245. Propionic acidemia
[
5 clinical trials,
9 drugs（DrugBank:
4 drugs),
1 target gene / 6 target pathways ]
Searched query = "Propionic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 |