245. プロピオン酸血症 ［臨床試験数：5，薬物数：9（DrugBank：4），標的遺伝子数：1，標的パスウェイ数：3］
Searched query = "Propionic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
|1||NCT02426775||November 2015||6 May 2019||Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia||Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)||Propionic Acidemia;Methylmalonic Acidemia||Drug: Carglumic Acid||King Abdullah International Medical Research Center||Not recruiting||N/A||15 Years||All||33||Phase 3||Saudi Arabia|
|2||NCT01597440||September 2012||16 December 2017||Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia||Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia||Propionic Acidemia;Methylmalonic Acidemia||Drug: N-carbamylglutamate;Other: Standard of Care||Mendel Tuchman||Children's Research Institute;Boston Children’s Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Lucile Packard Children's Hospital;University of Colorado, Denver||Not recruiting||N/A||4 Weeks||All||1||Phase 2||United States|
|3||NCT01599286||September 2012||24 June 2019||Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia||Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia||Propionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Deficiency Disease;Ornithine Carbamoyltransferase Deficiency||Drug: Carbaglu;Drug: Placebo;Drug: Standard of Care Treatment||Mendel Tuchman||Children's Research Institute;Boston Children’s Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Stanford University;Icahn School of Medicine at Mount Sinai;University of Pittsburgh;Children's Hospital Colorado||Not recruiting||N/A||99 Years||All||114||Phase 2||United States|
|4||NCT01341379||December 2010||19 February 2015||Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate||Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate||Urea Cycle Disorders, Inborn;Inborn Errors of Metabolism;Propionic Acidemia;Methylmalonic Acidemia;Carbamyl Phosphate Synthetase Deficiency||Drug: N-carbamylglutamate||Children's Hospital of Philadelphia||Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)||Not recruiting||3 Years||70 Years||Both||0||Phase 2||United States|
|5||NCT00645879||July 2008||19 February 2015||Anaplerotic Therapy in Propionic Acidemia||Safety & Efficacy of Investigational Products: Ornithine Alpha-ketoglutarate, Glutamine, or Disodium Citrate on Hyperammonemia in Propionic Acidemia.||Propionic Acidemia||Drug: ornithine alpha ketoglutarate;Drug: glutamine;Drug: disodium citrate||Nicola Longo||National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)||Not recruiting||5 Years||12 Years||Both||3||Phase 1||United States|