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.
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 |
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1 | EUCTR2015-000988-14-Outside-EU/EEA (EUCTR) | 12/10/2016 | Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders | Title: Open, Prospective, Historic-Controlled, Multicenter Study toEvaluate the Safety and Efficacy of Infusion of Liver Cell Suspension(HHLivC) in Children with Urea Cycle Disorders | Neonatal-onset type of the following urea cycle disorders(UCD):ornithine transcarbamylase deficiency (OTCD),carbamoylphosphate synthetase I deficiency (CPS1D), orargininosuccinate synthetase deficiency (ASSD orcitrullinemia);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Heparesc Product Code: HHLivC INN or Proposed INN: not available Other descriptive name: ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS | PROMETHERA Biosciences S.A./N.V. | NULL | NA | Female: yes Male: yes | 21 | Phase 2 | United States;Canada |