251. Urea cycle disorder
42 clinical trials, 45 drugs (DrugBank: 10 drugs), 4 drug target genes, 29 drug target pathways
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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2006-000136-27-DE (EUCTR) | 17/03/2008 | 04/09/2007 | Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (UCDs) | Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (UCDs) | Neonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with below listed deficiency and children aged > 3 months up to 5 years including with confirmed urea cycle disorder and unstable metabolism with deficiency of either: - Carbamyl-phosphate Synthetase I Deficiency (CPS1D) - Ornithine Transcarbamylase Deficiency (OTCD) - Argininosuccinate Synthetase Deficiency (ASSD/Citrullinaemia)can be included. MedDRA version: 18.1;Level: PT;Classification code 10052450;Term: Ornithine transcarbamoylase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10058298;Term: Argininosuccinate synthetase deficiency;Classification code 10058297;Term: Carbamoyl phosphate synthetase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Human heterologous liver cells (for infusion) Product Code: HHLivC | Cytonet GmbH & Co KG | NULL | Not Recruiting | Female: yes Male: yes | Germany | ||||
| 2 | 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 |