Status | Study |
Recruiting |
Study Name: Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency Condition: Ornithine Transcarbamylase (OTC) Deficiency Date: 2016-12-09 Interventions: Genetic: scAAV8OTC non-replicating, recombinant scAAV8 encoding human ornithine transcarbamylase (OTC) |
Not yet recruiting |
Study Name: Urease Inhibitor Drug Treatment for Urea Cycle Disorders Condition: Ornithine Transcarbamylase Deficiency Argininosuccinate Synthet Date: 2016-01-14 Interventions: Drug: Acetohydroxamic Acid Oth |
Completed |
Study Name: Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants Condition: Urea Cycle Disorders Organic Acidemias Date: 2012-06-13 Interventions: Other: Therapeutic Hypothermia |
Recruiting |
Study Name: Short-term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Condition: Propionic Acidemia, Type I and/or Type II Methylmalonic Acidemia Date: 2012-05-11 Interventions: Drug: Carbaglu |
Completed |
Study Name: Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI Condition: Ornithine Transcarbamylase Deficiency Date: 2012-03-30 Interventions: Other: MRI scanning 1H MRS, DT |
Terminated |
Study Name: The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity Condition: Argininosuccinic Aciduria Carbamoyl-Phosphate Synthase I Deficiency Date: 2011-08-20 |
Suspended |
Study Name: Hepatocyte Transplantation for Liver Based Metabolic Disorders Condition: Metabolic Diseases Date: 2011-04-26 Interventions: Drug: human hepatocyte transplantation |
Completed |
Study Name: Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders Condition: Urea Cycle Disorders Carbamoylphosphate Synthetase I Deficiency Date: 2008-07-16 Interventions: Biological: Human Heterologous Liver Cells Multiple applications of liver cell suspension for infusion |
Completed |
Study Name: Neurologic Injuries in Adults With Urea Cycle Disorders Condition: Brain Diseases, Metabolic, Inborn Urea Cycle Disorder Date: 2007-05-11 |
Terminated |
Study Name: Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency Condition: Ornithine Transcarbamylase Deficiency Disease Date: 1999-10-18 Interventions: Genetic: Adenoviral Vector-Mediated Gene Transfer |