CLINICAL TRIALS PROFILE FOR CARBAGLU

All Clinical Trials for Carbaglu

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00843921 ↗	N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia	Active, not recruiting	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2/Phase 3	2008-08-01	This study is based on the hypothesis that a new drug N-carbamylglutamate (Carbaglu®) will enhance the ability of the liver to dispose of toxic ammonia which accumulates in several metabolic diseases including urea cycle disorders and organic acid disorders.
NCT00843921 ↗	N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia	Active, not recruiting	Mendel Tuchman	Phase 2/Phase 3	2008-08-01	This study is based on the hypothesis that a new drug N-carbamylglutamate (Carbaglu®) will enhance the ability of the liver to dispose of toxic ammonia which accumulates in several metabolic diseases including urea cycle disorders and organic acid disorders.
NCT01341379 ↗	Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate	Withdrawn	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	2010-12-01	Hyperammonemia, which can cause brain damage, occurs in many different kinds of inborn errors of metabolism. The investigators propose to determine if short-term (3 day) treatment with N-carbamylglutamate can diminish hyperammonemia by enhancing ureagenesis in these patients. The investigators propose here a short-term (3 day) trial. If it succeeds, the investigators would consider more extensive long-term studies of the drug.
NCT01341379 ↗	Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate	Withdrawn	Children's Hospital of Philadelphia	Phase 2	2010-12-01	Hyperammonemia, which can cause brain damage, occurs in many different kinds of inborn errors of metabolism. The investigators propose to determine if short-term (3 day) treatment with N-carbamylglutamate can diminish hyperammonemia by enhancing ureagenesis in these patients. The investigators propose here a short-term (3 day) trial. If it succeeds, the investigators would consider more extensive long-term studies of the drug.
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Boston Children's Hospital	Phase 2	2012-09-01	Background: Very few drugs exist that treat hyperammonemia, specifically PA and MMA. Diet restrictions and alternate pathway agents are the current primary treatments, but they frequently fail to prohibit brain damage. Orthotopic liver transplantation cures the hyperammonemia of urea cycle disorders, but organ availability is limited and the procedure is highly invasive and requires life-long immunosuppression. A drug that could repair or stimulate a dysfunctional urea cycle such as this would have several advantages over current therapy. A drug called N-carbamyl-L-glutamate, Carglumic acid (NCG or Carbaglu)has recently been found to be virtually curative of another urea cycle defect called NAGS deficiency. In this disorder, treatment with NCG alone normalizes ureagenesis, blood ammonia and glutamine levels, allows normal protein tolerance and restores health. Knowledge from this study is being applied to acquired hyperammonemia, specifically in patients with propionic PA and MMA, to try and improve neurodevelopmental outcomes by improving the hyperammonemia. Aims: The overall objective of this project is to determine whether treatment of acute hyperammonemia with Carglumic acid in propionic acidemia (PA), methylmalonic acidemia (MMA) changes the long-term outcome of disease and to determine if it is effective in restoring urine ammonia levels to normal levels.
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Boston Children’s Hospital	Phase 2	2012-09-01	Background: Very few drugs exist that treat hyperammonemia, specifically PA and MMA. Diet restrictions and alternate pathway agents are the current primary treatments, but they frequently fail to prohibit brain damage. Orthotopic liver transplantation cures the hyperammonemia of urea cycle disorders, but organ availability is limited and the procedure is highly invasive and requires life-long immunosuppression. A drug that could repair or stimulate a dysfunctional urea cycle such as this would have several advantages over current therapy. A drug called N-carbamyl-L-glutamate, Carglumic acid (NCG or Carbaglu)has recently been found to be virtually curative of another urea cycle defect called NAGS deficiency. In this disorder, treatment with NCG alone normalizes ureagenesis, blood ammonia and glutamine levels, allows normal protein tolerance and restores health. Knowledge from this study is being applied to acquired hyperammonemia, specifically in patients with propionic PA and MMA, to try and improve neurodevelopmental outcomes by improving the hyperammonemia. Aims: The overall objective of this project is to determine whether treatment of acute hyperammonemia with Carglumic acid in propionic acidemia (PA), methylmalonic acidemia (MMA) changes the long-term outcome of disease and to determine if it is effective in restoring urine ammonia levels to normal levels.
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Children's Hospital of Philadelphia	Phase 2	2012-09-01	Background: Very few drugs exist that treat hyperammonemia, specifically PA and MMA. Diet restrictions and alternate pathway agents are the current primary treatments, but they frequently fail to prohibit brain damage. Orthotopic liver transplantation cures the hyperammonemia of urea cycle disorders, but organ availability is limited and the procedure is highly invasive and requires life-long immunosuppression. A drug that could repair or stimulate a dysfunctional urea cycle such as this would have several advantages over current therapy. A drug called N-carbamyl-L-glutamate, Carglumic acid (NCG or Carbaglu)has recently been found to be virtually curative of another urea cycle defect called NAGS deficiency. In this disorder, treatment with NCG alone normalizes ureagenesis, blood ammonia and glutamine levels, allows normal protein tolerance and restores health. Knowledge from this study is being applied to acquired hyperammonemia, specifically in patients with propionic PA and MMA, to try and improve neurodevelopmental outcomes by improving the hyperammonemia. Aims: The overall objective of this project is to determine whether treatment of acute hyperammonemia with Carglumic acid in propionic acidemia (PA), methylmalonic acidemia (MMA) changes the long-term outcome of disease and to determine if it is effective in restoring urine ammonia levels to normal levels.

Clinical Trial Conditions for Carbaglu

Condition Name

Condition Name for Carbaglu
Intervention	Trials
Methylmalonic Acidemia	4
Propionic Acidemia	3
Inborn Errors of Metabolism	2
Propionic Acidemia, Type I and/or Type II	1

Condition MeSH

Condition MeSH for Carbaglu
Intervention	Trials
Propionic Acidemia	5
Acidosis	5
Amino Acid Metabolism, Inborn Errors	4
Metabolism, Inborn Errors	2

Clinical Trial Locations for Carbaglu

Trials by Country

Trials by Country for Carbaglu
Location	Trials
United States	14
Taiwan	1
Saudi Arabia	1

Trials by US State

Trials by US State for Carbaglu
Location	Trials
District of Columbia	3
Pennsylvania	2
Ohio	2
Massachusetts	2
Colorado	2

Clinical Trial Progress for Carbaglu

Clinical Trial Phase

Clinical Trial Phase for Carbaglu
Clinical Trial Phase	Trials
Phase 3	1
Phase 2/Phase 3	1
Phase 2	3

Clinical Trial Status

Clinical Trial Status for Carbaglu
Clinical Trial Phase	Trials
Completed	2
Enrolling by invitation	1
Terminated	1

Clinical Trial Sponsors for Carbaglu

Sponsor Name

Sponsor Name for Carbaglu
Sponsor	Trials
Mendel Tuchman	3
Children's Hospital of Philadelphia	3
University of California, Los Angeles	2

Sponsor Type

Sponsor Type for Carbaglu
Sponsor	Trials
Other	26
NIH	2