245. Propionic acidemia Clinical trials / Disease details

Clinical trials : 13 / Drugs : 17 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathways : 6

3 trials found

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	NCT01597440 (ClinicalTrials.gov)	September 2012	10/5/2012	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	Terminated	N/A	4 Weeks	All	1	Phase 2	United States
2	NCT01599286 (ClinicalTrials.gov)	September 2012	11/5/2012	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 National 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	Active, not recruiting	N/A	99 Years	All	114	Phase 2	United States
3	NCT01341379 (ClinicalTrials.gov)	December 2010	22/4/2011	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Hospital of Philadelphia	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Withdrawn	3 Years	70 Years	Both	0	Phase 2	United States