DDrare: Database of Drug Development for Rare Diseases

Carglumic acid (carbaglu®) (DrugBank: Carglumic acid)

3 diseases

ID	Disease name (Link within this page)	Number of trials
245	Propionic acidemia	1
246	Methylmalonic acidemia	1
251	Urea cycle disorder	0

245. Propionic acidemia [ 9 clinical trials, 15 drugs, (DrugBank: 3 drugs), 1 drug target gene, 6 drug target pathways]

Searched query = "Propionic acidemia"
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.

1 / 9 trial 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	NCT04284917 (ClinicalTrials.gov)	November 28, 2019	24/2/2020	Long-term Efficacy of Carglumic Acid in Organic Acidemia.	Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).	Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)	Drug: Carglumic Acid (Carbaglu®)	National Taiwan University Hospital	NULL	Enrolling by invitation	18 Years	N/A	All	5	N/A	Taiwan

246. Methylmalonic acidemia [ 14 clinical trials, 22 drugs, (DrugBank: 2 drugs), 1 drug target gene, 6 drug target pathways]

Searched query = "Methylmalonic acidemia"
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.

1 / 14 trial 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	NCT04284917 (ClinicalTrials.gov)	November 28, 2019	24/2/2020	Long-term Efficacy of Carglumic Acid in Organic Acidemia.	Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).	Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)	Drug: Carglumic Acid (Carbaglu®)	National Taiwan University Hospital	NULL	Enrolling by invitation	18 Years	N/A	All	5	N/A	Taiwan

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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 42 trial found