United Kingdom